CINXE.COM
Enhancing Gene Editing Efficiency: Multi-Tailed mRNA Integration in CRISPR-Cas9 Systems
<!doctype html><html lang="en"><head> <meta charset="utf-8"> <title>Enhancing Gene Editing Efficiency: Multi-Tailed mRNA Integration in CRISPR-Cas9 Systems</title> <link rel="shortcut icon" href="https://oxfordglobal.com/hubfs/OXFORD_GLOBAL-LOGO_SYMBOL.jpg"> <meta name="description" content="A study conducted by the Broad Institute and the Massachusetts Institute of Technology (MIT) demonstrated that when multi-tailed mRNAs were integrated into a CRISPR-Cas9 system they proved to be more efficient gene editor than unmodified mRNA."> <meta name="viewport" content="width=device-width, initial-scale=1"> <meta property="og:description" content="A study conducted by the Broad Institute and the Massachusetts Institute of Technology (MIT) demonstrated that when multi-tailed mRNAs were integrated into a CRISPR-Cas9 system they proved to be more efficient gene editor than unmodified mRNA."> <meta property="og:title" content="Enhancing Gene Editing Efficiency: Multi-Tailed mRNA Integration in CRISPR-Cas9 Systems"> <meta name="twitter:description" content="A study conducted by the Broad Institute and the Massachusetts Institute of Technology (MIT) demonstrated that when multi-tailed mRNAs were integrated into a CRISPR-Cas9 system they proved to be more efficient gene editor than unmodified mRNA."> <meta name="twitter:title" content="Enhancing Gene Editing Efficiency: Multi-Tailed mRNA Integration in CRISPR-Cas9 Systems"> <style> a.cta_button{-moz-box-sizing:content-box !important;-webkit-box-sizing:content-box !important;box-sizing:content-box !important;vertical-align:middle}.hs-breadcrumb-menu{list-style-type:none;margin:0px 0px 0px 0px;padding:0px 0px 0px 0px}.hs-breadcrumb-menu-item{float:left;padding:10px 0px 10px 10px}.hs-breadcrumb-menu-divider:before{content:'›';padding-left:10px}.hs-featured-image-link{border:0}.hs-featured-image{float:right;margin:0 0 20px 20px;max-width:50%}@media (max-width: 568px){.hs-featured-image{float:none;margin:0;width:100%;max-width:100%}}.hs-screen-reader-text{clip:rect(1px, 1px, 1px, 1px);height:1px;overflow:hidden;position:absolute !important;width:1px} </style> <link rel="stylesheet" href="https://oxfordglobal.com/hs-fs/hub/8696823/hub_generated/template_assets/135726131582/1732113079147/git/og-stage/css/main.min.css"> <link rel="stylesheet" href="https://cdn.oxfordglobal.com/fontawesome/6.5.1/css/fontawesome.min.css"> <link rel="stylesheet" href="https://cdn.oxfordglobal.com/fontawesome/6.5.1/css/brands.min.css"> <link rel="stylesheet" href="https://cdn.oxfordglobal.com/fontawesome/6.5.1/css/solid.min.css"> <!-- Editor Styles --> <style id="hs_editor_style" type="text/css"> #hs_cos_wrapper_module_17019620504109 { display: block !important; margin-bottom: 0px !important } </style> <!-- Added by GoogleAnalytics4 integration --> <script> var _hsp = window._hsp = window._hsp || []; window.dataLayer = window.dataLayer || []; function gtag(){dataLayer.push(arguments);} var useGoogleConsentModeV2 = true; var waitForUpdateMillis = 1000; if (!window._hsGoogleConsentRunOnce) { window._hsGoogleConsentRunOnce = true; gtag('consent', 'default', { 'ad_storage': 'denied', 'analytics_storage': 'denied', 'ad_user_data': 'denied', 'ad_personalization': 'denied', 'wait_for_update': waitForUpdateMillis }); if (useGoogleConsentModeV2) { _hsp.push(['useGoogleConsentModeV2']) } else { _hsp.push(['addPrivacyConsentListener', function(consent){ var hasAnalyticsConsent = consent && (consent.allowed || (consent.categories && consent.categories.analytics)); var hasAdsConsent = consent && (consent.allowed || (consent.categories && consent.categories.advertisement)); gtag('consent', 'update', { 'ad_storage': hasAdsConsent ? 'granted' : 'denied', 'analytics_storage': hasAnalyticsConsent ? 'granted' : 'denied', 'ad_user_data': hasAdsConsent ? 'granted' : 'denied', 'ad_personalization': hasAdsConsent ? 'granted' : 'denied' }); }]); } } gtag('js', new Date()); gtag('set', 'developer_id.dZTQ1Zm', true); gtag('config', 'G-43QK8SYTST'); </script> <script async src="https://www.googletagmanager.com/gtag/js?id=G-43QK8SYTST"></script> <!-- /Added by GoogleAnalytics4 integration --> <link rel="canonical" href="https://oxfordglobal.com/nextgen-biomed/resources/enhancing-gene-editing-efficiency-multi-tailed-mrna-integration-in-crispr-cas9-systems"> <meta property="og:image" content="https://oxfordglobal.com/hubfs/Enhancing%20Gene%20Editing%20Efficiency%20Multi%20Tailed%20mRNA%20Integration%20in%20CRISPR%20Cas9%20Systems.png"> <meta property="og:image:width" content="1200"> <meta property="og:image:height" content="800"> <meta name="twitter:image" content="https://oxfordglobal.com/hubfs/Enhancing%20Gene%20Editing%20Efficiency%20Multi%20Tailed%20mRNA%20Integration%20in%20CRISPR%20Cas9%20Systems.png"> <meta property="og:url" content="https://oxfordglobal.com/nextgen-biomed/resources/enhancing-gene-editing-efficiency-multi-tailed-mrna-integration-in-crispr-cas9-systems"> <meta name="twitter:card" content="summary"> <meta http-equiv="content-language" content="en"> <meta name="generator" content="HubSpot"></head> <body class="nextgen-biomed"> <div class="body-wrapper hs-content-id-177607186815 hs-site-page page "> <div data-global-resource-path="git/og-stage/templates/partials/header.html"> <header class="website-header "> <div class="navbar"> <div class="navbar-brands"> <a href="/nextgen-biomed" class="logo-link current-brand"> <img src="https://oxfordglobal.com/hubfs/Website/Logos/Brand%20Logos/NextGen%20Biomed.svg" class="logo-image"> <i class="dropdown fa-solid fa-chevron-down"></i> </a> <div class="brands"> <div class="intro"> <h2> Explore our brands </h2> <div class="desc"> Our brands bring together the industry's most influential leaders and scientific experts, inspiring thought-provoking and insightful content that offers a unique perspective on R&D trends and challenges. </div> </div> <div class="items"> <div class="item primary-brand"> <a href="/" class="logo-link"><img src="https://oxfordglobal.com/hubfs/logo.jpg" class="logo-image"></a> </div> <div class="item"> <a href="/precision-medicine" class="logo-link"><img src="https://oxfordglobal.com/hubfs/Website/Logos/Brand%20Logos/Precision%20Medicine.svg" class="logo-image"></a> </div> <div class="item"> <a href="/discovery-development" class="logo-link"><img src="https://oxfordglobal.com/hubfs/Website/Logos/Brand%20Logos/Discovery%20%26%20Development.svg" class="logo-image"></a> </div> <div class="item active-item"> <a href="/nextgen-biomed" class="logo-link"><img src="https://oxfordglobal.com/hubfs/Website/Logos/Brand%20Logos/NextGen%20Biomed.svg" class="logo-image"></a> </div> </div> </div> </div> <div class="flex flex-align-center navbar-nav"> <a href="#" class="navbar-nav-toggle"> <div class="toggle-icons"> <i class="fa-solid fa-bars toggle-open"></i> <i class="fa-solid fa-xmark toggle-close"></i> </div> </a> <ul class="navbar-nav-items"> <li class="navbar-nav-item"> <a href="#" class="navbar-nav-item-link">Events <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-graphics"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Header%20Events.jpg" class="subnav-image"> </div> <div class="subnav-items"> <div class="subnav-item flex-3 divider g-20"> <div class="subnav-item-content bottom-mp-fix"> <h3> In-Person & Online Events </h3> <p>Pivotal moments where high-level industry experts and thought leaders come together for focused knowledge sharing and unparalleled networking opportunities.</p> </div> <div class="subnav-item"> <div class="event-pills"> <a href="/nextgen-biomed/events/nextgen-biomed-2025" class="pill center"> <div class="b">NextGen Biomed 2025</div> <div class="heading-smallcaps"> 12 - 14 Mar 2025 | London, UK </div> </a> <a href="/nextgen-biomed/events/cell-2025" class="pill center"> <div class="b">Cell 2025</div> <div class="heading-smallcaps"> 11-12 Nov 2025 | London, UK </div> </a> </div> </div> </div> <div class="subnav-item flex-2"> <div class="subnav-item-content bottom-mp-fix"> <h3> Online Activities </h3> <p>Our Monthly Science Exchanges and Webinars keep you connected to the conversation.</p> </div> <div class="online-activities-list flex flex-column g-10"> <!-- Individual Online Activity --> <div class="online-activity"> <a href="/nextgen-biomed/online#Process%20Challenges%20in%20ATMP%20Development:%20Overcoming%20Hurdles%20for%20Successful%20Manufacturing" class="cta" target="_blank"> <h3 class="other-resources">01/11/24 - Process Challenges in ATMP Development: Overcoming Hurdles for Successful Manufacturing</h3> </a> </div> </div> <div class="subnav-item-content-more"> <a href="/nextgen-biomed/online" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> All Online Activities</a> </div> </div> </div> </div> </li> <li class="navbar-nav-item"> <a href="#" class="navbar-nav-item-link">Community <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-graphics"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Header%20Community.jpg" class="subnav-image"> </div> <div class="subnav-items"> <div class="subnav-item flex flex-1 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> About </h3> <p>NextGen Biomed is the platform to advance the fields of biologics, vaccines & novel therapeutics approaches, helping to deliver life-saving medicines to patients faster.</p> </div> <div class="subnav-item-content-more"> <a href="/nextgen-biomed/about" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> <div class="subnav-item flex flex-1 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> Community </h3> <p>NextGen Biomed is committed to supporting R&D scientists, as well as drug development and manufacturing experts, working with biologics, peptides, and oligonucleotides to overcome complexities at every stage of the drug product lifecycle.</p> </div> <div class="subnav-item-content-more"> <a href="/nextgen-biomed/community" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> <div class="subnav-item flex flex-1 "> <div class="subnav-item-content bottom-mp-fix"> <h3> Media Partners </h3> <p>We work with leading journal publications on reciprocal marketing arrangements.</p> </div> <div class="subnav-item-content-more"> <a href="/nextgen-biomed/media-partners" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> </div> </div> </li> <li class="navbar-nav-item"> <a href="#" class="navbar-nav-item-link">Services <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-graphics"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Header%20Services.jpg" class="subnav-image"> </div> <div class="subnav-items"> <div class="subnav-item flex flex-1 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> In-Person Services </h3> <p>At Oxford Global, our events are designed to connect key life science figures and innovative solution providers, accelerating scientific advancement. Our goal is to create valuable connections and an impactful experience through innovative and collaborative formats.</p> </div> <div class="subnav-item-content-more"> <a href="/nextgen-biomed/in-person-services" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> <div class="subnav-item flex flex-1"> <div class="subnav-item-content bottom-mp-fix"> <h3> Digital Marketing Services </h3> <p>Oxford Global’s products offer a powerful channel to associate your brand with the latest advancements within the industry whilst educating and influencing the industry throughout their buying process.</p> </div> <div class="subnav-item-content-more"> <a href="/nextgen-biomed/digital-marketing-services" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> More Info</a> </div> </div> </div> </div> </li> <li class="navbar-nav-item"> <a href="#" class="navbar-nav-item-link">Resources <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-graphics"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Header%20Resources.jpg" class="subnav-image"> </div> <div class="subnav-items"> <div class="subnav-item flex-2 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> Browse All Resources </h3> <p>The latest in from nextgen biomed research & development</p> </div> <div class="subnav-item-content-more"> <a href="/nextgen-biomed/resources" class="subnav-item-content-more-link"><i class="fa-solid fa-circle-chevron-right"></i> All NextGen Biomed Resources</a> </div> </div> <div class="subnav-item flex-4 divider"> <div class="subnav-item-content bottom-mp-fix"> <h3> Latest Resources </h3> <div class="resources"> <div class="resource"> <div class="resource-cover"> <a href="/nextgen-biomed/resources/university-college-london-hospitals-begins-clinical-trial-for-one-off-car-t-treatment-for-lupus" class="resource-cover-link"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Resources/NextGen%20Biomed/University%20College%20London%20Hospitals%20Begins%20Clinical%20Trial%20for%20One-Off%20CAR%20T%20Treatment%20for%20Lupus%201.jpg" class="resource-cover-image"> </a> </div> <div class="resource-content"> <h3 class="resource-heading"> <a href="/nextgen-biomed/resources/university-college-london-hospitals-begins-clinical-trial-for-one-off-car-t-treatment-for-lupus">University College London Hospitals Begins Clinical Trial for One-Off CAR T Treatment for Lupus</a> </h3> <div class="resource-abstract"> <span class="resource-date">08/11/24 - </span> The possibility of a one-off treatment for lupus could offer hope to 69,000 patients in the UK. </div> </div> </div> <div class="top-divider"></div> <div class="resource"> <div class="other-resources"><a href="/nextgen-biomed/resources/clesrovimab-shows-positive-results-as-preventative-treatment-for-rsv-in-pre-term-and-full-term-infants">07/11/24 - Clesrovimab Shows Positive Results as Preventative Treatment for RSV in Pre-Term and Full-Term Infants</a></div> </div> <div class="top-divider"></div> <div class="resource"> <div class="other-resources"><a href="/nextgen-biomed/resources/the-nist-awards-1-5-million-to-improve-standardisation-in-regenerative-medicine">31/10/24 - The NIST Awards $1.5 Million to Improve Standardisation in Regenerative Medicine</a></div> </div> </div> </div> </div> <div class="subnav-item flex-3"> <div class="subnav-item-content bottom-mp-fix"> <h3> Featured Resource </h3> <div class="resources flex flex-column g-5"> <a href="https://oxfordglobal.com/nextgen-biomed/resources/sponsored/webinar/wuxibiologics/sp-webinar-optimizing-bispecific-antibody-production-from-identifying-optimal-pairings-to-scale-up-processes"> <div class="sponsor-resource flex-column"> <div class="resource-cover img-fluid"> <div class="sponsor-resource-cover-link"> <img src="https://oxfordglobal.com/hubfs/Digital%20Marketing/Webinars/Email%20and%20Social%20Collateral/NGB%20-%20Wuxi%20Biologics/WuXi%20Biologics.png" class="sponsor-resource-cover-image"> </div> </div> <div class="resource-content p-15"> <h3 class="resource-heading"> Optimizing Bispecific Antibody Production: From Identifying Optimal Pairings to Scale-up Processes </h3> <div class="resource-abstract"> On-demand webinar presented by Dr Jiansheng Wu, Head of Protein Sciences and Vice President at WuXi Biologics </div> </div> </div> </a> </div> </div> </div> </div> </div> </li> <li class="navbar-nav-item show-sm hide-md hide-lg hide-xl"> <a href="javascript:postMessage({type:'HS_DISPLAY_CALL_TO_ACTION',id:130207947529});" class="navbar-nav-item-link">Contact</a> </li> <li class="navbar-nav-item show-sm hide-md hide-lg hide-xl"> <a href="#" class="navbar-nav-item-link">Our Brands <i class="fa-solid fa-chevron-down"></i></a> <div class="navbar-subnav"> <div class="subnav-items"> <h2> Explore our brands </h2> <div class="desc"> Our brands bring together the industry's most influential leaders and scientific experts, inspiring thought-provoking and insightful content that offers a unique perspective on R&D trends and challenges. </div> <div class="subnav-item"> <div class="subnav-item-content"> <a href="/">Oxford Global</a> </div> </div> <div class="subnav-item"> <div class="subnav-item-content"> <a href="/precision-medicine">Precision Medicine</a> </div> </div> <div class="subnav-item"> <div class="subnav-item-content"> <a href="/discovery-development">Discovery & Development</a> </div> </div> <div class="subnav-item"> <div class="subnav-item-content"> <a href="/nextgen-biomed">NextGen Biomed</a> </div> </div> </div> </div> </li> </ul> </div> <div class="flex flex-align-center hide-sm show-md show-lg show-xl"> <a href="javascript:postMessage({type:'HS_DISPLAY_CALL_TO_ACTION',id:130207947529});" class="navbar-button">Contact</a> </div> </div> </header></div> <main id="main-content"> <div class="resource-single"> <div class="header bg-primary-light"> <div class="container-wrapper"> <div class="wide-container header-container"> <div class="header-content flex-1"> <div class="heading-smallcaps"> Cell Line Development | Industry Spotlights & Insight Articles </div> <h1 class="heading"> Enhancing Gene Editing Efficiency: Multi-Tailed mRNA Integration in CRISPR-Cas9 Systems </h1> <div class="byline"> Edited by Lucia Simmen | 27 March 2024 </div> <div class="abstract"> A study conducted by the Broad Institute and the Massachusetts Institute of Technology (MIT) demonstrated that when multi-tailed mRNAs were integrated into a CRISPR-Cas9 system they proved to be more efficient gene editors than unmodified mRNA. </div> </div> <div class="header-graphic flex-1"> <img src="https://oxfordglobal.com/hubfs/Enhancing%20Gene%20Editing%20Efficiency%20Multi%20Tailed%20mRNA%20Integration%20in%20CRISPR%20Cas9%20Systems.png" class="header-graphic-image"> </div> </div> </div> </div> <div class="container-wrapper"> <div class="wide-container"> <div class="content"> <div class="content-body text-gap"> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"><strong>Creating Multi-Tailed mRNA</strong></p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"><strong> </strong></p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">There is a growing expectation that mRNA will have broad therapeutic applications following its success and essential role in multiple COVID-19 vaccines. To build on mRNA's therapeutic potential, scientists from the Broad Institute and MIT engineered multi-tailed mRNAs.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">Their mRNA boosted mRNA activity levels in cells by as much as 20 times. Furthermore, their experiment showed that the mRNA lasted 2-3 times longer in animals in comparison to unmodified mRNA. </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">The <a href="https://www.genengnews.com/topics/genome-editing/mrna-therapeutics-may-act-longer-if-given-multiple-tails/" rel="noopener" target="_blank"><span style="color: #4a6ee0;">poly(A) tail of mRNA plays a vital part in protecting mRNA from degradation</span></a>. Xiao Wang, Assistant Professor of Chemistry at MIT, and her research group showed that chemically modifying the poly(A) tail using deadenylase-resistant oligonucleotides slowed the natural decay of mRNA and improved protein production making them useful for various therapies.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">Researchers hypothesised that engineering a more complex mRNA structure with multiple modified poly(A)tails instead of a single tail would enhance the therapeutic effects. According to a paper published in <a href="https://www.nature.com/articles/s41587-024-02174-7" rel="noopener" target="_blank"><em><span style="text-decoration: none;">Nature Biotechnology</span></em></a>, Wang, expressed the importance of modifying mRNA by stating that non-natural structures can function much better than naturally occurring ones.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">The team tested the multi-tailed mRNAs in human cell lines. The outcome showed that these mRNA sustained mRNA translation significantly longer than natural mRNA and mRNA-oligo conjugates (mocRNA), producing 5 - 20 times more proteins per dose over time.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"><strong>RELATED: </strong></p> <ul> <li style="text-align: justify;"><span style="color: windowtext;"><a href="https://oxfordglobal.com/cell/resources/abundant-stochastic-off-target-activity-by-base-editors" rel="noopener" target="_blank"><span style="color: #4a6ee0;">Abundant Stochastic Off-Target Activity by Base Editors</span></a></span> </li> <li style="text-align: justify;"><span style="color: windowtext;"><a href="https://oxfordglobal.com/cell/resources/when-to-cut-and-when-to-inhibit-a-low-throughput-hands-on-investigation-of-crispr-cut-and-crispri-efficiencies-in-ipsc-and-neural-cells" rel="noopener" target="_blank"><span style="color: #4a6ee0;">When to Cut and When to Inhibit? A Low-Throughput, Hands-On Investigation Of CRISPR-Cut and CRISPRi Efficiencies In iPSC And Neural Cells</span></a></span> </li> <li style="text-align: justify;"><span style="color: windowtext;"><a href="https://oxfordglobal.com/cell/resources/cell-line-engineering" rel="noopener" target="_blank"><span style="color: #4a6ee0;">Cell Line Engineering </span></a></span></li> </ul> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">Researchers took the experiment a step further by <a href="https://chemistry.mit.edu/chemistry-news/messenger-rnas-with-multiple-tails-could-lead-to-more-effective-therapeutics/" rel="noopener" target="_blank"><span style="color: #4a6ee0;">using the multi-tailed mRNA to encode the Cas9 protein as part of a CRISPR gene-editing system and tested it in mice models</span></a>. They used the gene editor to edit 2 genes implicated in high cholesterol - Pcsk9 and Angptl3.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">Results showed that a single dose of multi-tailed Cas9 mRNA induced high levels of gene editing and a lower cholesterol level in the mice's bloodstream compared to the control.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"><strong>Challenges</strong></p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">The positive results of testing in human cell lines and mice have boosted researchers' confidence regarding the potential for using their engineered <a href="https://oxfordglobal.com/cell/events/gene-therapy-development-2024" rel="noopener" target="_blank">mRNAs in therapies that edit genes</a> or replace faulty proteins. However, this study is in its infancy and still faces several significant obstacles.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">The main challenge involves striking a balance between therapeutic stability and activity. The<a href="https://phys.org/news/2024-03-messenger-rnas-multiple-tails-effective.html" rel="noopener" target="_blank"> mRNA in COVID-19 vaccines was effective because only a small amount was needed</a>.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">However, for that same type of mRNA to produce enough proteins to treat diseases that disrupt the regular production of essential proteins, a much larger dose would be needed which could produce toxic side effects. </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">This group of researchers aim to address logistical challenges. For example, they will focus on making the multi-tailed mRNA synthesis process more scalable and tweaking the purification process.</p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"><strong>Conclusion</strong></p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;"> </p> <p style="margin-top: 0cm; margin-right: 0cm; margin-bottom: 0cm; text-align: justify; padding-left: 0cm;">To conclude, this case study shows the potential of modifying mRNA to enhance therapeutic outcomes. Combining gene editing techniques such as CRISPR-Cas9 with mRNA modification could have a significant impact on treating complex genetic diseases. </p> <p style="text-align: justify;"> </p> <div class="content-body-footer"> </div> </div> <div class="content-sidebar"> <div class="related-articles"> <h3 class="center">Related Resources</h3> <div class="article"> <a href="/nextgen-biomed/resources/univercell-engineering-red-blood-cells-for-targeted-therapy"> <div class="cover-graphic"> <img src="https://oxfordglobal.com/hubfs/UNIVERCell%20Engineering%20Red%20Blood%20Cells%20for%20Targeted%20Therapy.png" class="cover-graphic-image"> </div> </a><div class="content"><a href="/nextgen-biomed/resources/univercell-engineering-red-blood-cells-for-targeted-therapy"> <h4> UNIVERCell: Engineering Red Blood Cells for Targeted Therapy </h4> <div class="abstract"> Scarlet Therapeutics uses genetic manipulation of red blood cells to carry a higher load of therapeutic proteins to help treat rare metabolic diseases. </div> <div class="read-more"> <a href="/nextgen-biomed/resources/univercell-engineering-red-blood-cells-for-targeted-therapy" class="read-more-link">Read More</a> </div></a> </div> </div> <div class="article"> <a href="/nextgen-biomed/resources/does-plate-choice-change-sampling-accuracy"> <div class="cover-graphic"> <img src="https://oxfordglobal.com/hubfs/Website/Images/Resources/Cell%20and%20Gene/Does-Plate-Choice-Change-Sampling-Accuracy.jpg" class="cover-graphic-image"> </div> </a><div class="content"><a href="/nextgen-biomed/resources/does-plate-choice-change-sampling-accuracy"> <h4> Does Plate Choice Change Sampling Accuracy </h4> <div class="abstract"> </div> <div class="read-more"> <a href="/nextgen-biomed/resources/does-plate-choice-change-sampling-accuracy" class="read-more-link">Read More</a> </div></a> </div> </div> </div> </div> </div> </div> </div> </div> <div class="newsletter-cta"> <h2 class="heading">Subscribe to our newsletter</h2> <div class="message"> Sign up for our monthly Newsletter to keep up with all things NextGen Biomed </div> <div class="button-wrapper"> <a href="#" class="button hs-cta-trigger-button hs-cta-trigger-button-177053686099">Newsletter Sign-Up</a> </div> </div> </main> <div data-global-resource-path="git/og-stage/templates/partials/footer.html"><footer class="website-footer flex flex-column g-20"> <div class="footer-columns"> <div class="footer-column"> <a href="https://oxfordglobal.com" class="site-logo-link"> <img src="https://oxfordglobal.com/wp-content/uploads/2023/03/Oxford-Global-Logo-Stacked-Websize.png" class="site-logo"> </a> </div> <div class="footer-column"> <h3>Brands</h3> <ul> <li> <a href="/precision-medicine/">Precision Medicine</a> </li> <li> <a href="/discovery-development/">Discovery & Development</a> </li> <li> <a href="/nextgen-biomed/">NextGen Biomed</a> </li> </ul> </div> <div class="footer-column"> <h3>Services</h3> <ul> <li> <a href="https://oxfordglobal.com/in-person-services/"> In-Person Services </a> </li> <li> <a href="https://oxfordglobal.com/digital-marketing-services/"> Digital Marketing Services </a> </li> </ul> <h3>Upcoming Events</h3> <ul> <li> <a href="https://oxfordglobal.com/calendar/"> Event Calendar </a> </li> </ul> </div> <div class="footer-column"> <h3>Company</h3> <ul> <li class="elementor-icon-list-item"> <a href="https://oxfordglobal.com/about-us/"> About </a> </li> <li class="elementor-icon-list-item"> <a href="https://oxfordglobal.com/vacancies/"> Vacancies </a> </li> <li class="elementor-icon-list-item"> <a href="https://oxfordglobal.com/csr-policy/"> CSR Policy </a> </li> <li class="elementor-icon-list-item"> <a href="javascript:postMessage({type:'HS_DISPLAY_CALL_TO_ACTION',id:130207947529});"> Contact </a> </li> </ul> </div> <div class="footer-column"> <p> <a href="tel:+441865248455">+44 1865 248 455</a><br> <a href="mailto:help@oxfordglobal.com">help@oxfordglobal.com</a> </p> <p> <strong>Our UK Address:</strong><br> Godstow Court<br> Minns Business Park<br> Botley<br> Oxford, OX2 0JB </p> <p> <strong>Our US Address:</strong><br> 100 Cambridge Street<br> 14th Floor<br> Boston<br> MA 02114 </p> </div> </div> <div class="footer-bottom grid g-20"> <div class="span8 span12-sm span12-md flex flex-column g-10"> <div class="copyright"> © Oxford Global Marketing Ltd. All rights reserved. </div> <ul class="legal-links flex flex-row g-30 flex-column-sm g-sm-10"> <li><a href="https://oxfordglobal.com/legal/">Terms & Conditions</a></li> <li><a href="https://oxfordglobal.com/legal/privacy-policy/">Privacy Policy</a></li> <li><a href="https://oxfordglobal.com/legal/website-terms/">Cookies</a></li> <li><a href="https://www.wishagency.co.uk/" target="_BLANK">Digital Agency Wish</a></li> </ul> </div> <div class="span4 span12-sm span12-md flex-align-end"> <ul class="social-links flex flex-row flex-justify-end flex-justify-center-sm flex-justify-center-md g-30"> <li><a href="https://www.facebook.com/OGConferences/" target="_BLANK"><i class="fa-brands fa-square-facebook"></i></a></li> <li><a href="https://www.linkedin.com/company/oxford-global/" target="_BLANK"><i class="fa-brands fa-linkedin"></i></a></li> <li><a href="https://twitter.com/OGConferences" target="_BLANK"><i class="fa-brands fa-square-x-twitter"></i></a></li> </ul> </div> </div> </footer></div> </div> <script src="/hs/hsstatic/jquery-libs/static-1.1/jquery/jquery-1.7.1.js"></script> <script>hsjQuery = window['jQuery'];</script> <!-- HubSpot performance collection script --> <script defer src="/hs/hsstatic/content-cwv-embed/static-1.1293/embed.js"></script> <script src="https://oxfordglobal.com/hs-fs/hub/8696823/hub_generated/template_assets/182600797781/1731148644227/git/og-stage/js/inlineSVG.min.js"></script> <script src="https://oxfordglobal.com/hs-fs/hub/8696823/hub_generated/template_assets/135724366742/1725380923802/git/og-stage/js/main.min.js"></script> <script> function getVideoId(url) { var regExp = /^.*(youtu.be\/|v\/|u\/\w\/|embed\/|watch\?v=|\&v=)([^#\&\?]*).*/; var match = url.match(regExp); if (match && match[2].length == 11) { return match[2]; } else { return false; } } function getVideoEmbed(id) { return '<iframe src="https://www.youtube.com/embed/' + id + '" title="YouTube video player" frameborder="0" allow="accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share" allowfullscreen style="aspect-ratio: 16 / 9; width: 100%;"></iframe>'; } jQuery("div.content-body .wp-block-embed__wrapper:contains('youtu')").each(function() { var yt_url = $(this).html().trim(); var yt_id = getVideoId(yt_url); var yt_embed = getVideoEmbed(yt_id); $(this).html(yt_embed); }); </script> <!-- Start of HubSpot Analytics Code --> <script type="text/javascript"> var _hsq = _hsq || []; _hsq.push(["setContentType", "standard-page"]); _hsq.push(["setCanonicalUrl", "https:\/\/oxfordglobal.com\/nextgen-biomed\/resources\/enhancing-gene-editing-efficiency-multi-tailed-mrna-integration-in-crispr-cas9-systems"]); _hsq.push(["setPageId", "hubdb-177607186815-30817717-181072999944"]); _hsq.push(["setContentMetadata", { "contentPageId": "hubdb-177607186815-30817717-181072999944", "legacyPageId": "hubdb-177607186815-30817717-181072999944", "contentFolderId": null, "contentGroupId": null, "abTestId": null, "languageVariantId": 177607186815, "languageCode": "en", }]); </script> <script type="text/javascript" id="hs-script-loader" async defer src="/hs/scriptloader/8696823.js"></script> <!-- End of HubSpot Analytics Code --> <script type="text/javascript"> var hsVars = { render_id: "1a8fa01e-2fa6-46b9-b05e-254cae304797", ticks: 1732678910377, page_id: 177607186815, dynamic_page_id: "hubdb-177607186815-30817717-181072999944", content_group_id: 0, portal_id: 8696823, app_hs_base_url: "https://app.hubspot.com", cp_hs_base_url: "https://cp.hubspot.com", language: "en", analytics_page_type: "standard-page", scp_content_type: "", analytics_page_id: "hubdb-177607186815-30817717-181072999944", category_id: 1, folder_id: 0, is_hubspot_user: false } </script> <script defer src="/hs/hsstatic/HubspotToolsMenu/static-1.354/js/index.js"></script> </body></html>