An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs

<!DOCTYPE html> <html lang="en" > <head itemscope itemtype="http://schema.org/WebPage" prefix="og: http://ogp.me/ns#" > <meta charset="UTF-8"> <meta http-equiv="X-UA-Compatible" content="IE=edge">  <meta name="HandheldFriendly" content="True"> <meta name="MobileOptimized" content="320"> <meta name="viewport" content="width=device-width, initial-scale=1.0"> <link rel="preconnect" href="https://cdn.ncbi.nlm.nih.gov"> <link rel="preconnect" href="https://www.ncbi.nlm.nih.gov"> <link rel="preconnect" href="https://www.google-analytics.com"> <link rel="stylesheet" href="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/CACHE/css/output.5ecf62baa0fa.css" type="text/css"> <link rel="stylesheet" href="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/CACHE/css/output.452c70ce66f7.css" type="text/css"> <link rel="stylesheet" href="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/CACHE/css/output.7bf600913d6e.css" type="text/css"> <link rel="stylesheet" href="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/CACHE/css/output.0bfa59184c7a.css" type="text/css"> <link rel="stylesheet" href="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/CACHE/css/output.b576742ffc3e.css" type="text/css"> <title>An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs - PubMed</title> <link rel="preload" as="font" href="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/uswds/dist/fonts/merriweather-bold-webfont.woff2" type="font/woff2" crossorigin />  <link rel="shortcut icon" type="image/ico" href="https://cdn.ncbi.nlm.nih.gov/coreutils/nwds/img/favicons/favicon.ico" /> <link rel="icon" type="image/png" href="https://cdn.ncbi.nlm.nih.gov/coreutils/nwds/img/favicons/favicon.png" />  <link rel="icon" type="image/png" sizes="192x192" href="https://cdn.ncbi.nlm.nih.gov/coreutils/nwds/img/favicons/favicon-192.png" />  <link rel="apple-touch-icon-precomposed" href="https://cdn.ncbi.nlm.nih.gov/coreutils/nwds/img/favicons/favicon-57.png">  <link rel="apple-touch-icon-precomposed" sizes="72x72" href="https://cdn.ncbi.nlm.nih.gov/coreutils/nwds/img/favicons/favicon-72.png">  <link rel="apple-touch-icon-precomposed" sizes="114x114" href="https://cdn.ncbi.nlm.nih.gov/coreutils/nwds/img/favicons/favicon-114.png">  <link rel="apple-touch-icon-precomposed" sizes="144x144" href="https://cdn.ncbi.nlm.nih.gov/coreutils/nwds/img/favicons/favicon-144.png">  <meta name="ncbi_sg_optimize_id" content="">  <meta name="theme-color" content="#20558a">  <meta name="referrer" content="origin-when-cross-origin"> <meta name="ncbi_pinger_gtm_track" content="true"/>  <meta name="ncbi_app" content="pubmed" /> <meta name="ncbi_db" content="pubmed" /> <meta name="ncbi_phid" content="3A7F0001B04DAA9500002B5E40FADCA1.1.m_6" /> <meta name="ncbi_pinger_stat_url" content="https://www.ncbi.nlm.nih.gov/stat" /> <meta name="log_category" content="literature" /> <meta name="ncbi_cost_center" content="pubmed" />  <meta name="ncbi_pdid" content="abstract" /> <meta name="log_op" content="retrieve" /> <meta name="uid" content="39524975" /> <meta name="ncbi_uid" content="39524975" /> <meta name="log_displayeduids" content="39524975" /> <meta name="log_source_db" content="pubmed,pmc" /> <meta name="log_icons_present" content="true" />  <meta name="description" content="Adrenomyeloneuropathy is a progressive neurodegenerative disease caused by pathogenic variants in the <i>ABCD1</i> gene, resulting in very-long-chain fatty acid (VLCFA) accumulation that leads to dying-back axonopathy. Our candidate gene therapy, SBT101 (AAV9-human <i>ABCD1</i> [h<i>ABCD1</i>]), aim …"><meta name="keywords" content="pmid:39524975, PMC11550353, doi:10.1016/j.omtm.2024.101354, Vidyullatha Vasireddy, Casey A Maguire, Karen Kozarsky, PubMed Abstract, NIH, NLM, NCBI, National Institutes of Health, National Center for Biotechnology Information, National Library of Medicine, MEDLINE"><meta name="robots" content="index,nofollow,noarchive"><meta property="og:title" content="An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs - PubMed"><meta property="og:url" content="https://pubmed.ncbi.nlm.nih.gov/39524975/"><meta property="og:description" content="Adrenomyeloneuropathy is a progressive neurodegenerative disease caused by pathogenic variants in the <i>ABCD1</i> gene, resulting in very-long-chain fatty acid (VLCFA) accumulation that leads to dying-back axonopathy. Our candidate gene therapy, SBT101 (AAV9-human <i>ABCD1</i> [h<i>ABCD1</i>]), aim …"><meta property="og:image" content="https://cdn.ncbi.nlm.nih.gov/pubmed/persistent/pubmed-meta-image-v2.jpg"><meta property="og:image:secure_url" content="https://cdn.ncbi.nlm.nih.gov/pubmed/persistent/pubmed-meta-image-v2.jpg"><meta property="og:type" content="website"><meta property="og:site_name" content="PubMed"><meta name="twitter:domain" content="pubmed.ncbi.nlm.nih.gov"><meta name="twitter:card" content="summary_large_image"><meta name="twitter:title" content="An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs - PubMed"><meta name="twitter:url" content="https://pubmed.ncbi.nlm.nih.gov/39524975/"><meta name="twitter:description" content="Adrenomyeloneuropathy is a progressive neurodegenerative disease caused by pathogenic variants in the <i>ABCD1</i> gene, resulting in very-long-chain fatty acid (VLCFA) accumulation that leads to dying-back axonopathy. Our candidate gene therapy, SBT101 (AAV9-human <i>ABCD1</i> [h<i>ABCD1</i>]), aim …"><meta name="twitter:image" content="https://cdn.ncbi.nlm.nih.gov/pubmed/persistent/pubmed-meta-image-v2.jpg">  <link rel="search" type="application/opensearchdescription+xml" href="https://cdn.ncbi.nlm.nih.gov/pubmed/persistent/opensearch.xml" title="PubMed search">  <noscript> <link rel="stylesheet" type="text/css" href="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/core/no-script.css"> </noscript> <meta name="citation_title" content="An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs"> <meta name="citation_authors" content="Vasireddy V;Maguire CA;Anderson DW;Ng C;Gong Y;Eichler F;Fourcade S;Guilera C;Onieva A;Sanchez A;Leal-Julià M;Verdés S;Dijkstra IME;Kemp S;Park H;Lutz T;Clark SW;Bosch A;Pujol A;Kozarsky K;"> <meta name="citation_date" content="10/09/2024"> <meta name="citation_publisher" content="Mol Ther Methods Clin Dev"> <meta name="citation_journal_title" content="Molecular therapy. Methods & clinical development"> <meta name="citation_pmid" content="39524975"> <meta name="citation_abstract_html_url" content="https://pubmed.ncbi.nlm.nih.gov/39524975/"> <meta name="citation_volume" content="32"> <meta name="citation_issue" content="4"> <meta name="citation_doi" content="10.1016/j.omtm.2024.101354"> <meta name="citation_issn" content="2329-0501"> <link rel="canonical" href="https://pubmed.ncbi.nlm.nih.gov/39524975/" /> </head> <body > <noscript> <div class="no-script-banner" id="no-script-banner"> <div class="warning-message"> <div class="warning-message-text"> This site needs JavaScript to work properly. Please enable it to take advantage of the complete set of features! </div> </div> </div> </noscript> <div class="no-session-banner" id="no-session-banner" hidden> <div class="warning-message"> <div class="warning-message-text"> Clipboard, Search History, and several other advanced features are temporarily unavailable. </div> <button class="close-banner-button" title="Close Clipboard and Search History not available warning banner" ref="linksrc=close_no_session_banner" aria-controls="no-session-banner" aria-label="Close Clipboard and Search History not available warning banner" aria-expanded="true"> </button> </div> </div> <a class="usa-skipnav" href="#article-details"> Skip to main page content </a>  <section class="usa-banner"> <div class="usa-accordion"> <header class="usa-banner-header"> <div class="usa-grid usa-banner-inner"> <img src="https://www.ncbi.nlm.nih.gov/coreutils/uswds/img/favicons/favicon-57.png" alt="U.S. flag" /> <p>An official website of the United States government</p> <button class="usa-accordion-button usa-banner-button" aria-expanded="false" aria-controls="gov-banner-top" > <span class="usa-banner-button-text">Here's how you know</span> </button> </div> </header> <div class="usa-banner-content usa-grid usa-accordion-content" id="gov-banner-top" > <div class="usa-banner-guidance-gov usa-width-one-half"> <img class="usa-banner-icon usa-media_block-img" src="https://www.ncbi.nlm.nih.gov/coreutils/uswds/img/icon-dot-gov.svg" alt="Dot gov" /> <div class="usa-media_block-body"> <p> <strong>The .gov means it’s official.</strong> <br /> Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site. </p> </div> </div> <div class="usa-banner-guidance-ssl usa-width-one-half"> <img class="usa-banner-icon usa-media_block-img" src="https://www.ncbi.nlm.nih.gov/coreutils/uswds/img/icon-https.svg" alt="Https" /> <div class="usa-media_block-body"> <p> <strong>The site is secure.</strong> <br /> The <strong>https://</strong> ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. </p> </div> </div> </div> </div> </section> <div class="usa-overlay"></div> <header class="ncbi-header" role="banner" data-section="Header"> <div class="usa-grid"> <div class="usa-width-one-whole"> <div class="ncbi-header__logo"> <a href="https://www.ncbi.nlm.nih.gov/" class="logo" aria-label="NCBI Logo" data-ga-action="click_image" data-ga-label="NIH NLM Logo"> <img src="https://www.ncbi.nlm.nih.gov/coreutils/nwds/img/logos/AgencyLogo.svg" alt="NIH NLM Logo" /> </a> </div> <div class="ncbi-header__account"> <a id="account_login" href="https://account.ncbi.nlm.nih.gov" class="usa-button header-button" style="display:none" data-ga-action="open_menu" data-ga-label="account_menu">Log in</a> <button id="account_info" class="header-button" style="display:none" aria-controls="account_popup"> <span class="fa fa-user" aria-hidden="true"></span> <span class="username desktop-only" aria-hidden="true" id="uname_short"></span> <span class="sr-only">Show account info</span> </button> </div> <div class="ncbi-popup-anchor"> <div class="ncbi-popup account-popup" id="account_popup" aria-hidden="true"> <div class="ncbi-popup-head"> <button class="ncbi-close-button" data-ga-action="close_menu" data-ga-label="account_menu"><span class="fa fa-times"></span><span class="usa-sr-only">Close</span></button> <h4>Account</h4> </div> <div class="account-user-info"> Logged in as:<br/> <b><span class="username" id="uname_long">username</span></b> </div> <div class="account-links"> <ul class="usa-unstyled-list"> <li><a id="account_myncbi" href="/myncbi/" class="set-base-url" data-ga-action="click_menu_item" data-ga-label="account_myncbi">Dashboard</a></li> <li><a id="account_pubs" href="/myncbi/collections/bibliography/" class="set-base-url" data-ga-action="click_menu_item" data-ga-label="account_pubs">Publications</a></li> <li><a id="account_settings" href="/account/settings/" class="set-base-url" data-ga-action="click_menu_item" data-ga-label="account_settings">Account settings</a></li> <li><a id="account_logout" href="/account/signout/" class="set-base-url" data-ga-action="click_menu_item" data-ga-label="account_logout">Log out</a></li> </ul> </div> </div> </div> </div> </div> </header> <div role="navigation" aria-label="access keys"> <a id="nws_header_accesskey_0" href="https://www.ncbi.nlm.nih.gov/guide/browsers/#ncbi_accesskeys" class="usa-sr-only" accesskey="0" tabindex="-1">Access keys</a> <a id="nws_header_accesskey_1" href="https://www.ncbi.nlm.nih.gov" class="usa-sr-only" accesskey="1" tabindex="-1">NCBI Homepage</a> <a id="nws_header_accesskey_2" href="/myncbi/" class="set-base-url usa-sr-only" accesskey="2" tabindex="-1">MyNCBI Homepage</a> <a id="nws_header_accesskey_3" href="#maincontent" class="usa-sr-only" accesskey="3" tabindex="-1">Main Content</a> <a id="nws_header_accesskey_4" href="#" class="usa-sr-only" accesskey="4" tabindex="-1">Main Navigation</a> </div> <section data-section="Alerts"> <div class="ncbi-alerts-placeholder"></div> </section>  <a id="maincontent" aria-label="Main page content below" role="navigation"></a> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <form action="/" method="get" autocomplete="off" class="usa-search usa-search-big search-form content-page-layout" id="search-form" role="search"> <div class="inner-wrap"> <a class="pubmed-logo" aria-label="Pubmed Logo" href="/" data-ga-category="featured_link" data-ga-action="pubmed_logo"> <img src="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/core/images/pubmed-logo-blue.svg" alt="pubmed logo" /> </a> <a href="#" class="search-input-trigger" data-alt-title="Hide search bar" title="Show search bar" aria-label="Show search bar"> </a> <div role="search" class="search-input"> <div class="form-field "> <label class="usa-sr-only" for="id_term"> Search: </label> <input type="search" name="term" data-skip-ie-scroll-to-top="" data-replace-term-with-exact="" data-exact-query="" placeholder="" class="term-input" required="required" id="id_term"/><a href="#" class="clear-btn" aria-label="Clear search input" title="Clear search input" role="button" data-pinger-ignore></a><button type="submit" class="search-btn" aria-label="Search" disabled data-pinger-ignore><span class="usa-search-submit-text">Search</span></button> </div> </div>   <div class="search-links-wrapper content-page-layout"> <div class="search-links"> <a href="/advanced/" class="search-input-link adv-search-link" data-ga-category="featured_link" data-ga-action="adv_search">Advanced</a> <a href="/clipboard/" class="search-input-link clipboard-link hidden" data-ga-category="featured_link" data-ga-action="clipboard"> Clipboard <span class="amount-in-clipboard"></span> </a> </div> <span class="search-input-link user-guide-link" role="navigation" aria-label="User guide"> <a href="/help/" data-ga-category="featured_link" data-ga-action="user_guide"> User Guide </a> </span> </div> </div> </form> <div id="article-page-header"> <div id="article-top-actions-bar" class="article-top-actions-bar"> <div class="inner-wrap"> <div class="actions-container"> <div class="return-to-search"> </div> <div class="multiple-results-actions " role="region" aria-label="save, email, send to"> <button id="save-results-panel-trigger" type="button" class="save-results save-results-panel-trigger" aria-expanded="false" aria-controls="save-action-panel" data-ga-category="save_share" data-ga-action="save" data-ga-label="open"> Save </button><button id="email-results-panel-trigger" type="button" class="email-results email-results-panel-trigger" aria-expanded="false" aria-controls="email-action-panel" data-ga-category="save_share" data-ga-action="email" data-ga-label="open"> Email </button><div class="more-actions dropdown-block"><button id="more-actions-trigger" type="button" class="trigger more-actions-trigger" ref="linksrc=show_moreactions_btn" aria-label="Send to" aria-controls="more-actions-dropdown">Send to</button><div id="more-actions-dropdown" class="dropdown dropdown-container" hidden><div class="content"><ul class="more-actions-links"><li><a class="clipboard-panel-trigger dropdown-block-link " href="#" role="button" data-ga-category="save_share" data-ga-action="clipboard" data-ga-label="send"> Clipboard </a></li><li><a role="button" class="dropdown-block-link bibliography-trigger-target" href="https://account.ncbi.nlm.nih.gov/?back_url=https%3A%2F%2Fpubmed.ncbi.nlm.nih.gov%2F39524975%2F%23open-bibliography-panel">My Bibliography</a></li><li><a role="button" class="dropdown-block-link collections-trigger-target" href="https://account.ncbi.nlm.nih.gov/?back_url=https%3A%2F%2Fpubmed.ncbi.nlm.nih.gov%2F39524975%2F%23open-collections-panel">Collections</a></li><li><a role="button" class="citation-manager-panel-trigger dropdown-block-link citation-manager-trigger-target" href="#">Citation manager</a></li></ul></div></div></div> </div> <div class="display-options" data-format-key="format" data-default-format-value="abstract" data-current-format-value="abstract" role="region"> <button class="trigger usa-button" title="Change format" data-ga-category="display_options" data-ga-action="display_options_dialog"> <span class="button-label">Display options</span> </button> <div class="dropdown" hidden> <div class="title"> Display options </div> <div class="content"> <div class="format-container"> <span class="option-label"> Format </span> <select name="format" data-initial-value="abstract" aria-label="Display format" id="article-display-format"> <option value="abstract">Abstract</option> <option value="pubmed">PubMed</option> <option value="pmid">PMID</option> </select> </div> </div> </div> </div> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> </div> </div> </div> <div id="article-panels-container" class="article-panels-container"> <div id="save-action-panel" class="save-action-panel action-panel" aria-hidden="true"> <div class="inner-wrap"> <h2 class="action-panel-heading"> Save citation to file </h2> <form id="save-action-panel-form" class="action-panel-content action-form" action="/results-export-ids/" data-by-search-action="/results-export-search-data/" data-by-ids-action="/results-export-ids/" method="post" data-by-search-method="post" data-by-ids-method="post"> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <div class="action-panel-control-wrap"> <label for="save-action-format" class="action-panel-label"> Format: </label> <select id="save-action-format" name="results-format" class="action-panel-selector"> <option value="summary-text">Summary (text)</option> <option value="pubmed-text">PubMed</option> <option value="pmid">PMID</option> <option value="abstract">Abstract (text)</option> <option value="csv">CSV</option> </select> </div> <div class="action-panel-actions"> <button class="action-panel-submit" type="submit" data-loading-label="Saving..." data-ga-category="save_share" data-ga-action="save" data-ga-label="save"> Create file </button> <button class="action-panel-cancel" aria-label="Close 'Save citations to file' panel" ref="linksrc=close_save_panel" aria-controls="save-action-panel" aria-expanded="false" data-ga-category="save_share" data-ga-action="save" data-ga-label="cancel"> Cancel </button> </div> </form> </div> </div> <div id="email-action-panel" class="email-action-panel action-panel" aria-hidden="true" data-recaptcha-script-url="https://www.recaptcha.net/recaptcha/enterprise.js?render=explicit&onload=onloadCaptchaCallback" data-recaptcha-enabled="true"> <div class="inner-wrap"> <h2 class="action-panel-heading"> Email citation </h2> <form id="email-action-panel-form" class="action-panel-content action-form" action="/send-email/" data-by-search-action="/results-export-email-by-search-data/" data-by-ids-action="/send-email/" data-by-search-method="post" data-by-ids-method="post" method="post" > <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <div class="action-panel-control-wrap"> <span class="action-panel-label"> Subject: </span> <span class="email-subject" id="email-subject" aria-label="Email subject">1 selected item: 39524975 - PubMed</span> <input type="hidden" name="email-subject" value="1 selected item: 39524975"/> <input type="hidden" name="email-subject-hash" value="debba11f40a14dce75ab702ad6cf00f0e3e1f1ffcf813e9780388bb6a75ce0ac"/> </div> <div class="action-panel-control-wrap"> <label for="email-to" class="action-panel-label required-field-asterisk"> To: </label> <input type="email" aria-label="Recipient Email Address" placeholder="email@example.com" id="email-to" class="email-to" pattern="^[a-zA-Z0-9_.+-]+@[a-zA-Z0-9-]+\.[a-zA-Z0-9-.]+$" maxlength="100" required> </div> <div class="action-panel-control-wrap"> <label for="email-from" class="action-panel-label"> From: </label> <input type="email" aria-label="Sender Email Address" placeholder="email@example.com" id="email-from" class="email-from" pattern="^[a-zA-Z0-9_.+-]+@[a-zA-Z0-9-]+\.[a-zA-Z0-9-.]+$" maxlength="256"> </div> <div class="action-panel-control-wrap"> <label for="email-citation-format" class="action-panel-label"> Format: </label> <select id="email-citation-format" name="citation-format" class="action-panel-selector email-citation-format"> <option selected="selected" value="summary">Summary</option> <option value="summary-text">Summary (text)</option> <option value="abstract">Abstract</option> <option value="abstract-text">Abstract (text)</option> </select> </div> <div class="include-supplemental-container"> <input type="checkbox" aria-label="Include MeSH and other data" name="include-supplemental" id="email-include-supplemental" class="email-include-supplemental"> <label for="email-include-supplemental" class="email-include-supplemental-label">MeSH and other data</label> </div> <div class="form-field recaptcha "> <div class="g-recaptcha" id="id-recaptcha" data-sitekey="6LfsWHMdAAAAAClKbtOpjQ2pMjgsGxvv7NdZW9uI"></div> </div> <div id="captcha-error-message" class="usa-input-error-message captcha-validation-message" role="alert"></div> <div class="action-panel-actions"> <button class="action-panel-submit" type="submit" data-loading-label="Sending..." data-ga-category="save_share" data-ga-action="email" data-ga-label="send"> Send email </button> <button class="action-panel-cancel" aria-label="Close 'Email citations' panel" ref="linksrc=close_email_panel" aria-controls="email-action-panel" aria-expanded="false" data-ga-category="save_share" data-ga-action="email" data-ga-label="cancel"> Cancel </button> </div> <input type="hidden" name="email-search-details" value="" /> <input type="hidden" name="email-search-details-hash" value="0e42663a6c3bd85498fcb88798998fed7bfdc45d457db35281e41afe13cc0524" /> </form> </div> </div> <div id="collections-action-panel" class="collections-action-panel action-panel in-progress-dots-panel" aria-hidden="true" data-collections-open-panel-enabled="false" data-collections-open-panel-url-hash="#open-collections-panel"> <div class="inner-wrap"> <h3 class="action-panel-heading"> Add to Collections </h3> <form id="collections-action-panel-form" class="collections-action-panel-form action-panel-content action-form action-panel-smaller-selectors" data-existing-collections-url="/list-existing-collections/" data-add-to-existing-collection-url="/add-to-existing-collection/" data-create-and-add-to-new-collection-url="/create-and-add-to-new-collection/" data-get-article-ids-by-search-url="/get-article-ids-by-search/" data-myncbi-max-collection-name-length="100" data-add-to-collection-max-amount="1000" data-collections-root-url="https://www.ncbi.nlm.nih.gov/myncbi/collections/"> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <div class="choice-group" role="radiogroup"> <ul class="radio-group-items"> <li> <input type="radio" id="collections-action-panel-new" class="collections-new" name="collections" value="new" data-ga-category="save_share" data-ga-action="collections" data-ga-label="collections_radio_new"> <label for="collections-action-panel-new">Create a new collection</label> </li> <li> <input type="radio" id="collections-action-panel-existing" class="collections-existing" name="collections" value="existing" checked="true" data-ga-category="save_share" data-ga-action="collections" data-ga-label="collections_radio_existing"> <label for="collections-action-panel-existing">Add to an existing collection</label> </li> </ul> </div> <div class="controls-wrapper"> <div class="action-panel-control-wrap new-collections-controls"> <label for="collections-action-panel-add-to-new" class="action-panel-label required-field-asterisk"> Name your collection: </label> <input type="text" name="add-to-new-collection" id="collections-action-panel-add-to-new" class="collections-action-add-to-new" pattern="[^"&=<>\/]*" title="The following characters are not allowed in the Name field: "&=<>/" maxlength="100" data-ga-category="save_share" data-ga-action="create_collection" data-ga-label="non_favorties_collection"> <div class="collections-new-name-too-long usa-input-error-message selection-validation-message"> Name must be less than 100 characters </div> </div> <div class="action-panel-control-wrap existing-collections-controls"> <label for="collections-action-panel-add-to-existing" class="action-panel-label"> Choose a collection: </label> <select id="collections-action-panel-add-to-existing" class="action-panel-selector collections-action-add-to-existing" data-ga-category="save_share" data-ga-action="select_collection" data-ga-label="($('#collections-action-add-to-existing').val() === 'Favorites') ? 'Favorites' : 'non_favorites_collection'"> </select> <div class="collections-retry-load-on-error usa-input-error-message selection-validation-message"> Unable to load your collection due to an error<br> <a href="#">Please try again</a> </div> </div> </div> <div class="action-panel-actions"> <button class="action-panel-submit" type="submit" data-loading-label="Adding..." data-pinger-ignore data-ga-category="save_share" data-ga-action="collections" data-ga-label="add"> Add </button> <button class="action-panel-cancel" aria-label="Close 'Add to Collections' panel" ref="linksrc=close_collections_panel" aria-controls="collections-action-panel" aria-expanded="false" data-ga-category="save_share" data-ga-action="collections" data-ga-label="cancel"> Cancel </button> </div> </form> </div> </div> <div id="bibliography-action-panel" class="bibliography-action-panel action-panel in-progress-dots-panel" aria-hidden="true" data-bibliography-open-panel-enabled="false" data-bibliography-open-panel-url-hash="#open-bibliography-panel"> <div class="inner-wrap"> <h3 class="action-panel-heading"> Add to My Bibliography </h3> <form id="bibliography-action-panel-form" class="bibliography-action-panel-form action-panel-content action-form action-panel-smaller-selectors" data-add-to-bibliography-max-amount="100" data-add-to-bibliography-batch-size="10" data-bibliography-delegates-url="/list-bibliography-delegates/" data-add-to-bibliography-url="/add-to-bibliography/" data-get-article-ids-by-search-url="/get-article-ids-by-search/" data-mybib-root-url="https://www.ncbi.nlm.nih.gov/myncbi/collections/mybibliography/"> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <div class="action-panel-control-wrap bibliographies-controls"> <div class="choice-group"> <ul class="bibliographies-action-add radio-group-items"> <li> <input name="bibliography" id="my-bibliography" class="my-bibliography" type="radio" checked/> <label for="my-bibliography">My Bibliography</label> </li> </ul> </div> </div> <div class="bibliographies-retry-load-on-error usa-input-error-message selection-validation-message"> Unable to load your delegates due to an error<br> <a href="#">Please try again</a> </div> <div class="action-panel-actions"> <button class="action-panel-submit" type="submit" data-loading-label="Adding..." data-pinger-ignore> Add </button> <button class="action-panel-cancel" aria-label="Close 'Add to bibliography' panel" ref="linksrc=close_bibliography_panel" aria-controls="bibliography-action-panel" aria-expanded="false" data-ga-category="save_share" data-ga-action="mybib" data-ga-label="cancel"> Cancel </button> </div> </form> </div> </div> <div id="saved-search-action-panel" class="saved-search-action-panel action-panel " aria-hidden="true" data-saved-search-open-panel-enabled="false" data-saved-search-open-panel-url-hash="#open-saved-search-panel"> <div class="inner-wrap"> <h2 class="action-panel-heading"> Your saved search </h2> <form id="saved-search-action-panel-form" class="saved-search-action-panel-form action-panel-content action-form" data-create-saved-search-url="/create-saved-search/" data-try-search-terms-url="/try-search-term/" data-saved-search-root-url="https://www.ncbi.nlm.nih.gov/myncbi/searches/"> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <div class="action-panel-control-wrap"> <label for="saved-search-name" class="action-panel-label saved-search-name-label required-field-asterisk"> Name of saved search: </label> <input maxlength="200" type="text" name="saved-search-name" id="saved-search-name" class="saved-search-name" value="" required pattern="[^"&=<>\/]*" title="The following characters are not allowed in the Name field: "&=<>/"> </div> <div class="action-panel-control-wrap"> <label for="saved-search-term" class="action-panel-label required-field-asterisk"> Search terms: </label> <textarea name="saved-search-term" id="saved-search-term" class="saved-search-term" required></textarea> </div> <div class="test-search-term-wrap"> <a href="#" class="try-search-term">Test search terms</a> </div> <div class="choice-group action-panel-extra-margin-top"> <span class="action-panel-label" id="fieldset-label"> Would you like email updates of new search results? </span> <fieldset id="saved-search-alert" aria-describedby="fieldset-label"> <legend class="usa-sr-only">Saved Search Alert Radio Buttons</legend> <ul class="radio-group-items"> <li> <input type="radio" id="saved-search-alert-yes" class="saved-search-alert-yes" name="saved-search-alert" value="yes" checked> <label for="saved-search-alert-yes" class="action-panel-label">Yes</label> </li> <li> <input aria-label="No radio input" type="radio" id="saved-search-alert-no" class="saved-search-alert-no" name="saved-search-alert" value="no"> <label for="saved-search-alert-no" class="action-panel-label">No</label> </li> </ul> </fieldset> </div> <div class="alert-schedule-wrap"> <div class="action-panel-control-wrap"> <label class="action-panel-label"> Email: </label> <span aria-label="Email address" id="saved-search-email" class="action-panel-label"><span class="action-panel-label-bold"></span> (<a class="myncbi-account-settings" href="https://www.ncbi.nlm.nih.gov/account/settings/">change</a>)</span> </div> <div class="action-panel-control-wrap action-panel-extra-margin-top"> <label for="saved-search-frequency" class="action-panel-label"> Frequency: </label> <select id="saved-search-frequency" class="no-border-panel-selector saved-search-frequency"> <option value="monthly">Monthly</option> <option value="weekly">Weekly</option> <option value="daily">Daily</option> </select> </div> <div class="action-panel-control-wrap saved-search-monthly-additional"> <label for="saved-search-monthly-on-day" class="action-panel-label"> Which day? </label> <select id="saved-search-monthly-on-day" class="no-border-panel-selector"> <option value="Sunday">The first Sunday</option> <option value="Monday">The first Monday</option> <option value="Tuesday">The first Tuesday</option> <option value="Wednesday">The first Wednesday</option> <option value="Thursday">The first Thursday</option> <option value="Friday">The first Friday</option> <option value="Saturday">The first Saturday</option> <option value="day">The first day</option> <option value="weekday">The first weekday</option> </select> </div> <div class="action-panel-control-wrap saved-search-weekly-additional"> <label for="saved-search-weekly-on-day" class="action-panel-label"> Which day? </label> <select id="saved-search-weekly-on-day" class="no-border-panel-selector saved-search-weekly-on-day"> <option value="Sunday">Sunday</option> <option value="Monday">Monday</option> <option value="Tuesday">Tuesday</option> <option value="Wednesday">Wednesday</option> <option value="Thursday">Thursday</option> <option value="Friday">Friday</option> <option value="Saturday">Saturday</option> </select> </div> <div class="action-panel-control-wrap"> <label for="saved-search-report" class="action-panel-label"> Report format: </label> <select id="saved-search-report" class="no-border-panel-selector saved-search-report"> <option value="DocSum">Summary</option> <option value="DocSumText">Summary (text)</option> <option value="Abstract">Abstract</option> <option value="AbstractText">Abstract (text)</option> <option value="MEDLINE">PubMed</option> </select> </div> <div class="action-panel-control-wrap"> <label for="saved-search-amount" class="action-panel-label"> Send at most: </label> <select id="saved-search-amount" class="no-border-panel-selector saved-search-amount"> <option value="1">1 item</option> <option value="5" selected>5 items</option> <option value="10">10 items</option> <option value="20">20 items</option> <option value="50">50 items</option> <option value="100">100 items</option> <option value="200">200 items</option> </select> </div> <div> <input type="checkbox" id="saved-search-send-if-no-result" class="saved-search-send-if-no-result" name="saved-search-send-if-no-result"> <label for="saved-search-send-if-no-result" class="action-panel-label smaller-checkbox"> Send even when there aren't any new results </label> </div> <div class="action-panel-control-wrap option-text-in-email-wrap"> <label for="saved-search-email-text" class="action-panel-label"> Optional text in email: </label> <textarea name="saved-search-email-text" id="saved-search-email-text" class="saved-search-email-text"></textarea> </div> </div> <div class="action-panel-actions"> <button class="action-panel-submit" type="submit" data-loading-label="Saving..." data-ga-category="save_share" data-ga-action="alert" data-ga-label="save"> Save </button> <button class="action-panel-cancel" aria-label="Close 'Your saved search' panel" ref="linksrc=close_saved_search_panel" aria-controls="saved-search-action-panel" aria-expanded="false" data-ga-category="save_share" data-ga-action="alert" data-ga-label="cancel"> Cancel </button> </div> </form> </div> </div> <div id="citation-manager-action-panel" class="citation-manager-action-panel action-panel" aria-hidden="true"> <div class="inner-wrap"> <h2 class="action-panel-heading"> Create a file for external citation management software </h2> <form id="citation-manager-action-panel-form" class="action-panel-content action-form" action="/results-export-ids/" data-by-search-action="/results-export-search-data/" data-by-ids-action="/results-export-ids/" method="post" data-by-search-method="post" data-by-ids-method="post"> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <input name="results-format" type="hidden" value="pubmed"/> <div class="action-panel-actions"> <button class="action-panel-submit" type="submit" data-loading-label="Sending..." data-ga-category="save_share" data-ga-action="citation_manager" data-ga-label="save"> Create file </button> <button class="action-panel-cancel" aria-label="Close 'Send citations to citation manager' panel" ref="linksrc=close_citation_manager_panel" aria-controls="citation-manager-action-panel" aria-expanded="false" data-ga-category="save_share" data-ga-action="citation_manager" data-ga-label="cancel"> Cancel </button> </div> </form> </div> </div> <div id="rss-action-panel" class="rss-action-panel action-panel " aria-hidden="true"> <div class="inner-wrap"> <h2 class="action-panel-heading"> Your RSS Feed </h2> <form id="rss-action-panel-form" class="rss-action-panel-form action-panel-content action-form" data-create-rss-feed-url="/create-rss-feed-url/" data-search-form-term-value=""> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <div class="action-panel-control-wrap"> <label for="rss-name" class="action-panel-label required-field-asterisk"> Name of RSS Feed: </label> <input maxlength="200" placeholder="Name" type="text" name="rss-name" id="rss-name" class="rss-name" value='' required pattern="[^"&=<>\/]*" title="The following characters are not allowed in the Name field: "&=<>/"> </div> <div class="rss-limit-wrap"> <div class="action-panel-control-wrap action-panel-extra-margin-top"> <label for="rss-limit" class="action-panel-label"> Number of items displayed: </label> <select id="rss-limit" class="no-border-panel-selector rss-limit"> <option value="5">5</option> <option value="10">10</option> <option value="15" selected="selected">15</option> <option value="20">20</option> <option value="50">50</option> <option value="100">100</option> </select> </div> </div> <div class="action-panel-actions"> <button class="action-panel-submit" type="submit" data-loading-label="Creating..." data-ga-category="save_share" data-ga-action="alert" data-ga-label="save"> Create RSS </button> <button class="action-panel-cancel" aria-label="Close 'Your RSS' panel" ref="linksrc=close_rss_panel" aria-controls="rss-action-panel" aria-expanded="false" data-ga-category="save_share" data-ga-action="alert" data-ga-label="cancel"> Cancel </button> </div> <div class="action-panel-control-wrap rss-link-copy-wrap"> <label for="rss-link" class="usa-sr-only">RSS Link</label> <input placeholder="Your RSS Feed Link" type="text" name="rss-link" id="rss-link" class="rss-link" title="RSS Link"> <button type="button" disabled class="rss-link-copy-button disabled" data-ga-category="save_share" data-ga-action="rss" data-ga-label="copy"> Copy </button> </div> </form> </div> </div> </div> </div> <div class="article-page" id="article-page" data-article-pmid="39524975"> <aside class="page-sidebar"> <div class="inner-wrap"> <div class="full-text-links"> <div class="full-view"> <h3 class="title"> Full text links </h3> <div class="full-text-links-list"> <a class="link-item dialog-focus" href="https://linkinghub.elsevier.com/retrieve/pii/S2329-0501(24)00170-0" target="_blank" rel="noopener" ref="linksrc=fulltextorjournal_fulltext&is_pmc=False&PrId=3048&itool=Abstract-def&log$=linkouticon&uid=39524975&db=pubmed&nlmid=101624857" title="See full text options at Elsevier Science" data-ga-category="full_text" data-ga-action="Elsevier Science" data-ga-label="39524975" ><img src="https://cdn.ncbi.nlm.nih.gov/corehtml/query/egifs/https:--linkinghub.elsevier.com-ihub-images-celloa.png" alt="full text provider logo"><span class="text"> Elsevier Science </span></a><a class="link-item pmc " href="https://pmc.ncbi.nlm.nih.gov/articles/pmid/39524975/" target="_blank" rel="noopener" ref="linksrc=fulltextorjournal_fulltext&is_pmc=True&PrId=3494&itool=Abstract-def&log$=linkouticon&uid=39524975&db=pubmed&nlmid=101624857" title="Free full text at PubMed Central" data-ga-category="full_text" data-ga-action="PMC" data-ga-label="39524975" ><span class="text"> Free PMC article </span></a> </div> </div> <div class="short-view"> <a href="#" class="full-text-links-button full-text-links-dialog-trigger"> Full text links </a> </div> </div> <div class="actions-buttons sidebar"><h3 class="title">Actions</h3><div class="inner-wrap"><button class="citation-button citation-dialog-trigger" aria-label="Open dialog with citation text in different styles" data-ga-category="save_share" data-ga-action="cite" data-ga-label="open" data-all-citations-url="/39524975/citations/" data-citation-style="nlm" data-pubmed-format-link="/39524975/export/"><span class="button-label">Cite</span></button><link type="text/css" href="ncbi-overlay-block/src/overlay-block.css"><div class="collections-button-container" data-article-id="39524975" data-article-db="pubmed"><button class="collections-button collections-dialog-trigger" aria-label="Save article in MyNCBI collections." data-ga-category="collections_button" data-ga-action="click" data-ga-label="collections_button" data-collections-open-dialog-enabled="false" data-collections-open-dialog-url="https://account.ncbi.nlm.nih.gov/?back_url=https%3A%2F%2Fpubmed.ncbi.nlm.nih.gov%2F39524975%2F%23open-collections-dialog" data-in-collections="false"><span class="button-label">Collections</span></button><div class="overlay" role="dialog"><div id="collections-action-dialog" class="dialog collections-dialog" aria-hidden="true"><div class="title">Add to Collections</div><div class="collections-action-panel action-panel"><form id="collections-action-dialog-form" class="collections-action-panel-form action-panel-content action-form action-panel-smaller-selectors" data-existing-collections-url="/list-existing-collections/" data-add-to-existing-collection-url="/add-to-existing-collection/" data-create-and-add-to-new-collection-url="/create-and-add-to-new-collection/" data-myncbi-max-collection-name-length="100" data-collections-root-url="https://www.ncbi.nlm.nih.gov/myncbi/collections/"><input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"><div class="choice-group" role="radiogroup"><ul class="radio-group-items"><li><input type="radio" id="collections-action-dialog-new" class="collections-new" name="collections" value="new" data-ga-category="collections_button" data-ga-action="click" data-ga-label="collections_radio_new"><label for="collections-action-dialog-new">Create a new collection</label></li><li><input type="radio" id="collections-action-dialog-existing" class="collections-existing" name="collections" value="existing" checked="true" data-ga-category="collections_button" data-ga-action="click" data-ga-label="collections_radio_existing"><label for="collections-action-dialog-existing">Add to an existing collection</label></li></ul></div><div class="controls-wrapper"><div class="action-panel-control-wrap new-collections-controls"><label for="collections-action-dialog-add-to-new" class="action-panel-label required-field-asterisk"> Name your collection: </label><input type="text" name="add-to-new-collection" id="collections-action-dialog-add-to-new" class="collections-action-add-to-new" pattern="[^"&=<>\/]*" title="The following characters are not allowed in the Name field: "&=<>/" maxlength="100" data-ga-category="collections_button" data-ga-action="create_collection" data-ga-label="non_favorties_collection"><div class="collections-new-name-too-long usa-input-error-message selection-validation-message"> Name must be less than 100 characters </div></div><div class="action-panel-control-wrap existing-collections-controls"><label for="collections-action-dialog-add-to-existing" class="action-panel-label"> Choose a collection: </label><select id="collections-action-dialog-add-to-existing" class="action-panel-selector collections-action-add-to-existing" data-ga-category="collections_button" data-ga-action="select_collection" data-ga-label="($('.collections-action-add-to-existing').val() === 'Favorites') ? 'Favorites' : 'non_favorites_collection'"></select><div class="collections-retry-load-on-error usa-input-error-message selection-validation-message"> Unable to load your collection due to an error<br><a href="#">Please try again</a></div></div></div><div class="action-panel-actions"><button class="action-panel-submit" type="submit" data-loading-label="Adding..." data-pinger-ignore data-ga-category="collections_button" data-ga-action="click" data-ga-label="add"> Add </button><button class="action-panel-cancel" aria-label="Close 'Add to Collections' panel" ref="linksrc=close_collections_panel" aria-controls="collections-action-panel" aria-expanded="false" data-ga-category="collections_button" data-ga-action="click" data-ga-label="cancel"> Cancel </button></div></form></div></div></div></div><button class="more-actions-button more-actions-dialog-trigger" title="Open dialog with more actions to take" ref="linksrc=more_actions_btn"></button><div class="display-options" data-format-key="format" data-default-format-value="abstract" data-current-format-value="abstract" role="region"><button class="trigger usa-button" title="Change format" data-ga-category="display_options" data-ga-action="display_options_dialog"><span class="button-label">Display options</span></button><div class="dropdown" hidden><div class="title"> Display options </div><div class="content"><div class="format-container"><span class="option-label"> Format </span><select name="format" data-initial-value="abstract" aria-label="Display format" id="sidebar-actions-display-format"><option value="abstract">Abstract</option><option value="pubmed">PubMed</option><option value="pmid">PMID</option></select></div></div></div></div></div></div> <div class="social-sharing"> <h3 class="title"> Share </h3> <ul class="social-sharing-links usa-unstyled-list"> <li> <a href="http://twitter.com/intent/tweet?text=An%20in%20vitro%20and%20in%20vivo%20efficacy%20evaluation%20of%20gene%20therapy%20candidate%20SBT101%20in%20mouse%20models%20of%20adrenomyeloneuropat%E2%80%A6%20https%3A//pubmed.ncbi.nlm.nih.gov/39524975/" class="share-link share-link-twitter" rel="noreferrer noopener" target="Pubmed-Social-Media-Share-Popup" title="Share article on Twitter" data-ga-category="save_share" data-ga-action="social" data-ga-label="twitter"></a> </li> <li> <a href="http://www.facebook.com/sharer/sharer.php?u=https%3A//pubmed.ncbi.nlm.nih.gov/39524975/" class="share-link share-link-facebook" rel="noreferrer noopener" target="Pubmed-Social-Media-Share-Popup" title="Share article on Facebook" data-ga-category="save_share" data-ga-action="social" data-ga-label="fb"></a> </li> <li> <div class="share-permalink"> <button class="trigger" title="Show article permalink" ref="linksrc=show_permalink_btn"> </button> <div class="dropdown" hidden> <div class="title"> Permalink </div> <div class="content"> <input type="text" class="permalink-text" value="https://pubmed.ncbi.nlm.nih.gov/39524975/" aria-label="Article permalink"/><button class="permalink-copy-button usa-button-primary" title="Copy article permalink" data-ga-category="save_share" data-ga-action="link" data-ga-label="copy_link"><span class="button-title">Copy</span></button> </div> </div> </div> </li> </ul> </div> <nav class="page-navigator" role="navigation" aria-label="Page navigation"> <div class="full-view"> <h3 class="title"> Page navigation </h3> <ul class="items-list"> <li class="heading "> <a href="#heading" aria-label="navigate to Title & authors section" data-ga-category="page_nav" data-ga-action="heading"> Title & authors </a> </li> <li class="abstract "> <a href="#abstract" aria-label="navigate to Abstract section" data-ga-category="page_nav" data-ga-action="abstract"> Abstract </a> </li> <li class="conflict-of-interest "> <a href="#conflict-of-interest" aria-label="navigate to Conflict of interest statement section" data-ga-category="page_nav" data-ga-action="conflict-of-interest"> Conflict of interest statement </a> </li> <li class="figures "> <a href="#figures" aria-label="navigate to Figures section" data-ga-category="page_nav" data-ga-action="figures"> Figures </a> </li> <li class="similar "> <a href="#similar" aria-label="navigate to Similar articles section" data-ga-category="page_nav" data-ga-action="similar"> Similar articles </a> </li> <li class="references "> <a href="#references" aria-label="navigate to References section" data-ga-category="page_nav" data-ga-action="references"> References </a> </li> <li class="related-links "> <a href="#related-links" aria-label="navigate to Related information section" data-ga-category="page_nav" data-ga-action="related-links"> Related information </a> </li> <li class="linkout "> <a href="#linkout" aria-label="navigate to LinkOut - more resources section" data-ga-category="page_nav" data-ga-action="linkout"> LinkOut - more resources </a> </li> </ul> </div> <select class="short-view" autocomplete="off" title="navigate to selected section" aria-label="Navigate to page section"> <option value="heading">Title & authors</option> <option value="abstract">Abstract</option> <option value="conflict-of-interest">Conflict of interest statement</option> <option value="figures">Figures</option> <option value="similar">Similar articles</option> <option value="references">References</option> <option value="related-links">Related information</option> <option value="linkout">LinkOut - more resources</option> </select> </nav> </div> </aside> <main class="article-details" id="article-details">   <header class="heading" id="heading"> <div class="full-view" id="full-view-heading"> <div class="article-citation"> <div class="article-source"> <div class="journal-actions dropdown-block"><button id="full-view-journal-trigger" class="journal-actions-trigger trigger" ref="linksrc=journal_actions_btn" title="Molecular therapy. Methods & clinical development" tabindex="0" aria-controls="full-view-journal" aria-expanded="false" aria-label="Toggle dropdown menu for journal Molecular therapy. Methods & clinical development" data-pinger-ignore> Mol Ther Methods Clin Dev </button><div id="full-view-journal" class="journal-actions-dropdown dropdown dropdown-container" hidden aria-label="Dropdown menu for journal Molecular therapy. Methods & clinical development"><div class="title">Actions</div><div class="content"><ul class="journal-actions-links"><li><a class="search-in-pubmed-link dropdown-block-link" href="/?term=%22Mol+Ther+Methods+Clin+Dev%22%5Bjour%5D&sort=date&sort_order=desc" data-href="/?term=%22Mol+Ther+Methods+Clin+Dev%22%5Bjour%5D&sort=date&sort_order=desc" ref="linksrc=search_in_pubmed_journal_name_link&journal_abbrev=Mol Ther Methods Clin Dev" data-ga-category="search" data-ga-action="journal_link" data-ga-label="Mol Ther Methods Clin Dev"> Search in PubMed </a></li><li><a class="search-in-nlm-catalog-link dropdown-block-link" ref ="linksrc=search_in_nlm_catalog_link" href="https://www.ncbi.nlm.nih.gov/nlmcatalog?term=%22Mol+Ther+Methods+Clin+Dev%22%5BTitle+Abbreviation%5D" data-ga-category="search_catalog" data-ga-action="journal_link" data-ga-label="Mol Ther Methods Clin Dev"> Search in NLM Catalog </a></li><li><a class="add-to-search-link dropdown-block-link" ref ="linksrc=add_to_search_link" role="button" data-search-term='"Mol Ther Methods Clin Dev"[jour]' data-ga-category="search" data-ga-action="add_to_search" data-ga-label='"Mol Ther Methods Clin Dev"[jour]' href="#"> Add to Search </a></li></ul></div></div></div><span class="period">. </span><span class="cit">2024 Oct 9;32(4):101354.</span> </div> <span class="citation-doi"> doi: 10.1016/j.omtm.2024.101354. </span> <span class="secondary-date"> eCollection 2024 Dec 12. </span> </div> <h1 class="heading-title"> An <i>in vitro</i> and <i>in vivo</i> efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs </h1> <div class="inline-authors"> <div class="authors"> <div class="authors-list"> <span class="authors-list-item "><a class="full-name" href="/?term=Vasireddy+V&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Vidyullatha Vasireddy">Vidyullatha Vasireddy</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#full-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Maguire+CA&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Casey A Maguire">Casey A Maguire</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA." href="#full-view-affiliation-2" ref="linksrc=author_aff"> 2 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Harvard Medical School, Boston, MA, USA." href="#full-view-affiliation-3" ref="linksrc=author_aff"> 3 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Anderson+DW&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="David W Anderson">David W Anderson</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#full-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Ng+C&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Carrie Ng">Carrie Ng</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA." href="#full-view-affiliation-2" ref="linksrc=author_aff"> 2 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Harvard Medical School, Boston, MA, USA." href="#full-view-affiliation-3" ref="linksrc=author_aff"> 3 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Gong+Y&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Yi Gong">Yi Gong</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA." href="#full-view-affiliation-2" ref="linksrc=author_aff"> 2 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Harvard Medical School, Boston, MA, USA." href="#full-view-affiliation-3" ref="linksrc=author_aff"> 3 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Eichler+F&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Florian Eichler">Florian Eichler</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA." href="#full-view-affiliation-2" ref="linksrc=author_aff"> 2 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Harvard Medical School, Boston, MA, USA." href="#full-view-affiliation-3" ref="linksrc=author_aff"> 3 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Fourcade+S&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Stéphane Fourcade">Stéphane Fourcade</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain." href="#full-view-affiliation-4" ref="linksrc=author_aff"> 4 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain." href="#full-view-affiliation-5" ref="linksrc=author_aff"> 5 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Guilera+C&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Cristina Guilera">Cristina Guilera</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain." href="#full-view-affiliation-4" ref="linksrc=author_aff"> 4 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain." href="#full-view-affiliation-5" ref="linksrc=author_aff"> 5 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Onieva+A&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Andrea Onieva">Andrea Onieva</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#full-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#full-view-affiliation-7" ref="linksrc=author_aff"> 7 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Sanchez+A&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Angela Sanchez">Angela Sanchez</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#full-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#full-view-affiliation-7" ref="linksrc=author_aff"> 7 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Leal-Juli%C3%A0+M&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Marc Leal-Julià">Marc Leal-Julià</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#full-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#full-view-affiliation-7" ref="linksrc=author_aff"> 7 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Verd%C3%A9s+S&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Sergi Verdés">Sergi Verdés</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#full-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#full-view-affiliation-7" ref="linksrc=author_aff"> 7 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Dijkstra+IME&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Inge M E Dijkstra">Inge M E Dijkstra</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands." href="#full-view-affiliation-8" ref="linksrc=author_aff"> 8 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Kemp+S&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Stephan Kemp">Stephan Kemp</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands." href="#full-view-affiliation-8" ref="linksrc=author_aff"> 8 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Park+H&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="HongGeun Park">HongGeun Park</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#full-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Lutz+T&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Tiffany Lutz">Tiffany Lutz</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#full-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Clark+SW&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Sean W Clark">Sean W Clark</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#full-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Bosch+A&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Assumpció Bosch">Assumpció Bosch</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#full-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#full-view-affiliation-7" ref="linksrc=author_aff"> 7 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Institudo de Salud Carlos III (ISCIII), Madrid, Spain." href="#full-view-affiliation-9" ref="linksrc=author_aff"> 9 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Pujol+A&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Aurora Pujol">Aurora Pujol</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain." href="#full-view-affiliation-4" ref="linksrc=author_aff"> 4 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain." href="#full-view-affiliation-5" ref="linksrc=author_aff"> 5 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Catalan Institution of Research and Advanced Studies (ICREA), 08010 Barcelona, Catalonia, Spain." href="#full-view-affiliation-10" ref="linksrc=author_aff"> 10 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Kozarsky+K&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Karen Kozarsky">Karen Kozarsky</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#full-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup></span> </div> </div> </div> <div class="short-article-details"> Affiliations <button class="more-details" id="toggle-authors" data-alt-text="Collapse" aria-controls="expanded-authors" aria-expanded="false" data-pinger-ignore data-fetch-url="/39524975/long-authors/" data-id-prefix=""> <span class="title"> Expand </span> </button> </div> <div class="extended-article-details" id="expanded-authors" aria-hidden="true"> <div class="expanded-authors" id="full-view-expanded-authors"> <div class="affiliations"> <h3 class="title"> Affiliations </h3> <ul class="item-list"> <li data-affiliation-id="full-view-affiliation-1" id="full-view-affiliation-1" ><sup class="key">1</sup> SwanBio Therapeutics, Inc., Philadelphia, PA, USA.</li> <li data-affiliation-id="full-view-affiliation-2" id="full-view-affiliation-2" ><sup class="key">2</sup> Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.</li> <li data-affiliation-id="full-view-affiliation-3" id="full-view-affiliation-3" ><sup class="key">3</sup> Harvard Medical School, Boston, MA, USA.</li> <li data-affiliation-id="full-view-affiliation-4" id="full-view-affiliation-4" ><sup class="key">4</sup> Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain.</li> <li data-affiliation-id="full-view-affiliation-5" id="full-view-affiliation-5" ><sup class="key">5</sup> Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.</li> <li data-affiliation-id="full-view-affiliation-6" id="full-view-affiliation-6" ><sup class="key">6</sup> Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.</li> <li data-affiliation-id="full-view-affiliation-7" id="full-view-affiliation-7" ><sup class="key">7</sup> Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain.</li> <li data-affiliation-id="full-view-affiliation-8" id="full-view-affiliation-8" ><sup class="key">8</sup> Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands.</li> <li data-affiliation-id="full-view-affiliation-9" id="full-view-affiliation-9" ><sup class="key">9</sup> Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.</li> <li data-affiliation-id="full-view-affiliation-10" id="full-view-affiliation-10" ><sup class="key">10</sup> Catalan Institution of Research and Advanced Studies (ICREA), 08010 Barcelona, Catalonia, Spain.</li> </ul> </div> </div> </div> <ul class="identifiers" id="full-view-identifiers"> <li> <span class="identifier pubmed"> <span class="id-label"> PMID: </span> <strong class="current-id" title="PubMed ID">39524975</strong> </span> </li> <li> <span class="identifier pmc"> <span class="id-label"> PMCID: </span> <a class="id-link" target="_blank" rel="noopener" ref="linksrc=article_id_link&article_id=PMC11550353&id_type=PMC" href="https://pmc.ncbi.nlm.nih.gov/articles/PMC11550353/" data-ga-category="full_text" data-ga-action="PMCID" > PMC11550353 </a> </span> </li> <li> <span class="identifier doi"> <span class="id-label"> DOI: </span> <a class="id-link" target="_blank" rel="noopener" ref="linksrc=article_id_link&article_id=10.1016/j.omtm.2024.101354&id_type=DOI" href="https://doi.org/10.1016/j.omtm.2024.101354" data-ga-category="full_text" data-ga-action="DOI" > 10.1016/j.omtm.2024.101354 </a> </span> </li> </ul> <div class="in-clipboard-label " hidden> Item in Clipboard </div> </div> <div class="short-view" id="short-view-heading"> <h1 class="heading-title"> An <i>in vitro</i> and <i>in vivo</i> efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs </h1> <div class="short-citation"> <span class="authors-list"> <span class="authors-list-item "><span class="full-name">Vidyullatha Vasireddy</span><span class="citation-separator"> et al.</span></span> </span> <span class="citation-journal"> Mol Ther Methods Clin Dev<span class="citation-separator">.</span> </span> <span class="date"> <time class="citation-year" datetime="2024">2024</time><span class="citation-separator">.</span> </span> </div> <div class="more-article-details-section with-display-settings"> <button class="more-article-details" id="toggle-article-details" data-alt-text="Hide details" ref="linksrc=toggle_article_details" aria-controls="full-article-details" aria-expanded="false" data-fetch-url="/39524975/long-authors/" data-id-prefix=""> <span class="title">Show details</span> </button> <div class="display-options" data-format-key="format" data-default-format-value="abstract" data-current-format-value="abstract" role="region"> <button class="trigger usa-button" title="Change format" data-ga-category="display_options" data-ga-action="display_options_dialog"> <span class="button-label">Display options</span> </button> <div class="dropdown" hidden> <div class="title"> Display options </div> <div class="content"> <div class="format-container"> <span class="option-label"> Format </span> <select name="format" data-initial-value="abstract" aria-label="Display format" id="mobile-display-format"> <option value="abstract">Abstract</option> <option value="pubmed">PubMed</option> <option value="pmid">PMID</option> </select> </div> </div> </div> </div> </div> <div class="extended-article-details" id="full-article-details" aria-hidden="true"> <div class="article-citation"> <div class="article-source"> <div class="journal-actions dropdown-block"><button id="short-view-journal-trigger" class="journal-actions-trigger trigger" ref="linksrc=journal_actions_btn" title="Molecular therapy. Methods & clinical development" tabindex="0" aria-controls="short-view-journal" aria-expanded="false" aria-label="Toggle dropdown menu for journal Molecular therapy. Methods & clinical development" data-pinger-ignore> Mol Ther Methods Clin Dev </button><div id="short-view-journal" class="journal-actions-dropdown dropdown dropdown-container" hidden aria-label="Dropdown menu for journal Molecular therapy. Methods & clinical development"><div class="title">Actions</div><div class="content"><ul class="journal-actions-links"><li><a class="search-in-pubmed-link dropdown-block-link" href="/?term=%22Mol+Ther+Methods+Clin+Dev%22%5Bjour%5D&sort=date&sort_order=desc" data-href="/?term=%22Mol+Ther+Methods+Clin+Dev%22%5Bjour%5D&sort=date&sort_order=desc" ref="linksrc=search_in_pubmed_journal_name_link&journal_abbrev=Mol Ther Methods Clin Dev" data-ga-category="search" data-ga-action="journal_link" data-ga-label="Mol Ther Methods Clin Dev"> Search in PubMed </a></li><li><a class="search-in-nlm-catalog-link dropdown-block-link" ref ="linksrc=search_in_nlm_catalog_link" href="https://www.ncbi.nlm.nih.gov/nlmcatalog?term=%22Mol+Ther+Methods+Clin+Dev%22%5BTitle+Abbreviation%5D" data-ga-category="search_catalog" data-ga-action="journal_link" data-ga-label="Mol Ther Methods Clin Dev"> Search in NLM Catalog </a></li><li><a class="add-to-search-link dropdown-block-link" ref ="linksrc=add_to_search_link" role="button" data-search-term='"Mol Ther Methods Clin Dev"[jour]' data-ga-category="search" data-ga-action="add_to_search" data-ga-label='"Mol Ther Methods Clin Dev"[jour]' href="#"> Add to Search </a></li></ul></div></div></div><span class="period">. </span><span class="cit">2024 Oct 9;32(4):101354.</span> </div> <span class="citation-doi"> doi: 10.1016/j.omtm.2024.101354. </span> <span class="secondary-date"> eCollection 2024 Dec 12. </span> </div> <div class="expanded-authors" id="short-view-expanded-authors"> <div class="authors"> <h3 class="title"> Authors </h3> <div class="authors-list"> <span class="authors-list-item "><a class="full-name" href="/?term=Vasireddy+V&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Vidyullatha Vasireddy">Vidyullatha Vasireddy</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#short-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Maguire+CA&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Casey A Maguire">Casey A Maguire</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA." href="#short-view-affiliation-2" ref="linksrc=author_aff"> 2 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Harvard Medical School, Boston, MA, USA." href="#short-view-affiliation-3" ref="linksrc=author_aff"> 3 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Anderson+DW&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="David W Anderson">David W Anderson</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#short-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Ng+C&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Carrie Ng">Carrie Ng</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA." href="#short-view-affiliation-2" ref="linksrc=author_aff"> 2 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Harvard Medical School, Boston, MA, USA." href="#short-view-affiliation-3" ref="linksrc=author_aff"> 3 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Gong+Y&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Yi Gong">Yi Gong</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA." href="#short-view-affiliation-2" ref="linksrc=author_aff"> 2 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Harvard Medical School, Boston, MA, USA." href="#short-view-affiliation-3" ref="linksrc=author_aff"> 3 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Eichler+F&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Florian Eichler">Florian Eichler</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA." href="#short-view-affiliation-2" ref="linksrc=author_aff"> 2 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Harvard Medical School, Boston, MA, USA." href="#short-view-affiliation-3" ref="linksrc=author_aff"> 3 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Fourcade+S&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Stéphane Fourcade">Stéphane Fourcade</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain." href="#short-view-affiliation-4" ref="linksrc=author_aff"> 4 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain." href="#short-view-affiliation-5" ref="linksrc=author_aff"> 5 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Guilera+C&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Cristina Guilera">Cristina Guilera</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain." href="#short-view-affiliation-4" ref="linksrc=author_aff"> 4 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain." href="#short-view-affiliation-5" ref="linksrc=author_aff"> 5 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Onieva+A&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Andrea Onieva">Andrea Onieva</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#short-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#short-view-affiliation-7" ref="linksrc=author_aff"> 7 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Sanchez+A&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Angela Sanchez">Angela Sanchez</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#short-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#short-view-affiliation-7" ref="linksrc=author_aff"> 7 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Leal-Juli%C3%A0+M&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Marc Leal-Julià">Marc Leal-Julià</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#short-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#short-view-affiliation-7" ref="linksrc=author_aff"> 7 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Verd%C3%A9s+S&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Sergi Verdés">Sergi Verdés</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#short-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#short-view-affiliation-7" ref="linksrc=author_aff"> 7 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Dijkstra+IME&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Inge M E Dijkstra">Inge M E Dijkstra</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands." href="#short-view-affiliation-8" ref="linksrc=author_aff"> 8 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Kemp+S&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Stephan Kemp">Stephan Kemp</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands." href="#short-view-affiliation-8" ref="linksrc=author_aff"> 8 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Park+H&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="HongGeun Park">HongGeun Park</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#short-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Lutz+T&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Tiffany Lutz">Tiffany Lutz</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#short-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Clark+SW&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Sean W Clark">Sean W Clark</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#short-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Bosch+A&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Assumpció Bosch">Assumpció Bosch</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain." href="#short-view-affiliation-6" ref="linksrc=author_aff"> 6 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain." href="#short-view-affiliation-7" ref="linksrc=author_aff"> 7 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Institudo de Salud Carlos III (ISCIII), Madrid, Spain." href="#short-view-affiliation-9" ref="linksrc=author_aff"> 9 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Pujol+A&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Aurora Pujol">Aurora Pujol</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain." href="#short-view-affiliation-4" ref="linksrc=author_aff"> 4 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain." href="#short-view-affiliation-5" ref="linksrc=author_aff"> 5 </a><span class="author-sup-separator"> </span><a class="affiliation-link" title="Catalan Institution of Research and Advanced Studies (ICREA), 08010 Barcelona, Catalonia, Spain." href="#short-view-affiliation-10" ref="linksrc=author_aff"> 10 </a></sup><span class="comma">, </span></span><span class="authors-list-item "><a class="full-name" href="/?term=Kozarsky+K&cauthor_id=39524975" ref="linksrc=author_name_link" data-ga-category="search" data-ga-action="author_link" data-ga-label="Karen Kozarsky">Karen Kozarsky</a><sup class="affiliation-links"><span class="author-sup-separator"> </span><a class="affiliation-link" title="SwanBio Therapeutics, Inc., Philadelphia, PA, USA." href="#short-view-affiliation-1" ref="linksrc=author_aff"> 1 </a></sup></span> </div> </div> <div class="affiliations"> <h3 class="title"> Affiliations </h3> <ul class="item-list"> <li data-affiliation-id="short-view-affiliation-1" id="short-view-affiliation-1" ><sup class="key">1</sup> SwanBio Therapeutics, Inc., Philadelphia, PA, USA.</li> <li data-affiliation-id="short-view-affiliation-2" id="short-view-affiliation-2" ><sup class="key">2</sup> Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.</li> <li data-affiliation-id="short-view-affiliation-3" id="short-view-affiliation-3" ><sup class="key">3</sup> Harvard Medical School, Boston, MA, USA.</li> <li data-affiliation-id="short-view-affiliation-4" id="short-view-affiliation-4" ><sup class="key">4</sup> Neurometabolic Disease Laboratory, Instituto de Investigación Biomédica de Bellvitge (IDIBELL), Hospital Duran i Reynalds, Barcelona, Spain.</li> <li data-affiliation-id="short-view-affiliation-5" id="short-view-affiliation-5" ><sup class="key">5</sup> Centre for Biomedical Research on Rare Diseases (CIBERER), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.</li> <li data-affiliation-id="short-view-affiliation-6" id="short-view-affiliation-6" ><sup class="key">6</sup> Department of Biochemistry and Molecular Biology and Institute of Neurosciences, Universitat Autònoma de Barcelona, Barcelona, Spain.</li> <li data-affiliation-id="short-view-affiliation-7" id="short-view-affiliation-7" ><sup class="key">7</sup> Unitat Mixta UAB-VHIR, Vall D'Hebron Institut de Recerca (VHIR), Barcelona, Spain.</li> <li data-affiliation-id="short-view-affiliation-8" id="short-view-affiliation-8" ><sup class="key">8</sup> Laboratory Genetic Metabolic Diseases, Department of Laboratory Medicine, Amsterdam UMC location University of Amsterdam, Amsterdam Neuroscience, Amsterdam Gastroenterology Endocrinology Metabolism, Amsterdam, the Netherlands.</li> <li data-affiliation-id="short-view-affiliation-9" id="short-view-affiliation-9" ><sup class="key">9</sup> Centro de Investigación Biomédica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), Institudo de Salud Carlos III (ISCIII), Madrid, Spain.</li> <li data-affiliation-id="short-view-affiliation-10" id="short-view-affiliation-10" ><sup class="key">10</sup> Catalan Institution of Research and Advanced Studies (ICREA), 08010 Barcelona, Catalonia, Spain.</li> </ul> </div> </div> <ul class="identifiers" id="short-view-identifiers"> <li> <span class="identifier pubmed"> <span class="id-label"> PMID: </span> <strong class="current-id" title="PubMed ID">39524975</strong> </span> </li> <li> <span class="identifier pmc"> <span class="id-label"> PMCID: </span> <a class="id-link" target="_blank" rel="noopener" ref="linksrc=article_id_link&article_id=PMC11550353&id_type=PMC" href="https://pmc.ncbi.nlm.nih.gov/articles/PMC11550353/" data-ga-category="full_text" data-ga-action="PMCID" > PMC11550353 </a> </span> </li> <li> <span class="identifier doi"> <span class="id-label"> DOI: </span> <a class="id-link" target="_blank" rel="noopener" ref="linksrc=article_id_link&article_id=10.1016/j.omtm.2024.101354&id_type=DOI" href="https://doi.org/10.1016/j.omtm.2024.101354" data-ga-category="full_text" data-ga-action="DOI" > 10.1016/j.omtm.2024.101354 </a> </span> </li> </ul> <div class="in-clipboard-label " hidden> Item in Clipboard </div> </div> </div> </header> <div class="actions-buttons inline"><div class="inner-wrap"><button class="full-text-links-button full-text-links-dialog-trigger" title="Open dialog with full text links" ref="linksrc=full_text_links"> Full text links </button><button class="citation-button citation-dialog-trigger" aria-label="Open dialog with citation text in different styles" data-ga-category="save_share" data-ga-action="cite" data-ga-label="open" data-all-citations-url="/39524975/citations/" data-citation-style="nlm" data-pubmed-format-link="/39524975/export/"><span class="button-label">Cite</span></button><button class="more-actions-button more-actions-dialog-trigger" title="Open dialog with more actions to take" ref="linksrc=more_actions_btn"></button><div class="display-options" data-format-key="format" data-default-format-value="abstract" data-current-format-value="abstract" role="region"><button class="trigger usa-button" title="Change format" data-ga-category="display_options" data-ga-action="display_options_dialog"><span class="button-label">Display options</span></button><div class="dropdown" hidden><div class="title"> Display options </div><div class="content"><div class="format-container"><span class="option-label"> Format </span><select name="format" data-initial-value="abstract" aria-label="Display format" id="inline-actions-display-format"><option value="abstract">Abstract</option><option value="pubmed">PubMed</option><option value="pmid">PMID</option></select></div></div></div></div></div></div> <div class="abstract" id="abstract"> <h2 class="title"> Abstract </h2> <div class="abstract-content selected" id="eng-abstract"> <p> Adrenomyeloneuropathy is a progressive neurodegenerative disease caused by pathogenic variants in the <i>ABCD1</i> gene, resulting in very-long-chain fatty acid (VLCFA) accumulation that leads to dying-back axonopathy. Our candidate gene therapy, SBT101 (AAV9-human <i>ABCD1</i> [h<i>ABCD1</i>]), aims to ameliorate pathology by delivering functional copies of h<i>ABCD1</i> to the spinal cord. Transduced cells produce functional ABCD1 protein, thereby repairing the underlying biochemical defect. <i>In vitro</i> and <i>in vivo</i> mouse studies were conducted to assess the biochemical and functional efficacy of SBT101 and show effective delivery to target tissues involved in the disease pathology: spinal cord and dorsal root ganglia. Administration of SBT101 to mixed glial cell cultures from <i>Abcd1</i>-Null mice, and to male <i>Abcd1</i> knockout (<i>Abcd1</i> <sup><i>-/y</i></sup> ) and double-knockout (<i>Abcd1</i> <sup><i>-/y</i></sup> /<i>Abcd2</i> <sup><i>-/-</i></sup> ) mice led to increased hABCD1 production and reduced VLCFA. Double-knockout mice also exhibited improved grip strength. Furthermore, we conducted biodistribution and safety assessments in nonhuman primates. Six-hour intrathecal lumbar infusions demonstrated effective transduction throughout target tissues, supporting the clinical feasibility of the procedure. SBT101 was well tolerated, with no observed SBT101-related mortality or clinical signs. These findings not only provide preclinical efficacy data for SBT101 but also inform clinically relevant SBT101 dose selection for patients with adrenomyeloneuropathy. </p> </div> <p> <strong class="sub-title"> Keywords: </strong> SBT101; adeno-associated virus; adrenomyeloneuropathy; biodistribution; gene therapy; human ABCD1; in vivo mouse model; neurodegenerative disease; nonhuman primates; very-long-chain fatty acids. </p> </div> <p class="copyright" id="copyright"> © 2024 The Author(s). </p> <p class="disclaimer" id="disclaimer"> <a href="/disclaimer/" target="_blank" ga_category="literature_resources" ga_action="disclaimer_link" ga_label="under_abstract">PubMed Disclaimer</a> </p> <div class="conflict-of-interest" id="conflict-of-interest"> <h2 class="title"> Conflict of interest statement </h2> <div class="statement"> <p xmlns:xlink="http://www.w3.org/1999/xlink" xmlns:mml="http://www.w3.org/1998/Math/MathML" xmlns:p1="http://pubmed.gov/pub-one">T.L. and K.K. are employees and stock/shareholders of SwanBio Therapeutics. V.V., D.W.A., S.W.C., and H.P. were employees and stock/shareholders of SwanBio Therapeutics at the time the studies were performed: V.V. is an employee of Code Biotherapeutics; D.W.A. is an employee of Code Biotherapeutics; S.W.C. is an employee of Clithero-Clark Consulting, LLC; H.P. is an employee of Center for Breakthrough Medicines. C.A.M. has financial interests in/received consultancy fees from Chameleon Biosciences, Skylark Bio, and Sphere Gene Therapeutics; resequenceceived research support/grants from BridgeBio, SwanBio Therapeutics, and Waypoint Capital; and received royalties for licensing agreements from BridgeBio, Partners Healthcare, Skylark Bio, Sphere Gene Therapeutics, and SwanBio Therapeutics. Y.G. received royalties for licensing agreements from SwanBio Therapeutics. F.E. received research support/grants from Aspa Therapeutics, bluebird bio, Ionis Pharmaceuticals, Minoryx Therapeutics, and Sio Gene Therapies; received consultancy fees from Autobahn Therapeutics, Poxel, SwanBio Therapeutics, Takeda, Taysha, and UpToDate; and is a founder and stock/shareholder of SwanBio Therapeutics. A.P., A.B., and S.K. received unrestricted research support/grants and consultancy fees from SwanBio Therapeutics.</p> </div> </div> <div class="figures" id="figures"> <h2 class="title"> Figures </h2> <div class="figures-list" id="slides-container" itemscope itemtype="http://schema.org/ImageGallery"> <figure class="figure-item " itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="0" data-label-slug="captionless-figure"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/b0efa842e18a/fx1.jpg" itemprop="contentUrl" aria-describedby="figure-caption-0" role="button" data-image-width="664" data-image-height="664" data-image-alt="captionless-figure" data-pmc-id="PMC11550353" data-figure-id="undfig1"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-0" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/21a5de4fca45/fx1.gif" alt="None" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="664"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="664"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Graphical abstract </p> </strong> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Graphical abstract </p> </strong> </div> <figcaption id="figure-caption-0" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Graphical abstract </strong> </figcaption> </figure> <figure class="figure-item " itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="1" data-label-slug="figure-1"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/f4ebe1f7e2a4/gr1.jpg" itemprop="contentUrl" aria-describedby="figure-caption-1" role="button" data-image-width="741" data-image-height="317" data-image-alt="Figure 1" data-pmc-id="PMC11550353" data-figure-id="fig1"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-1" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/3fd007a4d913/gr1.gif" alt="Figure 1" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="741"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="317"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Figure 1 </p> </strong> <div class="figure-caption-contents"><p> Increased hABCD1 expression and VLCFA… </p></div> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Figure 1 </p> </strong> <div class="figure-caption-contents"><p> Increased hABCD1 expression and VLCFA reduction in mixed glial cell cultures following SBT101… </p></div> </div> <figcaption id="figure-caption-1" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Figure 1 </strong> <div class="figure-caption-contents">Increased hABCD1 expression and VLCFA reduction in mixed glial cell cultures following SBT101 administration Human ABCD1 expression (A) and VLCFA levels (B) in mixed glial cell cultures (from <i>Abcd1</i>-Null and WT mice) exposed to SBT101. ∗<i>p</i> < 0.05, ∗∗<i>p</i> < 0.01, ∗∗∗<i>p</i> < 0.0001. Data represent mean ±95% confidence interval.</div> </figcaption> </figure> <figure class="figure-item " itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="2" data-label-slug="figure-2"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/df792d61ec4e/gr2.jpg" itemprop="contentUrl" aria-describedby="figure-caption-2" role="button" data-image-width="741" data-image-height="606" data-image-alt="Figure 2" data-pmc-id="PMC11550353" data-figure-id="fig2"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-2" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/108c06ab68f3/gr2.gif" alt="Figure 2" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="741"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="606"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Figure 2 </p> </strong> <div class="figure-caption-contents"><p> Increased levels of hABCD1 and… </p></div> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Figure 2 </p> </strong> <div class="figure-caption-contents"><p> Increased levels of hABCD1 and mtDNA, and reduced VLCFA levels, in <i> Abcd1 </i> <sup> <i> −/y… </i> </sup> </p></div> </div> <figcaption id="figure-caption-2" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Figure 2 </strong> <div class="figure-caption-contents">Increased levels of hABCD1 and mtDNA, and reduced VLCFA levels, in <i>Abcd1</i><sup><i>−/y</i></sup> mice 8 weeks after SBT101 administration Viral genomes copy number (A), hABCD1 expression (B), VLCFA levels (C), and mtDNA levels (D) within the spinal cord of <i>Abcd1</i><sup><i>−/y</i></sup> mice assessed at 8 weeks after SBT101 administration. ∗<i>p</i> < 0.05, ∗∗<i>p</i> < 0.01, ∗∗∗<i>p</i> < 0.0001. Data represent mean ± 95% confidence interval.</div> </figcaption> </figure> <figure class="figure-item " itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="3" data-label-slug="figure-3"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/98e21844fa2e/gr3.jpg" itemprop="contentUrl" aria-describedby="figure-caption-3" role="button" data-image-width="741" data-image-height="1062" data-image-alt="Figure 3" data-pmc-id="PMC11550353" data-figure-id="fig3"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-3" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/0c0c98b251d7/gr3.gif" alt="Figure 3" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="741"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="1062"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Figure 3 </p> </strong> <div class="figure-caption-contents"><p> Increased levels of hABCD1 and… </p></div> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Figure 3 </p> </strong> <div class="figure-caption-contents"><p> Increased levels of hABCD1 and mtDNA, and reduced VLCFA levels in <i> Abcd1 </i> <sup> <i> −/y… </i> </sup> </p></div> </div> <figcaption id="figure-caption-3" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Figure 3 </strong> <div class="figure-caption-contents">Increased levels of hABCD1 and mtDNA, and reduced VLCFA levels in <i>Abcd1</i><sup><i>−/y</i></sup> mice at 13 and 24 weeks after SBT101 administration Viral genomes copy number (A and E), hABCD1 expression (B and F), VLCFA levels (C and G), and mtDNA levels (D and H) within the spinal cord of <i>Abcd1</i><sup><i>−/y</i></sup> mice assessed at 13 weeks (A–D) and 24 weeks (E–H) after SBT101 administration. ∗<i>p</i> < 0.05, ∗∗<i>p</i> < 0.01, ∗∗∗<i>p</i> < 0.0001. Data represent mean ± 95% confidence interval.</div> </figcaption> </figure> <figure class="figure-item " itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="4" data-label-slug="figure-4"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/085538a5ae22/gr4.jpg" itemprop="contentUrl" aria-describedby="figure-caption-4" role="button" data-image-width="741" data-image-height="571" data-image-alt="Figure 4" data-pmc-id="PMC11550353" data-figure-id="fig4"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-4" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/dd1a5ba44e0e/gr4.gif" alt="Figure 4" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="741"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="571"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Figure 4 </p> </strong> <div class="figure-caption-contents"><p> Biochemical improvements following SBT101 administration… </p></div> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Figure 4 </p> </strong> <div class="figure-caption-contents"><p> Biochemical improvements following SBT101 administration in <i> Abcd1 </i> <sup> <i> −/y </i> </sup> / <i> Abcd2 </i> <sup> <i> −/− </i> </sup> DKO mice… </p></div> </div> <figcaption id="figure-caption-4" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Figure 4 </strong> <div class="figure-caption-contents">Biochemical improvements following SBT101 administration in <i>Abcd1</i><sup><i>−/y</i></sup>/<i>Abcd2</i><sup><i>−/−</i></sup> DKO mice Viral genome distribution (A) and hABCD1 mRNA expression (B) in the spinal cord of <i>Abcd1</i><sup><i>−/y</i></sup>/<i>Abcd2</i><sup><i>−/−</i></sup> DKO mice at 18 months of age (approximately 11 months after SBT101 administration). VLCFA levels (C) and inflammatory cytokine TNF-α levels (D) in the lumbar spinal cord of <i>Abcd1</i><sup><i>−/y</i></sup>/<i>Abcd2</i><sup><i>−/−</i></sup> DKO mice at 18 months of age. ∗<i>p</i> < 0.05, ∗∗<i>p</i> < 0.01. Data represent mean ± 95% confidence interval.</div> </figcaption> </figure> <figure class="figure-item " itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="5" data-label-slug="figure-5"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/348b92bd73b6/gr5.jpg" itemprop="contentUrl" aria-describedby="figure-caption-5" role="button" data-image-width="650" data-image-height="816" data-image-alt="Figure 5" data-pmc-id="PMC11550353" data-figure-id="fig5"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-5" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/f741dc05f553/gr5.gif" alt="Figure 5" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="650"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="816"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Figure 5 </p> </strong> <div class="figure-caption-contents"><p> Improved grip strength in <i> Abcd1… </i> </p></div> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Figure 5 </p> </strong> <div class="figure-caption-contents"><p> Improved grip strength in <i> Abcd1 </i> <sup> <i> −/y </i> </sup> / <i> Abcd2 </i> <sup> <i> −/− </i> </sup> DKO mice after SBT101… </p></div> </div> <figcaption id="figure-caption-5" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Figure 5 </strong> <div class="figure-caption-contents">Improved grip strength in <i>Abcd1</i><sup><i>−/y</i></sup>/<i>Abcd2</i><sup><i>−/−</i></sup> DKO mice after SBT101 administration Four-paw grip strength in <i>Abcd1</i><sup><i>−/y</i></sup>/<i>Abcd2</i><sup><i>−/−</i></sup> DKO mice at 15 months of age. ∗<i>p</i> < 0.05. Data represent mean ± 95% confidence interval.</div> </figcaption> </figure> <figure class="figure-item tail" itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="6" data-label-slug="figure-6"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/5ee33b021265/gr6.jpg" itemprop="contentUrl" aria-describedby="figure-caption-6" role="button" data-image-width="726" data-image-height="467" data-image-alt="Figure 6" data-pmc-id="PMC11550353" data-figure-id="fig6"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-6" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/b32e55107bf2/gr6.gif" alt="Figure 6" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="726"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="467"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Figure 6 </p> </strong> <div class="figure-caption-contents"><p> Vector genome biodistribution in the… </p></div> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Figure 6 </p> </strong> <div class="figure-caption-contents"><p> Vector genome biodistribution in the spinal cord and DRG according to delivery method… </p></div> </div> <figcaption id="figure-caption-6" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Figure 6 </strong> <div class="figure-caption-contents">Vector genome biodistribution in the spinal cord and DRG according to delivery method in NHP study 1 Vector genome biodistribution in the spinal cord and DRG was assessed via ddPCR quantification of GFP copies (A) and via immunohistochemical quantification of neuron positivity (B). Plotted values represent mean ± 95% confidence interval. <i>n</i> = 3 NHPs per treatment group, and A1–A3 represent individual NHPs. Percentage neuron positivity is presented as grades 1–5: grade 1, <1%; grade 2, 1%–25%; grade 3, 26%–50%; grade 4, 51%–75%; grade 5, 76%–100%; blanks, unremarkable (no observable GFP immunoreactivity). C2, cervical section 2; ddPCR, droplet digital PCR; DRG, dorsal root ganglia; GFP, green fluorescent protein; <i>HPRT1</i>, hypoxanthine-guanine phosphoribosyltransferase 1; IT-C, intrathecal cervical; IT-L, intrathecal lumbar; L5, lumbar section 5; T5, thoracic section 5; vg/an, vector genomes/animal.</div> </figcaption> </figure> <figure class="figure-item tail" itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="7" data-label-slug="figure-7"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/16022fd7c454/gr7.jpg" itemprop="contentUrl" aria-describedby="figure-caption-7" role="button" data-image-width="726" data-image-height="856" data-image-alt="Figure 7" data-pmc-id="PMC11550353" data-figure-id="fig7"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-7" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/0c5b4a17feec/gr7.gif" alt="Figure 7" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="726"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="856"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Figure 7 </p> </strong> <div class="figure-caption-contents"><p> Vector genome biodistribution and h… </p></div> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Figure 7 </p> </strong> <div class="figure-caption-contents"><p> Vector genome biodistribution and h <i> ABCD1 </i> mRNA expression Vector genome biodistribution (A, C,… </p></div> </div> <figcaption id="figure-caption-7" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Figure 7 </strong> <div class="figure-caption-contents">Vector genome biodistribution and h<i>ABCD1</i> mRNA expression Vector genome biodistribution (A, C, and E) and h<i>ABCD1</i> mRNA expression (B, D, and F) in NHP target tissues at 1 month (A and B), 3 months (C and D), and 6 months (E and F) post SBT101 administration in NHP study 4. Data represent mean ± 95% confidence interval. Vector genome copies and h<i>ABCD1</i> mRNA expression were below the limit of detection (LOD) for almost all controls (<i>n</i> = 3; data not plotted). Control NHPs received vehicle control (poloxamer [Kolliphor P188], potassium chloride, potassium dihydrogen phosphate dibasic in sterile water). <sup>a</sup>One NHP did not have a calculated measurement. <sup>b</sup>One NHP had a measurement below the limit of quantification. <sup>c</sup>One NHP had a measurement below the LOD. Vector genome biodistribution and h<i>ABCD1</i> mRNA expression in NHP study 3 and NHP study 5 are shown in Figures S9 and S10, respectively. Vector genome biodistribution and h<i>ABCD1</i> mRNA expression in other tissues are shown in Figures S11–S13. DRG, dorsal root ganglia; h<i>ABCD1</i> human adenosine triphosphate-binding cassette sub-family D member 1; mRNA, messenger RNA; vg/an, vector genomes/animal.</div> </figcaption> </figure> <figure class="figure-item tail" itemscope itemtype="http://schema.org/ImageObject" itemprop="associatedMedia" data-slide-index="8" data-label-slug="figure-8"> <a class="figure-link" href="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/aef9f6f6dfb7/gr8.jpg" itemprop="contentUrl" aria-describedby="figure-caption-8" role="button" data-image-width="726" data-image-height="300" data-image-alt="Figure 8" data-pmc-id="PMC11550353" data-figure-id="fig8"> <img class="figure-thumb" itemprop="thumbnail" id="article-image-8" src="https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3be1/11550353/c54d6c1225b2/gr8.gif" alt="Figure 8" /> </a> <meta itemprop="width" itemtype="http://schema.org/ImageObject" content="726"> <meta itemprop="height" itemtype="http://schema.org/ImageObject" content="300"> <div class="figure-caption figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="caption" aria-hidden="true"> <div class="caption-wrap"> <strong class="figure-label"> <p> Figure 8 </p> </strong> <div class="figure-caption-contents"><p> ALT and AST levels post… </p></div> </div> </div> <div class="figure-caption-medium figure-caption-text" aria-hidden="true"> <strong class="figure-label"> <p> Figure 8 </p> </strong> <div class="figure-caption-contents"><p> ALT and AST levels post SBT101 administration in NHP study 4 ALT and… </p></div> </div> <figcaption id="figure-caption-8" class="figure-caption-full figure-caption-text" itemtype="http://schema.org/ImageObject" itemprop="description"> <strong class="figure-label"> Figure 8 </strong> <div class="figure-caption-contents">ALT and AST levels post SBT101 administration in NHP study 4 ALT and AST activity were assessed in samples collected before and after a 6-h lumbar infusion of SBT101. (A and B) show samples collected before SBT101 administration on days −14 and −6, and after administration on days 7, 15, 21, 29, 56, 93, and 183. Data represent mean ± 95% confidence interval. Blue shaded areas indicate normal ranges for ALT and AST levels. Control NHPs received vehicle control (poloxamer [Kolliphor P188], potassium chloride, potassium dihydrogen phosphate dibasic in sterile water). (A and B) Data at day 56 and day 93 were based on <i>n</i> = 6 for controls and both low- and medium-dose SBT101 and <i>n</i> = 8 for high-dose SBT101; data at day 183 were based on <i>n</i> = 3 for controls and both low- and medium-dose SBT101 and <i>n</i> = 4 for high-dose SBT101. ALT and AST levels in NHP study 3 and NHP study 5 are shown in Figures S14 and S15, respectively. ALT, alanine aminotransferase; AST, aspartate aminotransferase; vg/an, vector genomes/animal.</div> </figcaption> </figure> </div> <div class="see-all actions-bar"> <button class="show-more" aria-controls="slides-container" data-ga-category="figure" data-ga-action="show_all"> All figures (9) </button> </div>  <div class="pswp figures-dialog" id="figures-dialog" tabindex="-1" role="dialog" aria-hidden="true" data-image-gallery-uid="article-figures" aria-label="Figures Dialog">  <div class="pswp__bg"></div>  <div class="pswp__scroll-wrap">  <div class="pswp__container"> <div class="pswp__item"></div> <div class="pswp__item"></div> <div class="pswp__item"></div> </div>  <div class="pswp__ui pswp__ui--hidden"> <div class="pswp__top-bar">  <div class="pswp__counter"></div> <button class="pswp__button pswp__button--close" title="Close (Esc)"></button> <button class="pswp__button pswp__button--share" title="Share"></button> <button class="pswp__button pswp__button--fs" title="Toggle fullscreen"></button> <button class="pswp__button pswp__button--zoom" title="Zoom in/out"></button>   <div class="pswp__preloader"> <div class="pswp__preloader__icn"> <div class="pswp__preloader__cut"> <div class="pswp__preloader__donut"></div> </div> </div> </div> </div> <div class="pswp__share-modal pswp__share-modal--hidden pswp__single-tap"> <div class="pswp__share-tooltip"></div> </div> <button class="pswp__button pswp__button--arrow--left" title="Previous (arrow left)"> </button> <button class="pswp__button pswp__button--arrow--right" title="Next (arrow right)"> </button> <div class="pswp__caption"> <div class="pswp__caption__center"></div> </div> </div> </div> </div> <div class="figure-dialog-caption"> <div class="figure-caption-link-container"> <a class="figure-caption-link" href="#">See this image and copyright information in PMC</a> </div> <div class="figure-caption-center" data-expand-title="click to show full caption" data-collapse-title="click to hide full caption"> </div> <button class="figure-caption-toggle-btn" aria-label="show full caption" data-expand-text="show full caption" data-collapse-text="hide full caption">  </button> </div> </div> <div class="similar-articles" id="similar"> <h2 class="title"> Similar articles </h2> <ul class="articles-list" id="similar-articles-list"> <li class="full-docsum"> <div class="docsum-content" style="display:inline;"> <a class="docsum-title" href="/30358470/" ref="article_id=30358470&linksrc=similar_articles_link&ordinalpos=1" data-ga-category="similar_article" data-ga-action="30358470" data-ga-label=""> Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy. </a> <div class="docsum-citation full-citation"> <span class="docsum-authors full-authors">Gong Y, Berenson A, Laheji F, Gao G, Wang D, Ng C, Volak A, Kok R, Kreouzis V, Dijkstra IM, Kemp S, Maguire CA, Eichler F.</span> <span class="docsum-authors short-authors">Gong Y, et al.</span> <span class="docsum-journal-citation full-journal-citation">Hum Gene Ther. 2019 May;30(5):544-555. doi: 10.1089/hum.2018.079. Epub 2018 Dec 18.</span> <span class="docsum-journal-citation short-journal-citation">Hum Gene Ther. 2019.</span> <span class="citation-part">PMID: <span class="docsum-pmid">30358470</span></span> <span class="free-resources spaced-citation-item citation-part">Free PMC article.</span> </div> </div> </li> <li class="full-docsum"> <div class="docsum-content" style="display:inline;"> <a class="docsum-title" href="/35166123/" ref="article_id=35166123&linksrc=similar_articles_link&ordinalpos=2" data-ga-category="similar_article" data-ga-action="35166123" data-ga-label=""> Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy. </a> <div class="docsum-citation full-citation"> <span class="docsum-authors full-authors">Özgür-Günes Y, Chedik M, Le Stunff C, Fovet CM, Bougnères P.</span> <span class="docsum-authors short-authors">Özgür-Günes Y, et al.</span> <span class="docsum-journal-citation full-journal-citation">Hum Gene Ther. 2022 Sep;33(17-18):936-949. doi: 10.1089/hum.2021.293. Epub 2022 Jul 18.</span> <span class="docsum-journal-citation short-journal-citation">Hum Gene Ther. 2022.</span> <span class="citation-part">PMID: <span class="docsum-pmid">35166123</span></span> </div> </div> </li> <li class="full-docsum"> <div class="docsum-content" style="display:inline;"> <a class="docsum-title" href="/25592337/" ref="article_id=25592337&linksrc=similar_articles_link&ordinalpos=3" data-ga-category="similar_article" data-ga-action="25592337" data-ga-label=""> Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy. </a> <div class="docsum-citation full-citation"> <span class="docsum-authors full-authors">Gong Y, Mu D, Prabhakar S, Moser A, Musolino P, Ren J, Breakefield XO, Maguire CA, Eichler FS.</span> <span class="docsum-authors short-authors">Gong Y, et al.</span> <span class="docsum-journal-citation full-journal-citation">Mol Ther. 2015 May;23(5):824-834. doi: 10.1038/mt.2015.6. Epub 2015 Jan 16.</span> <span class="docsum-journal-citation short-journal-citation">Mol Ther. 2015.</span> <span class="citation-part">PMID: <span class="docsum-pmid">25592337</span></span> <span class="free-resources spaced-citation-item citation-part">Free PMC article.</span> </div> </div> </li> <li class="full-docsum"> <div class="docsum-content" style="display:inline;"> <a class="docsum-title" href="/20301491/" ref="article_id=20301491&linksrc=similar_articles_link&ordinalpos=4" data-ga-category="similar_article" data-ga-action="20301491" data-ga-label=""> X-Linked Adrenoleukodystrophy. </a> <div class="docsum-citation full-citation"> <span class="docsum-authors full-authors">Raymond GV, Moser AB, Fatemi A.</span> <span class="docsum-authors short-authors">Raymond GV, et al.</span> <span class="docsum-journal-citation full-journal-citation">1999 Mar 26 [updated 2023 Apr 6]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, editors. GeneReviews<sup>®</sup> [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024.</span> <span class="docsum-journal-citation short-journal-citation">1999 Mar 26 [updated 2023 Apr 6]. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Amemiya A, editors. GeneReviews<sup>®</sup> [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2024.</span> <span class="citation-part">PMID: <span class="docsum-pmid">20301491</span></span> <span class="free-resources spaced-citation-item citation-part">Free Books & Documents.</span> <span class="publication-type spaced-citation-item citation-part">Review.</span> </div> </div> </li> <li class="full-docsum"> <div class="docsum-content" style="display:inline;"> <a class="docsum-title" href="/17532287/" ref="article_id=17532287&linksrc=similar_articles_link&ordinalpos=5" data-ga-category="similar_article" data-ga-action="17532287" data-ga-label=""> [X-linked adrenoleukodystrophy]. </a> <div class="docsum-citation full-citation"> <span class="docsum-authors full-authors">Aubourg P.</span> <span class="docsum-authors short-authors">Aubourg P.</span> <span class="docsum-journal-citation full-journal-citation">Ann Endocrinol (Paris). 2007 Dec;68(6):403-11. doi: 10.1016/j.ando.2007.04.002. Epub 2007 May 29.</span> <span class="docsum-journal-citation short-journal-citation">Ann Endocrinol (Paris). 2007.</span> <span class="citation-part">PMID: <span class="docsum-pmid">17532287</span></span> <span class="publication-type spaced-citation-item citation-part">Review.</span> <span class="language spaced-citation-item citation-part">French.</span> </div> </div> </li> </ul> <div class="actions-bar"> <a class="show-all-linked-articles" data-href="/?linkname=pubmed_pubmed&from_uid=39524975" data-ga-category="similar_article" data-ga-action="show_all" > See all similar articles </a> </div> </div> <div class="references" id="references"> <h2 class="title"> References </h2> <div id='top-references-list' class="references-lists-container"> <div class="refs-list"> <ol id="top-references-list-1" class="references-list"> <li> <ol class="references-and-notes-list"> <li class="skip-numbering" value="1"> Kemp S., Huffnagel I.C., Linthorst G.E., Wanders R.J., Engelen M. Adrenoleukodystrophy - neuroendocrine pathogenesis and redefinition of natural history. Nat. Rev. Endocrinol. 2016;12:606–615. - <a class="reference-link" href="/27312864/" ref="linksrc=references_link&ordinalpos=1" data-ga-category="reference" data-ga-action="27312864" > PubMed </a> </li> </ol> </li> <li> <ol class="references-and-notes-list"> <li class="skip-numbering" value="1"> Zhu J., Eichler F., Biffi A., Duncan C.N., Williams D.A., Majzoub J.A. The Changing Face of Adrenoleukodystrophy. Endocr. Rev. 2020;41:577–593. - <a class="reference-link" href="https://pmc.ncbi.nlm.nih.gov/articles/PMC7286618/" ref="linksrc=references_link&ordinalpos=1" data-ga-category="reference" data-ga-action="PMC7286618" > PMC </a> - <a class="reference-link" href="/32364223/" ref="linksrc=references_link&ordinalpos=2" data-ga-category="reference" data-ga-action="32364223" > PubMed </a> </li> </ol> </li> <li> <ol class="references-and-notes-list"> <li class="skip-numbering" value="1"> Berger J., Forss-Petter S., Eichler F.S. Pathophysiology of X-linked adrenoleukodystrophy. Biochimie. 2014;98:135–142. - <a class="reference-link" href="https://pmc.ncbi.nlm.nih.gov/articles/PMC3988840/" ref="linksrc=references_link&ordinalpos=1" data-ga-category="reference" data-ga-action="PMC3988840" > PMC </a> - <a class="reference-link" href="/24316281/" ref="linksrc=references_link&ordinalpos=2" data-ga-category="reference" data-ga-action="24316281" > PubMed </a> </li> </ol> </li> <li> <ol class="references-and-notes-list"> <li class="skip-numbering" value="1"> Mallack E.J., Gao K., Engelen M., Kemp S. Structure and Function of the ABCD1 Variant Database: 20 Years, 940 Pathogenic Variants, and 3400 Cases of Adrenoleukodystrophy. Cells. 2022;11 - <a class="reference-link" href="https://pmc.ncbi.nlm.nih.gov/articles/PMC8773697/" ref="linksrc=references_link&ordinalpos=1" data-ga-category="reference" data-ga-action="PMC8773697" > PMC </a> - <a class="reference-link" href="/35053399/" ref="linksrc=references_link&ordinalpos=2" data-ga-category="reference" data-ga-action="35053399" > PubMed </a> </li> </ol> </li> <li> <ol class="references-and-notes-list"> <li class="skip-numbering" value="1"> Kawaguchi K., Morita M., ABC Transporter Subfamily D. Distinct Differences in Behavior between ABCD1-3 and ABCD4 in Subcellular Localization, Function, and Human Disease. BioMed Res. Int. 2016;2016 - <a class="reference-link" href="https://pmc.ncbi.nlm.nih.gov/articles/PMC5059523/" ref="linksrc=references_link&ordinalpos=1" data-ga-category="reference" data-ga-action="PMC5059523" > PMC </a> - <a class="reference-link" href="/27766264/" ref="linksrc=references_link&ordinalpos=2" data-ga-category="reference" data-ga-action="27766264" > PubMed </a> </li> </ol> </li> </ol> <div class="actions-bar"> <button class="show-all" ref="linksrc=show_all_references_link" aria-controls="top-references-list-1" data-next-page-url="/39524975/references/" data-ga-category="reference" data-ga-action="show_more" > Show all 43 references </button> </div> </div> </div> </div> <div id="related-links" class="related-links"> <h2 class="title"> Related information </h2> <ul class="related-links-list"><li><a title="Related information in MedGen" href="https://www.ncbi.nlm.nih.gov/medgen?linkname=pubmed_medgen&from_uid=39524975" ref="log$=recordlinks&logdbfrom=pubmed&ncbi_uid=39524975&ordinalpos=1&linkpos=1"> MedGen </a></li></ul> </div> <div id="linkout" class="linkout"> <h2 class="title"> LinkOut - more resources </h2> <ul class="linkout-list"><li><h3 class="linkout-category">Full Text Sources</h3><ul class="linkout-category-links"><li><a href="https://linkinghub.elsevier.com/retrieve/pii/S2329-0501(24)00170-0" target="_blank" rel="noopener" ref="linksrc=linkout_link&PrId=3048&uid=39524975&db=pubmed&itool=Abstract&log$=linkoutlink&nlmid=101624857" data-ga-category="link_out" data-ga-action="Full Text Sources" data-ga-label="Elsevier Science" > Elsevier Science </a></li><li><a href="https://pmc.ncbi.nlm.nih.gov/articles/pmid/39524975/" target="_blank" rel="noopener" ref="linksrc=linkout_link&PrId=3494&uid=39524975&db=pubmed&itool=Abstract&log$=linkoutlink&nlmid=101624857" data-ga-category="link_out" data-ga-action="Full Text Sources" data-ga-label="PubMed Central" > PubMed Central </a></li></ul></li></ul> </div> </main> <div class="overlay" role="dialog" aria-label="Full Text Links Dialog"> <div id="full-text-links-dialog" class="dialog full-text-links-dialog"> <strong class="title">Full text links</strong> <button class="close-overlay" tabindex="3">[x]</button> <div class="full-text-links-list"> <a class="link-item dialog-focus" href="https://linkinghub.elsevier.com/retrieve/pii/S2329-0501(24)00170-0" target="_blank" rel="noopener" ref="linksrc=fulltextorjournal_fulltext&is_pmc=False&PrId=3048&itool=Abstract-def&log$=linkouticon&uid=39524975&db=pubmed&nlmid=101624857" title="See full text options at Elsevier Science" tabindex="1" data-ga-category="full_text" data-ga-action="Elsevier Science" data-ga-label="39524975" ><img src="https://cdn.ncbi.nlm.nih.gov/corehtml/query/egifs/https:--linkinghub.elsevier.com-ihub-images-celloa.png" alt="full text provider logo"><span class="text"> Elsevier Science </span></a><a class="link-item pmc " href="https://pmc.ncbi.nlm.nih.gov/articles/pmid/39524975/" target="_blank" rel="noopener" ref="linksrc=fulltextorjournal_fulltext&is_pmc=True&PrId=3494&itool=Abstract-def&log$=linkouticon&uid=39524975&db=pubmed&nlmid=101624857" title="Free full text at PubMed Central" tabindex="2" data-ga-category="full_text" data-ga-action="PMC" data-ga-label="39524975" ><span class="text"> Free PMC article </span></a> </div> </div> </div> <div class="overlay" role="dialog" aria-label="Citation Dialog"> <div class="dialog citation-dialog"> <button class="close-overlay" tabindex="1">[x]</button> <div class="title">Cite</div> <div class="citation-text-block"> <div class="citation-text"></div> <div class="citation-actions"> <button class="copy-button dialog-focus" data-ga-category="save_share" data-ga-action="cite" data-ga-label="copy" tabindex="2"> Copy </button> <form method="post"> <input type="hidden" name="csrfmiddlewaretoken" value="Sa7OjD62rxvKy8LSscRNpZonEztXlgowcdkleIzQGUPpRYTAEipVjab3EFQFrXVK"> <button type="submit" class="export-button" data-ga-category="save_share" data-ga-action="cite" data-ga-label="download" title="Download a file for external citation management software" tabindex="3"> <span class="download-title">Download .nbib</span> <span class="download-title-mobile">.nbib</span> </button> </form> <div class="citation-style-selector-wrapper"> <label class="selector-label">Format:</label> <select aria-label="Format" class="citation-style-selector" tabindex="4"> <option data-style-url-name="ama" value="AMA" > AMA </option> <option data-style-url-name="apa" value="APA" > APA </option> <option data-style-url-name="mla" value="MLA" > MLA </option> <option data-style-url-name="nlm" value="NLM" selected="selected"> NLM </option> </select> </div> </div> </div> </div> </div> <div class="overlay" role="dialog" aria-label="More Actions Dialog"> <div id="more-actions-dialog" class="dialog more-actions-dialog"> <strong class="title">Send To</strong> <ul class="more-actions-links"> <li><a id="clipboard-trigger" role="button" class="submit-button clipboard-trigger clipboard-trigger-target dialog-focus link-item " href="#">Clipboard</a></li> <li><a class="email-trigger link-item" role="button" href="#">Email</a></li> <li><a class="save-trigger link-item" role="button" href="#">Save</a></li> <li><a class="link-item" role="button" href="https://account.ncbi.nlm.nih.gov/?back_url=https%3A%2F%2Fpubmed.ncbi.nlm.nih.gov%2F39524975%2F%23open-bibliography-panel">My Bibliography</a></li> <li><a class="link-item" role="button" href="https://account.ncbi.nlm.nih.gov/?back_url=https%3A%2F%2Fpubmed.ncbi.nlm.nih.gov%2F39524975%2F%23open-collections-panel">Collections</a></li> <li><a class="citation-manager-trigger link-item" role="button" href="#">Citation Manager</a></li> </ul> <button class="close-overlay">[x]</button> </div> </div> </div> <div id="ncbi-footer"> <div class="literature-footer" role="complementary" title="Links to NCBI Literature Resources"> <div class="usa-grid"> <p class="literature-footer-text">NCBI Literature Resources</p> <p class="literature-footer-text"> <a class="literature-footer-link" data-ga-category="literature_resources" data-ga-action="mesh_link" href="https://www.ncbi.nlm.nih.gov/mesh/">MeSH</a> <a class="literature-footer-link" data-ga-category="literature_resources" data-ga-action="pmc_link" href="https://www.ncbi.nlm.nih.gov/pmc/">PMC</a> <a class="literature-footer-link" data-ga-category="literature_resources" data-ga-action="bookshelf_link" href="https://www.ncbi.nlm.nih.gov/books">Bookshelf</a> <a class="literature-footer-link" data-ga-category="literature_resources" data-ga-action="disclaimer_link" href="/disclaimer/">Disclaimer</a> </p> <p class="literature-footer-text attribution-statement">The PubMed wordmark and PubMed logo are registered trademarks of the U.S. Department of Health and Human Services (HHS). Unauthorized use of these marks is strictly prohibited.</p> </div> </div>  <footer> <section class="icon-section"> <div id="icon-section-header" class="icon-section_header">Follow NCBI</div> <div class="grid-container container"> <div class="icon-section_container"> <a class="footer-icon" id="footer_twitter" href="https://twitter.com/ncbi" aria-label="Twitter"> <svg width="40" height="40" viewBox="0 0 40 37" fill="none" xmlns="http://www.w3.org/2000/svg"> <title>Twitter</title> <g id="twitterx1008"> <path id="path1008" d="M6.06736 7L16.8778 20.8991L6.00001 32.2H10.2L18.6 23.1L25.668 32.2H34L22.8 17.5L31.9 7H28.4L20.7 15.4L14.401 7H6.06898H6.06736ZM9.66753 8.73423H12.9327L29.7327 30.4658H26.5697L9.66753 8.73423Z" fill="#5B616B"/> </g> </svg> </a> <a class="footer-icon" id="footer_facebook" href="https://www.facebook.com/ncbi.nlm" aria-label="Facebook"><svg data-name="Layer 1" xmlns="http://www.w3.org/2000/svg" viewBox="0 0 300 300"> <title>Facebook</title> <path class="cls-11" d="M210.5,115.12H171.74V97.82c0-8.14,5.39-10,9.19-10h27.14V52l-39.32-.12c-35.66,0-42.42,26.68-42.42,43.77v19.48H99.09v36.32h27.24v109h45.41v-109h35Z"> </path> </svg></a> <a class="footer-icon" id="footer_linkedin" href="https://www.linkedin.com/company/ncbinlm" aria-label="LinkedIn"><svg data-name="Layer 1" xmlns="http://www.w3.org/2000/svg" viewBox="0 0 300 300"> <title>LinkedIn</title> <path class="cls-11" d="M101.64,243.37H57.79v-114h43.85Zm-22-131.54h-.26c-13.25,0-21.82-10.36-21.82-21.76,0-11.65,8.84-21.15,22.33-21.15S101.7,78.72,102,90.38C102,101.77,93.4,111.83,79.63,111.83Zm100.93,52.61A17.54,17.54,0,0,0,163,182v61.39H119.18s.51-105.23,0-114H163v13a54.33,54.33,0,0,1,34.54-12.66c26,0,44.39,18.8,44.39,55.29v58.35H198.1V182A17.54,17.54,0,0,0,180.56,164.44Z"> </path> </svg></a> <a class="footer-icon" id="footer_github" href="https://github.com/ncbi" aria-label="GitHub"><svg data-name="Layer 1" xmlns="http://www.w3.org/2000/svg" viewBox="0 0 300 300"> <defs> <style> .cls-11, .cls-12 { fill: #737373; } .cls-11 { fill-rule: evenodd; } </style> </defs> <title>GitHub</title> <path class="cls-11" d="M151.36,47.28a105.76,105.76,0,0,0-33.43,206.1c5.28,1,7.22-2.3,7.22-5.09,0-2.52-.09-10.85-.14-19.69-29.42,6.4-35.63-12.48-35.63-12.48-4.81-12.22-11.74-15.47-11.74-15.47-9.59-6.56.73-6.43.73-6.43,10.61.75,16.21,10.9,16.21,10.9,9.43,16.17,24.73,11.49,30.77,8.79,1-6.83,3.69-11.5,6.71-14.14C108.57,197.1,83.88,188,83.88,147.51a40.92,40.92,0,0,1,10.9-28.39c-1.1-2.66-4.72-13.42,1-28,0,0,8.88-2.84,29.09,10.84a100.26,100.26,0,0,1,53,0C198,88.3,206.9,91.14,206.9,91.14c5.76,14.56,2.14,25.32,1,28a40.87,40.87,0,0,1,10.89,28.39c0,40.62-24.74,49.56-48.29,52.18,3.79,3.28,7.17,9.71,7.17,19.58,0,14.15-.12,25.54-.12,29,0,2.82,1.9,6.11,7.26,5.07A105.76,105.76,0,0,0,151.36,47.28Z"> </path> <path class="cls-12" d="M85.66,199.12c-.23.52-1.06.68-1.81.32s-1.2-1.06-.95-1.59,1.06-.69,1.82-.33,1.21,1.07.94,1.6Zm-1.3-1"> </path> <path class="cls-12" d="M90,203.89c-.51.47-1.49.25-2.16-.49a1.61,1.61,0,0,1-.31-2.19c.52-.47,1.47-.25,2.17.49s.82,1.72.3,2.19Zm-1-1.08"> </path> <path class="cls-12" d="M94.12,210c-.65.46-1.71,0-2.37-.91s-.64-2.07,0-2.52,1.7,0,2.36.89.65,2.08,0,2.54Zm0,0"></path> <path class="cls-12" d="M99.83,215.87c-.58.64-1.82.47-2.72-.41s-1.18-2.06-.6-2.7,1.83-.46,2.74.41,1.2,2.07.58,2.7Zm0,0"> </path> <path class="cls-12" d="M107.71,219.29c-.26.82-1.45,1.2-2.64.85s-2-1.34-1.74-2.17,1.44-1.23,2.65-.85,2,1.32,1.73,2.17Zm0,0"> </path> <path class="cls-12" d="M116.36,219.92c0,.87-1,1.59-2.24,1.61s-2.29-.68-2.3-1.54,1-1.59,2.26-1.61,2.28.67,2.28,1.54Zm0,0"> </path> <path class="cls-12" d="M124.42,218.55c.15.85-.73,1.72-2,1.95s-2.37-.3-2.52-1.14.73-1.75,2-2,2.37.29,2.53,1.16Zm0,0"></path> </svg></a> <a class="footer-icon" id="footer_blog" href="https://ncbiinsights.ncbi.nlm.nih.gov/" aria-label="Blog"> <svg id="Layer_1" data-name="Layer 1" xmlns="http://www.w3.org/2000/svg" viewBox="0 0 40 40"><defs><style>.cls-1{fill:#737373;}</style></defs><path class="cls-1" d="M14,30a4,4,0,1,1-4-4,4,4,0,0,1,4,4Zm11,3A19,19,0,0,0,7.05,15a1,1,0,0,0-1,1v3a1,1,0,0,0,.93,1A14,14,0,0,1,20,33.07,1,1,0,0,0,21,34h3a1,1,0,0,0,1-1Zm9,0A28,28,0,0,0,7,6,1,1,0,0,0,6,7v3a1,1,0,0,0,1,1A23,23,0,0,1,29,33a1,1,0,0,0,1,1h3A1,1,0,0,0,34,33Z"/></svg> </a> </div> </div> </section> <section class="container-fluid bg-primary"> <div class="container pt-5"> <div class="row mt-3"> <div class="col-lg-3 col-12"> <p><a class="text-white" href="https://www.nlm.nih.gov/socialmedia/index.html">Connect with NLM</a></p> <ul class="list-inline social_media"> <li class="list-inline-item"><a href="https://twitter.com/NLM_NIH" aria-label="Twitter" target="_blank" rel="noopener noreferrer"> <svg width="35" height="35" viewBox="0 0 38 35" fill="none" xmlns="http://www.w3.org/2000/svg"> <title>Twitter</title> <g id="twitterx1009" clip-path="url(#clip0_65276_3946)"> <path id="Vector" d="M17.5006 34.6565C26.9761 34.6565 34.6575 26.9751 34.6575 17.4996C34.6575 8.02416 26.9761 0.342773 17.5006 0.342773C8.02514 0.342773 0.34375 8.02416 0.34375 17.4996C0.34375 26.9751 8.02514 34.6565 17.5006 34.6565Z" fill="#205493" stroke="white" stroke-width="1.2" stroke-miterlimit="10"></path> <path id="path1009" d="M8.54811 8.5L16.2698 18.4279L8.50001 26.5H11.5L17.5 20L22.5486 26.5H28.5L20.5 16L27 8.5H24.5L19 14.5L14.5007 8.5H8.54927H8.54811ZM11.1197 9.73873H13.4519L25.4519 25.2613H23.1926L11.1197 9.73873Z" fill="white"></path> </g> <defs> <clipPath id="clip0_65276_3946"> <rect width="38" height="38" fill="white"></rect> </clipPath> </defs> </svg></a> </li> <li class="list-inline-item"><a href="https://www.facebook.com/nationallibraryofmedicine" aria-label="Facebook" rel="noopener noreferrer" target="_blank"> <svg version="1.1" xmlns="http://www.w3.org/2000/svg" xmlns:xlink="http://www.w3.org/1999/xlink" x="0px" y="0px" viewBox="0 0 249 249" style="enable-background:new 0 0 249 249;" xml:space="preserve"> <style type="text/css"> .st10 { fill: #FFFFFF; } .st110 { fill: none; stroke: #FFFFFF; stroke-width: 8; stroke-miterlimit: 10; } </style> <title>SM-Facebook</title> <g> <g> <path class="st10" d="M159,99.1h-24V88.4c0-5,3.3-6.2,5.7-6.2h16.8V60l-24.4-0.1c-22.1,0-26.2,16.5-26.2,27.1v12.1H90v22.5h16.9 v67.5H135v-67.5h21.7L159,99.1z"></path> </g> </g> <circle class="st110" cx="123.6" cy="123.2" r="108.2"></circle> </svg> </a></li> <li class="list-inline-item"><a href="https://www.youtube.com/user/NLMNIH" aria-label="Youtube" target="_blank" rel="noopener noreferrer"><svg version="1.1" xmlns="http://www.w3.org/2000/svg" xmlns:xlink="http://www.w3.org/1999/xlink" x="0px" y="0px" viewBox="0 0 249 249" style="enable-background:new 0 0 249 249;" xml:space="preserve"> <title>SM-Youtube</title> <style type="text/css"> .st4 { fill: none; stroke: #FFFFFF; stroke-width: 8; stroke-miterlimit: 10; } .st5 { fill: #FFFFFF; } </style> <circle class="st4" cx="124.2" cy="123.4" r="108.2"></circle> <g transform="translate(0,-952.36218)"> <path class="st5" d="M88.4,1037.4c-10.4,0-18.7,8.3-18.7,18.7v40.1c0,10.4,8.3,18.7,18.7,18.7h72.1c10.4,0,18.7-8.3,18.7-18.7 v-40.1c0-10.4-8.3-18.7-18.7-18.7H88.4z M115.2,1058.8l29.4,17.4l-29.4,17.4V1058.8z"></path> </g> </svg></a></li> </ul> </div> <div class="col-lg-3 col-12"> <p class="address_footer text-white">National Library of Medicine<br> <a href="https://www.google.com/maps/place/8600+Rockville+Pike,+Bethesda,+MD+20894/@38.9959508,-77.101021,17z/data=!3m1!4b1!4m5!3m4!1s0x89b7c95e25765ddb:0x19156f88b27635b8!8m2!3d38.9959508!4d-77.0988323" class="text-white" target="_blank" rel="noopener noreferrer">8600 Rockville Pike<br> Bethesda, MD 20894</a></p> </div> <div class="col-lg-3 col-12 centered-lg"> <p><a href="https://www.nlm.nih.gov/web_policies.html" class="text-white">Web Policies</a><br> <a href="https://www.nih.gov/institutes-nih/nih-office-director/office-communications-public-liaison/freedom-information-act-office" class="text-white">FOIA</a><br> <a href="https://www.hhs.gov/vulnerability-disclosure-policy/index.html" class="text-white" id="vdp">HHS Vulnerability Disclosure</a></p> </div> <div class="col-lg-3 col-12 centered-lg"> <p><a class="supportLink text-white" href="https://support.nlm.nih.gov/">Help</a><br> <a href="https://www.nlm.nih.gov/accessibility.html" class="text-white">Accessibility</a><br> <a href="https://www.nlm.nih.gov/careers/careers.html" class="text-white">Careers</a></p> </div> </div> <div class="row"> <div class="col-lg-12 centered-lg"> <nav class="bottom-links"> <ul class="mt-3"> <li> <a class="text-white" href="//www.nlm.nih.gov/">NLM</a> </li> <li> <a class="text-white" href="https://www.nih.gov/">NIH</a> </li> <li> <a class="text-white" href="https://www.hhs.gov/">HHS</a> </li> <li> <a class="text-white" href="https://www.usa.gov/">USA.gov</a> </li> </ul> </nav> </div> </div> </div> </section> </footer>   <script type="text/javascript"> var nwds_version = "1.2.3"; var meta_nwds_ver = document.createElement('meta'); meta_nwds_ver.name = 'ncbi_nwds_ver'; meta_nwds_ver.content = nwds_version; document.getElementsByTagName('head')[0].appendChild(meta_nwds_ver); var meta_nwds = document.createElement('meta'); meta_nwds.name = 'ncbi_nwds'; meta_nwds.content = 'yes'; document.getElementsByTagName('head')[0].appendChild(meta_nwds); var alertsUrl = "/core/alerts/alerts.js"; if (typeof ncbiBaseUrl !== 'undefined') { alertsUrl = ncbiBaseUrl + alertsUrl; } </script> </div>  <script src="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/CACHE/js/output.293fbf76aa18.js"></script>  <script src="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/CACHE/js/output.550e9c8906a2.js"></script> <script> ncbi.awesome.basePage.init({ userInfo: { isLoggedIn: false, username: "", loginUrl: "https://account.ncbi.nlm.nih.gov/?back_url=https%3A%2F%2Fpubmed.ncbi.nlm.nih.gov%2F39524975%2F", logoutUrl: "https://www.ncbi.nlm.nih.gov/account/signout/?back_url=https%3A//pubmed.ncbi.nlm.nih.gov/39524975/" }, baseUrl: 'https://www.ncbi.nlm.nih.gov', noSessionCookieName: "pm-ns", searchIdCookieName: "pm-sid", adjNavSearchIdCookieName: "pm-adjnav-sid", searchFormSubmittedCookieName: "pm-sfs", isOnSearchPageCookieName: "pm-iosp", backToSearchCookieName: "pm-btsl", clinicalSeeAllCookieName: "pm-csa", jsCookieDomain: "pubmed.ncbi.nlm.nih.gov", userShareRequestFailed: "", noSharedSettings: "myncbi_no_shared_settings", noSharedAccount: "myncbi_no_shared_account" }); </script> <script type="module"> import dismissableNCBIAlert from "https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/core/dismissable-ncbi-alert.js"; jQuery.getScript("https://www.ncbi.nlm.nih.gov/core/alerts/alerts.js", function () { galert(['body > *:last-child']); dismissableNCBIAlert(); }).fail(function() { }); </script> <script defer type="text/javascript" src="https://cdn.ncbi.nlm.nih.gov/core/pinger/pinger.js"> </script> <script src="https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/CACHE/js/output.04d45d85d031.js"></script> <script> ncbi.awesome.articlePage.init({ baseSuggestionsUrl: "/suggestions/", mathJaxUrl: "https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/mathjax/unpacked/MathJax.js?config=MML_CHTML", clipboardAPIRoot: "/ajax/clipboard/", clipboardNextPageUrl: "/clipboard-next-page/", searchIdCookieName: "pm-sid", searchFormSubmittedCookieName: "pm-sfs", backToSearchCookieName: "pm-btsl", jsCookieDomain: "pubmed.ncbi.nlm.nih.gov", longTermPrefix: "longquery", adjNavURLPath: "/39524975/adj-nav/", adjNavSearchIdCookieName: "pm-adjnav-sid", articlePageUrl: "/39524975/", markJSUrl: "https://cdn.ncbi.nlm.nih.gov/pubmed/0d576970-ea4f-4f8d-9bab-bf51569c42d2/mark.js/dist/jquery.mark.js", termHighlightColor: "", highlightedTokens: "", citeFormatPreferenceCookieName: "pm-cf" }); </script> </body> </html>

CINXE.COM

An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs - PubMed