Different Approaches | ASGCT - American Society of Gene & Cell Therapy |

<!DOCTYPE html> <html xmlns="http://www.w3.org/1999/xhtml"> <head>  <script type="text/javascript">(function (w, d, s, l, i) { w[l] = w[l] || []; w[l].push({ 'gtm.start': new Date().getTime(), event: 'gtm.js' }); var f = d.getElementsByTagName(s)[0], j = d.createElement(s), dl = l != 'dataLayer' ? '&l=' + l : ''; j.async = true; j.src = 'https://www.googletagmanager.com/gtm.js?id=' + i + dl; f.parentNode.insertBefore(j, f); })(window, document, 'script', 'dataLayer', 'GTM-NFW5DS9');</script>   <script type="text/javascript"> //------------------------------------------------------- var z_account = "2632C1D8-6432-4D3A-93D6-08AAECC53F86"; var z_collector = "asgct.informz.net"; var z_cookieDomain = ".asgct.org"; //------------------------------------------------------- (function (e, o, n, r, t, a, s) { e[t] || (e.GlobalSnowplowNamespace = e.GlobalSnowplowNamespace || [], e.GlobalSnowplowNamespace.push(t), e[t] = function () { (e[t].q = e[t].q || []).push(arguments) }, e[t].q = e[t].q || [], a = o.createElement(n), s = o.getElementsByTagName(n)[0], a.async = 1, a.src = r, s.parentNode.insertBefore(a, s)) }(window, document, "script", "https://cdn.informz.net/web_trk/sp.js", "informz_trk")), informz_trk("newTracker", "infz", z_collector + "/web_trk/collector/", { appId: z_account, cookieDomain: z_cookieDomain }), informz_trk("setUserIdFromLocation", "_zs"), informz_trk("enableActivityTracking", 30, 15); informz_trk("trackPageView", null); </script>  <meta charset="utf-8" /> <meta http-equiv="X-UA-Compatible" content="IE=edge" /> <meta name="viewport" content="width=device-width,user-scalable=no,maximum-scale=1.0,initial-scale=1.0" /> <link href="/BlueKey/pe_assets/build/styles.css" rel="stylesheet" /> <script type="text/javascript" defer="defer" src="https://extend.vimeocdn.com/ga/127349852.js"></script> <meta name="twitter:card" content="summary_large_image" /> <meta name="twitter:site" content="https://twitter.com/ASGCTherapy" <meta name="twitter:creator" content="@ASGCTherapy" <meta name="twitter:title" content="Gene Therapy Approaches" <meta name="twitter:description" content="Learn more about some common approaches that target the underlying problem including: gene addition, gene editing, RNA therapy and cell therapy." <meta name="twitter:image" content="~/getattachment/Gene-Therapy-101/Gene-Therapy-Approaches/Screen-Shot-2023-01-20-at-3-16-26-PM.png.aspx" <meta name="description" content="Gene therapy, cell therapy, and gene editing are fields of biomedical research with a similar goal in mind: To treat disease by changing our bodies at a microscopic level." /> <meta charset="UTF-8" /> <link href="/App_Themes/Default/Images/favicon.ico" type="image/x-icon" rel="shortcut icon" /> <link href="/App_Themes/Default/Images/favicon.ico" type="image/x-icon" rel="icon" /> <title> Different Approaches | ASGCT - American Society of Gene & Cell Therapy | </title></head> <body class="LTR IE IE7 ENUS ContentBody">  <noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-NFW5DS9" height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript>  <form method="post" action="/gene-therapy-101/gene-therapy-approaches" id="aspnetForm"> <div class="aspNetHidden"> <input type="hidden" name="__CMSCsrfToken" id="__CMSCsrfToken" value="KddrgqU5azuFrtc4dj8GAm8sfHkOEk8B4tVx+/BLZEZr0xFngtJRXQue0b//CPejoxel3yPa0HQQZzPobBNWrHdVnNE=" /> <input type="hidden" name="__EVENTTARGET" id="__EVENTTARGET" value="" /> <input type="hidden" name="__EVENTARGUMENT" id="__EVENTARGUMENT" value="" /> <input type="hidden" name="__VIEWSTATE" id="__VIEWSTATE" value="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" /> </div> <script type="text/javascript"> //<![CDATA[ var theForm = document.forms['aspnetForm']; if (!theForm) { theForm = document.aspnetForm; } function __doPostBack(eventTarget, eventArgument) { if (!theForm.onsubmit || (theForm.onsubmit() != false)) { theForm.__EVENTTARGET.value = eventTarget; theForm.__EVENTARGUMENT.value = eventArgument; theForm.submit(); } } //]]> </script> <script src="/WebResource.axd?d=UZQNzmZXG1IdUaHxtavOjRvGEvOmz1kvw97PqEaxzBy7Ys-tUotw-RqgdbsvC3g_7cXjehqAdA_N6kbuK73WBAVGdxo1&t=638562384440000000" type="text/javascript"></script> <input type="hidden" name="lng" id="lng" value="en-US" /> <script type="text/javascript"> //<![CDATA[ function PM_Postback(param) { if (window.top.HideScreenLockWarningAndSync) { window.top.HideScreenLockWarningAndSync(1080); } if(window.CMSContentManager) { CMSContentManager.allowSubmit = true; }; __doPostBack('ctl00$CMSPortalManager1$am',param); } function PM_Callback(param, callback, ctx) { if (window.top.HideScreenLockWarningAndSync) { window.top.HideScreenLockWarningAndSync(1080); }if (window.CMSContentManager) { CMSContentManager.storeContentChangedStatus(); };WebForm_DoCallback('ctl00$CMSPortalManager1$am',param,callback,ctx,null,true); } //]]> </script> <script src="/ScriptResource.axd?d=WTJtoz6EJe-loUBJ9stbRxqzgpVp8oo0KdZGkhGfcu6Lr7kb5LeNfx21FKi8mxEL1oDZOLAxiAqERpClgBx-A6lZ0DJ1qKcDZB1NuIbRd8o9S7aG0&t=7c776dc1" type="text/javascript"></script> <script src="/ScriptResource.axd?d=y6KGkkRaceYF_mkApXCXMQ_rMQVX59BlJ-ut-lQDHw88bR5bbS86epdVNwimj01duE6_OophLX4sBTOgb8_5dNLaY2Mx5DHMJj7Rbr4kPMV1-AOS5VF5-QEC8oG-_-aHoWoyqA2&t=7c776dc1" type="text/javascript"></script> <script type="text/javascript"> //<![CDATA[ var CMS = CMS || {}; CMS.Application = { "isDialog": false, "isDebuggingEnabled": false, "applicationUrl": "/", "isRTL": "false", "imagesUrl": "/CMSPages/GetResource.ashx?image=%5bImages.zip%5d%2f" }; //]]> </script> <div class="aspNetHidden"> <input type="hidden" name="__VIEWSTATEGENERATOR" id="__VIEWSTATEGENERATOR" value="AD1448C3" /> <input type="hidden" name="__EVENTVALIDATION" id="__EVENTVALIDATION" value="/wEdAAPoNqHlTSDZ96ws6cDK31uIDMN6QAizBz4KoPbUd79oAZD1NQdfl/cWuar5Vh7dGPT0N2lV4Pe7fFq0WzNL590A/qxEcQ==" /> </div> <script type="text/javascript"> //<![CDATA[ Sys.WebForms.PageRequestManager._initialize('ctl00$manScript', 'aspnetForm', ['tctl00$ctxM',''], [], [], 90, 'ctl00'); //]]> </script> <div id="ctl00_ctxM"> </div> <header class="c-header"> <a href="#content" class="show-on-focus">Skip to main content</a> <div class="c-header-utilities"> <div class="container"> <div class="columns is-vcentered"> <div class="column"> <a href="https://asgct.org" class="is-external">ASGCT.org</a> </div> <div class="column is-half"> <a href="/"> <div class="c-header-images"> <div class="c-header-image"> <img src="/PatientEducation/media/PatientEducation/ASGCT_Web_Horz_Navy.png?ext=.png" alt="" /> </div> <div class="c-header-image"> <img src="/PatientEducation/media/PatientEducation/temp-pe-logo.png?ext=.png" alt="Patient Education Logo" /> </div> </div> </a> </div>  <div class="column"> <div class="c-header-search"> <div class="c-header-search-wrap"> <input name="keyword" type="text" id="searchtext" placeholder="Search" aria-required="false" /> <label for="searchtext" class="is-sr-only">Keyword search</label> <button type="button" data-encode="searchtext" data-path="/search-results" data-query="searchtext"> <span class="is-sr-only">Search</span> <span class="c-search-icon"></span> </button> </div> </div> </div> </div> </div> </div> <div class="c-header-utilities-mobile"> <div class="c-header-images"> <div class="c-header-image"> <a href="https://asgct.org" class="is-external"><img src="/PatientEducation/media/PatientEducation/ASGCT_Web_Horz_Navy.png?ext=.png" alt="" /></a> </div> <div class="c-header-image"> <a href="/"><img src="/PatientEducation/media/PatientEducation/temp-pe-logo.png?ext=.png" alt="Patient Education Logo" /></a> </div> </div> <button class="c-nav-toggle" type="button" aria-controls="c-nav-main" aria-expanded="false" data-togglevisibility="data-togglevisibility"> <p class="is-sr-only">Open Navigation</p> <p class="is-sr-only" aria-hidden="true">Close Navigation</p> <span class="c-nav-toggle-menu"></span> </button> </div> <div id="ctl00_plcMain_bkSiteHeader_headerBanner"> <div class="header-alert"> <div class="container"> <p id="ctl00_plcMain_bkSiteHeader_pBanner"><b><a href="https://patienteducation.asgct.org/newsletter/subscribe-to-the-patient-press">Subscribe today to ASGCT's patient-centered newsletter, The Patient Press!</a></b></p> </div> </div> </div> <nav class="c-nav-main" id="c-nav-main" aria-hidden="true"> <div class="container"> <div class="c-header-search is-hidden-desktop"> <div class="c-header-search-wrap"> <input name="" type="text" id="global-search" placeholder="Search" aria-required="false" /> <label for="global-search" class="is-sr-only">Keyword search</label> <button type="button" data-encode="global-search" data-path="/search-results" data-query="searchtext"> <span class="is-sr-only">Search</span> <span class="c-search-icon"></span> </button> </div> </div> <ul> <li> <a href="/gene-therapy-101">Gene & Cell Therapy 101</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/gene-therapy-101/gene-therapy-basics">Gene Therapy Basics</a> </li> <li> <a href="/gene-therapy-101/cell-therapy-basics">Cell Therapy Basics</a> </li> <li> <a href="/gene-therapy-101/gene-therapy-approaches">Gene Therapy Approaches</a> </li> <li> <a href="/gene-therapy-101/gene-editing">Gene Editing</a> </li> <li> <a href="/gene-therapy-101/clinical-trials">Clinical Trials</a> </li> <li> <a href="/gene-therapy-101/vectors-101">Vectors 101</a> </li> <li> <a href="/gene-therapy-101/glossary">Glossary</a> </li> <li> <a href="/gene-therapy-101/covid19-vaccines">COVID-19</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/disease-treatments">Condition Treatments</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/disease-treatments/fda-approved-cell-gene-therapy-products">FDA-Approved Cell & Gene Therapy Products</a> </li> <li> <a href="/disease-treatments/batten">Batten Disease</a> </li> <li> <a href="/disease-treatments/blood-disorders">Beta Thalassemia & Other Blood Disorders</a> </li> <li> <a href="/disease-treatments/leukodystrophy">CALD and MLD</a> </li> <li> <a href="/disease-treatments/car-t-basics">CAR-T Basics</a> </li> <li> <a href="/disease-treatments/fabry">Fabry Disease</a> </li> <li> <a href="/disease-treatments/gaucher-disease">Gaucher Disease</a> </li> <li> <a href="/disease-treatments/gm1-gangliosidosis">GM1 Gangliosidosis</a> </li> <li> <a href="/disease-treatments/gm2-tay-sachs-sandhoff">Tay-Sachs and Sandhoff Disease</a> </li> <li> <a href="/disease-treatments/hemophilia">Hemophilia</a> </li> <li> <a href="/disease-treatments/huntingtons-disease">Huntington's Disease</a> </li> <li> <a href="/disease-treatments/inherited-retinal-diseases">Inherited Retinal Diseases</a> </li> <li> <a href="/disease-treatments/mps">Mucopolysaccharidoses (MPS)</a> </li> <li> <a href="/disease-treatments/muscular-dystrophy">Muscular Dystrophy</a> </li> <li> <a href="/disease-treatments/pompe-disease">Pompe Disease</a> </li> <li> <a href="/disease-treatments/parkinsons-disease">Parkinson's Disease</a> </li> <li> <a href="/disease-treatments/sickle-cell-disease">Sickle Cell Disease</a> </li> <li> <a href="/disease-treatments/sma">Spinal Muscular Atrophy</a> </li> <li> <a href="/disease-treatments/wilson-disease">Wilson Disease</a> </li> <li> <a href="/disease-treatments/xlmtm">X-Linked Myotubular Myopathy</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/patient-journey">Patient Journey</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/patient-journey/identifying-a-genetic-disease">Identifying a Genetic Disease</a> </li> <li> <a href="/patient-journey/next-steps-with-a-diagnosis">Next Steps With a Diagnosis</a> </li> <li> <a href="/patient-journey/patient-focused-policy-positions">Patient-Focused Policy Positions</a> </li> <li> <a href="/patient-journey/stimulating-research">Stimulating Research</a> </li> <li> <a href="/patient-journey/considering-a-clinical-trial">Considering a Clinical Trial</a> </li> <li> <a href="/patient-journey/ethical-issues-germline-gene-editing">Ethical Issues: Germline Gene Editing</a> </li> <li> <a href="/patient-journey/ethical-issues-illegitimate-clinical-trials">Ethical Issues: Unproven Stem Cell Products</a> </li> <li> <a href="/patient-journey/gene-therapy-centers">Gene Therapy Centers</a> </li> <li> <a href="/patient-journey/clinical-trials-finder">Clinical Trials Finder</a> </li> <li> <a href="/patient-journey/on-demand">On Demand Webinars</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/newsletter">Newsletter</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/newsletter/subscribe-to-the-patient-press">Subscribe to The Patient Press</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/quick-takes">Quick Takes</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/quick-takes/focused-ultrasound-in-cell-and-gene-therapies">Focused Ultrasound in CGTs</a> </li> <li> <a href="/quick-takes/access-to-approved-cell-and-gene-therapies">Access to Approved CGTs</a> </li> <li> <a href="/quick-takes/informed-consent">Informed Consent</a> </li> <li> <a href="/quick-takes/state-of-the-field-cgt-highlights-and-approvals-20">State of the Field: CGT Highlights and Approvals of 2023</a> </li> <li> <a href="/quick-takes/genetic-testing-and-clinical-trial-eligibility">Genetic Testing and Clinical Trial Eligibility</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/events">Events</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/events/empowering-patients-2025-a-cell-and-gene-therapies">Empowering Patients 2025: A Cell and Gene Therapies Summit</a> </li> <li> <a href="/events/annual-meeting-patient-advocate-offerings">Annual Meeting-Patient Advocate Offerings</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/about-asgct">About</a> <button type="button"><span class="is-sr-only">toggle subnavigation</span></button> <ul> <li> <a href="/about-asgct/overview">Overview</a> </li> <li> <a href="/about-asgct/about-asgct">About ASGCT</a> </li> <li> <a href="/about-asgct/news">ASGCT News</a> </li> <li> <a href="/about-asgct/privacy-policy">Privacy Policy</a> </li> </ul> <div class="navDropToggle"></div> </li> <li> <a href="/support">Support</a> </li> </ul> <a href="asgct.org" class="px-1 is-external is-hidden-desktop">ASGCT.org</a> </div> </nav> </header> <main class="c-main" id="content"> <section class="c-breadcrumbs"> <div class="container"> <span><a href="/">Home</a> > </span> <a href="/gene-therapy-101" title="Gene & Cell Therapy 101" class="CMSBreadCrumbsLink">Gene & Cell Therapy 101</a> > <span class="CMSBreadCrumbsCurrentItem">Gene Therapy Approaches</span> </div> </section> <section class="section"> <div class="container"> <div class="columns"> <div class="column is-8"> <h1 class="is-like-h2">Gene Therapy Approaches</h1> </div> </div> <div class="columns"> <div class="column is-8"> <div class="c-leader"> <div class="c-leader-content"><p>There are many possible ways to target a disease or genetic variation, so there are different gene and cell therapy approaches that can be considered. Learn more about some common approaches that target the underlying problem including: gene addition, gene editing, RNA therapy and cell therapy.</p></div> </div> <div class="content mb-6"> <p><iframe allow="accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture" allowfullscreen="" frameborder="0" height="315" src="https://www.youtube.com/embed/8DqG1D5DVUw" title="YouTube video player" width="560"></iframe> </p> <h2><strong>Use of Genetic Material</strong></h2> <p>As we describe the different approaches of gene and cell therapy we often say “genetic material” is used or delivered to cells. Genetic material is a broad term and there are different types that may be used.</p> <p><b>Genetic Material </b>most commonly refers to DNA or RNA. These are strings of molecules with the information to instruct cells to produce proteins. Proteins play an important role in how our body functions. </p> <p><strong><img alt="Screen-Shot-2023-01-20-at-12-18-58-PM.png" src="/getattachment/Gene-Therapy-101/Different-Approaches/Screen-Shot-2023-01-20-at-12-18-58-PM.png.aspx?width=300&height=171" style="width: 300px; height: 171px; float: right;" title="Screen-Shot-2023-01-20-at-12-18-58-PM.png" />DNA </strong>stores the genetic information so the cell can continue building proteins. It is permanent and stored in the nucleus in a form called chromosomes. DNA is made up of two strands and is much larger than RNA. Genes are specific sections of DNA that encode sequences (I.e. a set of instructions)<a> </a>for making proteins. Other sections of DNA can control when, where, and how much of the protein is made. </p> <p><strong>RNA</strong> is a copy of the genetic information contained within DNA. While DNA is the permanent storage format of genetic material, RNA molecules help turn those instructions into proteins. After RNA is made within the nucleus of a cell, most RNA moves to the cytoplasm, which is the fluid space between the nucleus and cell membrane. There are many different types of RNA. Some RNA carries the instructions for making proteins, while other types of RNA can prevent proteins from being made. RNA molecules are only active for a limited period of time in the cell.  Keep reading to learn more about the types of RNA used in RNA Therapies.</p> <p><a href="https://asgct.org/asgct-gene-therapy-approaches-infographic-jan22" target="_blank"><img alt="Add-a-heading.png" src="/getattachment/Gene-Therapy-101/Different-Approaches/Add-a-heading.png.aspx" style="width: 350px; height: 350px;" title="Add-a-heading.png" /></a></p> <h2><strong>Breaking Down the Approaches</strong></h2> <p><strong>Gene Therapy </strong>is the use of genetic material to treat or prevent disease. Learn more about <a href="/gene-therapy-101/gene-therapy-basics" target="_blank">Gene therapy Basics</a> or <a href="/gene-therapy-101/vectors-101" target="_blank">Vectors 101</a>. Three common effects of gene therapies in cells are: </p> <ul> <li><strong><img alt="ShapeDescription automatically generated" src="https://asgct.org/getmedia/fa8e46e0-d562-424a-8e7e-acf810e8e486/Screen-Shot-2023-01-20-at-12-00-42-PM.aspx?width=350&height=294" style="width: 350px; height: 294px; float: right; margin: auto 12px;" />Gene addition </strong>adds in a working gene that has the instructions for the cell to make more of the specific protein needed. Vectors, which are often viruses, are used to deliver the working gene to the cell’s nucleus, where the DNA is stored. This gene will now live in the nucleus which gives a greater chance of being permanent and is only given one time. Sometimes the therapy is designed for the new gene to insert itself into the main DNA storage while other times it will stay <em>next to</em> the main DNA storage, like an extra set of instructions.</li> <li><strong>Gene silencing</strong> is where the delivered genetic material prevents or inhibits the activity of a gene that is already present in a cell. Gene silencing often decreases the amount of a specific protein being made. </li> <li><strong>Gene </strong><strong>Editing </strong>corrects pieces of DNA by changing or deleting the information <em>within</em> the affected individual’s gene. Genetic material is sent to directly edit or change pieces of DNA already located within a cell to correct the protein being made by that DNA. Gene editing uses technology that is highly precise to make these types of changes. Learn more about <a href="/gene-therapy-101/gene-editing" target="_blank">Gene Editing</a>.</li> </ul> <blockquote> <p style="text-align: center;"><b>Please note</b>, gene therapy is a rapidly evolving field that changes in response to new scientific discoveries and from what is learned about treatments being tested in the clinic and laboratory.  Below we highlight different types of gene therapies and strategies that are currently being tested and may be approved for use in humans.  As DNA and RNA work together, there may be overlap in the way each is applied.</p> </blockquote> <p><strong>DNA therapy </strong>is the use of DNA that codes for the production of a specific RNA or protein to treat a disorder.  To have a therapeutic effect, the DNA must be delivered to the nucleus of a cell, where it can then be used by the cell to affect protein expression.  This gene will now live in the nucleus which gives a greater chance of being permanent and is typically only given one time. </p> <ul> <li><strong>DNA plasmids </strong>are large, double-stranded circular DNA molecules that code for a therapeutic protein.  DNA plasmids can be used for gene addition, vaccination, and cell therapy.  A challenge for DNA plasmids is getting them into the cell and then into the nucleus for therapeutic benefit.  Clinical trials are ongoing to test the safety and benefit of DNA plasmid therapeutics.</li> <li><strong>Viral vectors</strong> are modified viruses used as vehicles to deliver therapeutic genetic material or specific DNA sequences into a cell.  In a viral vector, the viral genes are removed and replaced by a therapeutic DNA sequence encodes genes, RNAs or other substances and packaged inside the shell of a virus.  The viral vector is then delivered directly to the body (in-vivo therapy) or to cells (ex-vivo therapy; see <strong>Gene-modified cell therapy</strong> below<strong>) </strong>to deliver the therapeutic genetic material to the nucleus of the cell. <img alt="DiagramDescription automatically generated" src="https://asgct.org/getmedia/94083675-f4fb-4672-8b9d-8cc29742deb3/Screen-Shot-2023-01-20-at-12-04-12-PM.aspx?width=360&height=234" style="width: 360px; height: 234px; float: right; margin: auto 12px;" />In-vivo viral vector therapies are frequently limited to a one-time delivery due to the innate immune response to the virus that usually prevents re-dosing. Learn more about innate immunity in <a href="/gene-therapy-101/vectors-101" target="_blank">Vectors 101</a>. <em>There are multiple FDA approved viral vector DNA therapies.</em></li> </ul> <p> </p> <p> </p> <p> </p> <p><strong>RNA therapy</strong> is the use of shorter sequences of genetic material in RNA format to treat or prevent a disease. There are many different types of RNA therapy because there are so many different types of RNA sequences that can affect cell functions. These types of therapies are delivered using viral vectors, or other non-viral vehicles such as lipid nanoparticles. They often need repeat dosing to maintain a therapeutic (good) effect since the DNA is not being altered or supplemented.  Let’s explore the different types of RNA and corresponding therapies: </p> <ul> <li><strong>Ribosomal RNA (rRNA)</strong> is theRNA that helps form ribosomes, which are the molecular machines used in building proteins. </li> <li><strong>Messenger RNA (mRNA)</strong> is a middle message that can move through different parts of the cell to provide instructions to make proteins. It is a single stand that carries the information, initially stored within DNA, out of the nucleus to the cytoplasm of a cell where proteins are made. <ul style="list-style-type:circle;"> <li><strong>mRNA therapy</strong> is designed to produce more of a specific protein when the gene for that protein is missing, not working the way it should, or is beneficial for our bodies to create. <strong>Lipid nanoparticles</strong> may be used as containers to deliver therapeutic mRNA into cells because its structure protects the contents from being degraded after being injected into the body. Learn more about <a href="/gene-therapy-101/covid19-vaccines/mrna-vaccines-for-covid19" target="_blank">mRNA in vaccines</a>.</li> </ul> </li> <li><strong>microRNA (miRNA)</strong> is a small form of single-strand RNA that typically targets multiple mRNAs to regulate expression of several different genes at the same time.   <ul style="list-style-type:circle;"> <li><strong>miRNA therapies </strong>can be in the form of synthetic, double-stranded miRNAs (also called miRNA mimics), recombinant expression vectors that carry miRNA encoding sequences (naturally occurring or artificial), and oligonucleotide-based inhibitors (anti-miRs).  <em>Testing of miRNA therapies are ongoing in clinical trials.</em></li> </ul> </li> <li><strong>Small interfering RNA (siRNA)</strong> are double stranded RNA molecules that usually target a specific mRNA to prevent production of unwanted proteins.  <ul style="list-style-type:circle;"> <li><strong>siRNA therapies</strong> are designed outside of the body to target expression of a specific gene.  <em>There are FDA approved siRNA therapies.</em></li> </ul> </li> <li><strong>Transfer RNA (tRNA) </strong>carry the building blocks of proteins, called amino acids, to the ribosome to help make a protein based off the mRNA instructions.  <ul style="list-style-type:circle;"> <li><strong>Suppressor tRNA therapies </strong>are designed to override incorrect mRNA instructions that may cause disease by stopping protein production too early.  Binding of suppressor tRNA to the incorrectly made mRNA allows the correct, full protein to be made by the ribosome.  <em>Suppressor tRNA therapies are still in the preclinical <a>p</a></em><em>hase.</em></li> </ul> </li> <li><b><img alt="Screen-Shot-2023-01-20-at-12-19-10-PM.png" src="/getattachment/Gene-Therapy-101/Different-Approaches/Screen-Shot-2023-01-20-at-12-19-10-PM.png.aspx?width=300&height=156" style="width: 300px; height: 156px; float: right;" title="Screen-Shot-2023-01-20-at-12-19-10-PM.png" />Antisense oligonucleotide</b><strong>s (ASO's)</strong> are synthetic (fake), single-stranded chains of molecules that target mRNA from a specific gene. <ul style="list-style-type:circle;"> <li><strong>A</strong><strong>SO therapies </strong>act inside a cell to alter how proteins are made. ASOs can silence a gene so a protein is not made. It can also alter how mRNA is made to then change how a protein is made. These stay in the cell for a limited duration and may need to be given repeatedly to maintain a therapeutic effect. <em>There are multiple FDA approved ASO therapies.</em></li> </ul> </li> <li><strong>RNA aptamers </strong>are short pieces of RNA that bind to a specific protein to control their functions (for example, blocking or activating).  <ul style="list-style-type:circle;"> <li><strong>Aptamer therapies </strong>are designed outside of the body. Unlike other RNA based therapeutics that must be brought inside a cell to be beneficial, RNA aptamers can tightly bind to proteins on the outside of the cell to provide unique therapeutic advantages<i>. There are FDA approved RNA apatmers.</i><i></i></li> </ul> </li> </ul> <p><strong>Cell therapy </strong>is the transfer of a specific cell type(s) into a patient to treat or prevent a disease. Depending on the cell therapy, the cells can come from either the affected individual or an unaffected donor.  Some cell therapies are more common, like a hematopoietic stem cell (blood forming cells) transplant. Depending on the treatment, conditioning to prepare the body to receive the biological material is done to reduce the risk of an immune response and help the body successfully accept the cells. <em>There are many FDA approved cell therapies.</em><a><em><b><img alt="Screen-Shot-2023-01-20-at-12-13-49-PM.png" src="/getattachment/Gene-Therapy-101/Different-Approaches/Screen-Shot-2023-01-20-at-12-13-49-PM.png.aspx?width=350&height=262" style="width: 350px; height: 262px; float: right;" title="Screen-Shot-2023-01-20-at-12-13-49-PM.png" /></b></em></a></p> <ul> <li><strong>Gene-modified cell therapy (or ex vivo gene therapy) </strong>is a combination of gene and cell therapy. It first removes a person’s own cells from the body. Certain cell types are then treated by either adding a working or healthy copy of the gene or modifying/editing the affected one. The modified or treated cells are then returned to the person. Learn more about the FDA approved <a href="/disease-treatments/car-t-basics" target="_blank">CAR T-cell Therapies</a> for certain forms of cancers.</li> </ul> <p> </p> <p>Next, explore how these approaches are used in various <a href="/disease-treatments" target="_blank">Disease Treatments</a>. Learn more about the potentials, risks, and challenges of <a href="/gene-therapy-101/gene-therapy-basics" target="_blank">gene therapy</a>.</p> <p> </p> <p><strong>Was this information helpful?</strong> If so, please share! All ASGCT Patient Education resources are free to use by sharing on social media, embedding the video, or simply linking to this page! <em>Please credit the American Society of Gene and Cell Therapy or tag </em><a href="https://twitter.com/ASGCTherapy" target="_blank"><em>@ASGCTherapy</em></a><em>. </em></p> <div> </div> </div> </div> <div class="column is-4 pl-4"> <div class="c-nav-local-wrap"> <div class="asideBox"> <h3> Related Pages</h3> <hr class="is-secondary" /> <ul class="c-nav-local-list"> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/gene-therapy-basics">Gene Therapy Basics</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/cell-therapy-basics">Cell Therapy Basics</a> </li> <li class="c-nav-local-item is-active"><a class="c-nav-local-link" href="/gene-therapy-101/gene-therapy-approaches">Gene Therapy Approaches</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/gene-editing">Gene Editing</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/clinical-trials">Clinical Trials</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/vectors-101">Vectors 101</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/covid19-vaccines">COVID-19</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/lunch-learn-gene-therapy-for-sma">Lunch & Learn: Gene Therapy for SMA</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/gene-therapy-101/helpful-websites">Helpful Websites</a> </li> </ul> </div> </div> </div> </div> </div> <div class="lastUpdatedTag"> <p>Last Updated: 08/30/2024</p> </div> </section> </main> <footer class="c-footer"> <div class="c-footer-primary"> <section class="section"> <div class="container"> <div class="columns"> <div class="column c-footer-section"> <p><img alt="" src="/PatientEducation/media/PatientEducation/logo_white.png" /></p> <p>20800 Swenson Dr., Suite 300<br /> Waukesha, WI 53186</p> </div> <div class="column is-two-fifths c-footer-section"> <ul class="c-utility-list"> <li><a href="#">Gene Therapy 101</a></li> <li><a href="#">Disease Treatments</a></li> <li><a href="#">Clinical Trials</a></li> <li><a href="/patient-journey">Patient Journey</a></li> <li><a href="/about-asgct">About</a></li> <li><a href="/support">Support</a></li> <li><a href="https://asgct.org/about/privacy-policy" target="_blank">Privacy Policy</a></li> </ul> </div> <div class="column c-footer-section"> <h3 class="mb-3">Connect with ASGCT</h3> <p class="mb-0"><span>Phone: 414</span>.278.1341</p> <p><a href="mailto:info@asgct.org">info@asgct.org</a></p> <p> <div class="c-social"> <ul class="c-social-list"> <li class="c-social-item"><a href="https://twitter.com/ASGCTherapy" target="_blank" class="c-social-link twitter"><span class="is-sr-only">Twitter</span></a></li> <li class="c-social-item"><a href="https://www.facebook.com/ASGCTherapy" target="_blank" class="c-social-link facebook"><span class="is-sr-only">Facebook</span></a></li> <li class="c-social-item"><a href="https://www.instagram.com/asgctherapy" target="_blank" class="c-social-link instagram"><span class="is-sr-only">Instagram</span></a></li> <li class="c-social-item"><a href="https://www.linkedin.com/company/asgct" target="_blank" class="c-social-link linkedin"><span class="is-sr-only">LinkedIn</span></a></li> <li class="c-social-item"><a href="https://www.youtube.com/user/ASGCT" target="_blank" class="c-social-link youtube"><span class="is-sr-only">YouTube</span></a></li> </ul> </div> </p> </div> </div> </div> </section> </div> <div class="c-footer-secondary"> <section class="section py-4"> <div class="container"> <div class="columns"> <div class="column"> <p class="is-small">© 2024 All rights reserved. American Society of Gene & Cell Therapy.</p> <p class="is-tiny">Web design by <a href="https://www.reasononeinc.com/kentico" target="_blank">Reason One</a>. Powered by <a href="http://www.kentico.com" target="_blank">Kentico</a>.</p> </div> </div> </div> </section> </div> </footer> <div class="CookieConsent"> <span id="ctl00_cookieLawQ_lblText" class="ConsentText">This site uses cookies to offer you a better user experience and to analyze site traffic. By continuing to use this website, you accept our use of cookies.</span> <span class="ConsentButtons"> <input type="submit" name="ctl00$cookieLawQ$btnAllowSpecific" value="OPT OUT" id="ctl00_cookieLawQ_btnAllowSpecific" class="ConsentButton btn btn-default" /> <input type="submit" name="ctl00$cookieLawQ$btnAllowAll" value="ACCEPT" id="ctl00_cookieLawQ_btnAllowAll" class="ConsentButton btn btn-default" /> </span> </div> <script src="/BlueKey/pe_assets/build/main.js" type="text/javascript"></script> <script type="text/javascript"> //<![CDATA[ var callBackFrameUrl='/WebResource.axd?d=nNME_ObE2oIDm535eYM9xWmIMy-HXBVdo54Ly6sA5tAYPS1aA-gFDzodlP9ijTBRgmjStONrH4-ISuRTp7slHiYkACw1&t=638562384440000000'; WebForm_InitCallback();//]]> </script> </form> </body> </html>

CINXE.COM

Different Approaches | ASGCT - American Society of Gene & Cell Therapy |