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Disease Treatments | ASGCT - American Society of Gene & Cell Therapy |
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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> <div class="c-banner" style="background: url('/getattachment/Disease-Treatments/Batten_Disease_1280x720_1_00000.png.aspx') 50%/cover no-repeat;"> <div class="c-banner-content container"> <h1>Condition Treatments</h1> <hr /> <p class="is-large">Gene and cell therapy research shows promise for treating certain rare inherited diseases and forms of cancer.</p> </div> </div> </header> <main class="c-main" id="content"> <section class="section wrap"> <div class="container"> <div class="is-flex-center tile is-ancestor py-5"> <div class="tile is-6 is-parent"> <a href="/Disease-Treatments/Batten" class="tile is-child c-tile-link-alternate"> <h2>Batten Disease</h2><p>Different forms of the disease being researched for potential treatments聽include CLN1, 2, 3, 5, 6, 7, and 8.</p> </a> </div> <div class="tile is-6 is-parent"> <a href="/disease-treatments/fabry" class="tile is-child c-tile-link-alternate"> <h2>Fabry Disease</h2><p>Gene therapy approaches target a faulty GLA gene.</p> </a> </div> </div> </div> </section> <section class="section wrap pattern pb-x1" style="background: url('/PatientEducation/media/PatientEducation/square-background.png') repeat;"> <div class="container pt-6"> <div class="columns"> <div class="column is-three-fifths is-offset-one-fifth is-centered"> <h2>Disease Resources</h2><p>Visit these pages for information on specific diseases and how gene therapy can help.</p> </div> </div> <div class="columns"> <div class="column is-10 is-offset-1"> <div class="c-tile-wrap mb-5"> <div class="c-tile"> <a href="/disease-treatments/blood-disorders" class="c-tile-link"> <h3>Blood Disorders</h3> <hr /> <p>Take a closer look at beta thalassemia and Fanconi anemia.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/car-t-basics" class="c-tile-link"> <h3>CAR-T Basics</h3> <hr /> <p>Learn about approved treatments for blood cancers, such as leukemia and lymphoma.</p> </a> </div> <div class="c-tile"> <a href="https://patienteducation.asgct.org/disease-treatments/fabry" class="c-tile-link"> <h3>Fabry Disease</h3> <hr /> <p>Learn how <em>in vivo</em> and <em>ex vivo</em> gene therapy approaches can be used to treat Fabry disease by delivering a working GLA gene into cells.</p> </a> </div> <div class="c-tile"> <a href="https://patienteducation.asgct.org/disease-treatments/gaucher-disease" class="c-tile-link"> <h3>Gaucher Disease</h3> <hr /> <p>Learn about this rare genetic disorder that causes the harmful buildup of glycolipids throughout the body and how gene therapy may be able to address its symptoms.</p> </a> </div> <div class="c-tile"> <a href="https://patienteducation.asgct.org/disease-treatments/gm1-gangliosidosis" class="c-tile-link"> <h3>GM1 Gangliosidosis </h3> <hr /> <p>Gene therapy for GM1 aims to be a one-time treatment that delivers a working copy of the <em>GLB1</em> gene into cells using a viral vector.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/hemophilia" class="c-tile-link"> <h3>Hemophilia</h3> <hr /> <p>Gene therapy has the potential to increase clotting factors to treat hemophilia A and B.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/huntingtons-disease" class="c-tile-link"> <h3>Huntington's Disease</h3> <hr /> <p>A genetic disorder caused by a breakdown of nerve cells in the brain.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/inherited-retinal-diseases" class="c-tile-link"> <h3>Inherited Retinal Diseases</h3> <hr /> <p>A closer look at gene therapy for Leber congenital amaurosis (LCA), retinitis pigmentosa, choroideremia, Stargardt’s disease, and achromatopsia.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/leukodystrophy" class="c-tile-link"> <h3>Leukodystrophies</h3> <hr /> <p>These are a set of rare genetic disorders including cerebral adrenoleukodystrophy (CALD), and metachromatic leukodystrophy (MLD).</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/mps" class="c-tile-link"> <h3>MPS</h3> <hr /> <p>Learn more about gene therapy research for mucopolysaccharidoses (MPS) types I, II, III and VI, also referred to as Hurler syndrome, Hunter syndrome, and Sanfilippo syndrome. </p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/muscular-dystrophy" class="c-tile-link"> <h3>Muscular Dystrophy</h3> <hr /> <p>Gene therapy has the potential to treat Duchenne and certain subtypes of limb-girdle muscular dystrophy.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/parkinsons-disease" class="c-tile-link"> <h3>Parkinson's Disease</h3> <hr /> <p>Learn about the potential to treat a degenerative neurological disease that over time causes the death of brain cells.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/condition-detail" class="c-tile-link"> <h3>Pompe Disease</h3> <hr /> <p>This rare genetic glycogen storage disease is caused by a faulty GAA gene.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/sickle-cell-disease" class="c-tile-link"> <h3>Sickle Cell Disease</h3> <hr /> <p>Learn how gene therapy and gene editing may address the genetic cause of sickle cell disease.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/sma" class="c-tile-link"> <h3>Spinal Muscular Atrophy</h3> <hr /> <p>Learn more about the FDA-approved gene therapy Zolgensma that targets the SMN1 gene.</p> </a> </div> <div class="c-tile"> <a href="https://patienteducation.asgct.org/disease-treatments/wilson-disease" class="c-tile-link"> <h3>Wilson Disease</h3> <hr /> <p>Gene therapy may target the cause of this disease, which causes excess copper to be stored in the body.</p> </a> </div> <div class="c-tile"> <a href="/disease-treatments/xlmtm" class="c-tile-link"> <h3>X-Linked Myotubular Myopathy</h3> <hr /> <p>Also known as XLMTM, this rare genetic disorder primarily causes muscle weakness caused by a faulty MTM1 gene.</p> </a> </div> </div> </div> </div> </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 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