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Breakthroughs in Muscular Dystrophy | Events | American Society of Gene & Cell Therapy ASGCT | ASGCT - American Society of Gene & Cell Therapy

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src="https://cdnjs.cloudflare.com/ajax/libs/iframe-resizer/4.3.1/iframeResizer.min.js?ver=6.0.2" id="iframeResizer-js"></script> <h2>Ready to Register?</h2> <p><a class="btn btn-accent3" href="https://www.xcdsystem.com/asgct/attendee/index.cfm?ID=4Pa6Ppp" target="_blank">Register now!</a></p> <h2>Program</h2> <h3>Tuesday, November 19</h3> <p><strong>Attendee Breakfast</strong><br /> 7:30 - 8:30 a.m.</p> <p><strong>Opening Remarks from Jeffrey Chamberlain, PhD</strong><br /> 8:30 - 8:35 a.m.</p> <p><strong>Keynote - Genetic Therapies for Muscular Dystrophies: Opportunities and Challenges</strong><br /> 8:35 - 9:35 a.m.<br /> <strong>Chair:&nbsp;</strong>Jeffrey Chamberlain, PhD, University of Washington</p> <ul> <li><strong>Cloning the Duchenne Gene - An Origin Story </strong><br /> Louis Kunkel, PhD, Children&#39;s Hospital Boston</li> <li><strong>Clinical Aspects of Gene Therapies for Muscular Dystrophies - Challenges and Opportunities </strong><br /> Carsten Bönnemann, MD, NINDS/NIH</li> </ul> <p><strong>Non-Viral Delivery</strong><br /> 9:35 - 10:35 a.m.<br /> <strong>Chair:&nbsp;</strong>Monkol Lek, PhD, Yale School of Medicine</p> <ul> <li><strong>Advancing Non-Viral Delivery via Battelle&#39;s HIT SCAN Platform</strong><br /> Kenneth Sims, PhD, Battelle Memorial Institute</li> <li><strong>A targeted, non-viral platform to deliver the full-length dystrophin gene</strong><br /> Nick Whitehead, PhD, Myosana Therapeutics</li> </ul> <p><strong>Coffee Break, Paid Presentation, and Exhibits</strong><br /> 10:35 - 11:05 a.m.</p> <p><img alt="" src="/getmedia/48d45120-8e56-4a57-966b-5ebe87d1f1d4/sarepta-(1).aspx?width=200&amp;height=100" style="width: 200px; height: 100px;" /></p> <p><strong>Viral Delivery</strong><br /> 11:05 a.m.&nbsp;- 12:05 p.m.<br /> <strong>Chair: </strong>Melissa Spencer, PhD, UCLA</p> <ul> <li><strong>Monitoring Viral Delivery with Non-invasive Imaging</strong><br /> Oluwatayo&nbsp;Ikotun, PhD, University of California, Los Angeles</li> <li><strong>Split-Inteins</strong><br /> Jeffrey Chamberlain, PhD, University of Washington</li> </ul> <p><strong>Attendee Lunch + Exhibits</strong><br /> 12:05 - 1:05 p.m.</p> <p><strong>Monitoring AAV Delivery</strong><br /> 1:05&nbsp;- 2:30 p.m.<br /> <strong>Chair:&nbsp;</strong>Carsten Bönnemann, MD, NINDS/NIH</p> <ul> <li><strong>Intro to Monitoring AAV Delivery</strong><br /> Federico Mingozzi, PhD, stealth company</li> <li> <p><strong>Vector Immunology</strong><br /> J. Fraser Wright, PhD, Stanford School of Medicine</p> </li> <li> <p><strong>Modeling and Tolerizing Dystrophin Immunity in Mice </strong><br /> Armando Villalta, PhD, UC Irvine</p> </li> <li> <p><strong>Enabling Readministration/Redosing</strong><br /> Barry Byrne, MD, PhD, University of Florida</p> </li> </ul> <p><strong>Coffee Break + Exhibits</strong><br /> 2:30 - 2:40 p.m.</p> <p><strong>Gene Editing</strong><br /> 2:40 - 3:30&nbsp;p.m.<br /> <strong>Chair:&nbsp;</strong>Angela Lek, PhD, VP of Research, MDA</p> <ul> <li><strong>A Novel Dual Vector Approach for Safe and Efficient Expression of Large Genes using RNA End-joining (REJ) </strong><br /> Samuel Pfaff, PhD, The Salk Institute for Biological Studies</li> <li><strong>Genome Editing for Duchenne Muscular Dystrophy</strong><br /> Charles Gersbach, PhD, Duke University</li> </ul> <p><strong>Coffee Break (Paid Presentation) + Exhibits</strong><br /> 3:30 - 4 p.m.</p> <p><img alt="" src="/ASGCT/media/about/emit-imaging-(2).png" /></p> <p><strong>Oral Abstract Session</strong><br /> 4 - 5:45 p.m.<br /> <strong>Chairs:&nbsp;</strong>Evrim Atas, PhD and Angela Lek, PhD MDA</p> <ul> <li> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{91}" paraid="1227743782"><strong>1. Editing of Postmitotic Muscle and Satellite Cells for Continuous Correction of Dystrophin Expression in Duchenne Muscular Dystrophy&nbsp;</strong><br /> Niclas Bengtsson, PhD, University of Washington&nbsp;</p> </li> <li> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{91}" paraid="1227743782"><strong>2. CSF Delivery of INS1201 AAV9-Micro-Dystrophin as a Potential Therapy for DMD&nbsp;</strong><br /> Gretchen Thomsen, PhD, Insmed, Inc.&nbsp;</p> </li> <li> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{91}" paraid="1227743782"><strong>3. Therapeutic Potential of ENTR-601-44, an Endosomal Escape Vehicle (EEVTM) - Oligonucleotide Conjugate for the Treatment of Exon 44 skip Amenable DMD&nbsp;</strong><br /> Mahasweta Girgenrath, PhD, Entrada Therapeutics Inc&nbsp;</p> </li> <li> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{149}" paraid="226360522"><strong>4. Development of Gene Therapy for LGMD-R9&nbsp;</strong><br /> Evelyne Gicquel, PhD, Genethon</p> </li> <li> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{149}" paraid="226360522"><strong>6. GNT0004, Genethon&#39;s AAV8 Vector-delivered Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy: First Data from Phase 1/2 Part of GNT-016-MDYF All-in-one Clinical Trial in Ambulant Boys&nbsp;</strong><br /> Serge Braun, PhD, AFM TELETHON / GENETHON &nbsp;</p> </li> <li> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{149}" paraid="226360522"><strong>58. RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data&nbsp;</strong><br /> Jahannaz Dastgir, DO, REGENXBIO Inc.&nbsp;</p> </li> <li> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>5. The Venture Philanthropy Model of Drug Development - Working with Non-Profit Foundations and Governments from Concept to Drug Approval&nbsp;</strong><br /> Eric Hoffman, PhD, Binghamton University&nbsp;</p> </li> </ul> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>Poster Reception + Exhibits</strong><br /> 5:45 - 7 p.m.</p> <p paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>Poster Hall 1: Great Lakes Room</strong></p> <ul> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>7. U7snRNA as a Tool for Restoring Dystrophin Expression in Patients with Exon 17 Skip-Amenable Deletions&nbsp;</strong><br /> Liubov Gushchina, PhD, The Ohio State University and Nationwide Children&#39;s Hospital RI&nbsp;<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>8. KT809 Dose-Dependently Improves Muscle Function in a Severe DMD Mouse Model and Results in High and Uniform Microdystrophin Expression in the Heart and Skeletal Muscles of Non-Human Primates&nbsp;</strong><br /> Chady Hakim, PhD, Kate Therapeutics&nbsp;<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>9. Full-Length Dystrophin Gene Delivery Using Triple MyoAAV Vectors&nbsp;</strong><br /> Renzhi Han, PhD, Indiana University School of Medicine<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"> <div class="ql-block" data-block-id="block-6136cf83-509f-4fe5-b8b8-32b248167bac"><strong>11. (Withdrawn) CRISPR/hfCas12Max DNA Editing Therapy Restores Dystrophin Protein and Muscle Functions in Duchenne Muscular Dystrophy</strong></div> <div class="ql-block" data-block-id="block-1a3a25f0-a3e0-40e1-8d9e-470aa9a2af6d">Guoling Li, PhD, HuidaGene Therapeutics Co., Ltd<br /> &nbsp;</div> </li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"> <div class="ql-block" data-block-id="block-1a3a25f0-a3e0-40e1-8d9e-470aa9a2af6d"> <div class="ql-block" data-block-id="block-49197343-6fca-4f34-b1f9-dd3fdade80f7"><strong>12. (Withdrawn) Precise Correction of Duchenne Muscular Dystrophy via Exon-Skipping Using Compact Base Editor</strong></div> <div class="ql-block" data-block-id="block-f964e661-c884-4f7f-bd57-1ac4fd982572">Guoling Li, PhD, HuidaGene Therapeutics Co., Ltd<br /> &nbsp;</div> </div> </li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong> 13. A Comparative Analysis of Long-Term and Short-Term Outcomes of AAV-CRISPR Treatment in a Canine Model of Duchenne Muscular Dystrophy&nbsp;</strong><br /> Rika Maruyama, PhD, University of Alberta&nbsp;<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"> <div class="ql-block" data-block-id="block-153639fc-7d0e-4f6d-afe8-81a297edb7a6"><strong>14. (Virtual) Rational Design and Rigorous Testing of Novel, Non-Immunogenic Dystrophin Mimetics Providing Superior Cardioprotection and Biomechanical Stability in vivo</strong></div> <div class="ql-block" data-block-id="block-2b7a9e32-b522-43b7-959e-c0ab1718ff98">Hansell Stedman, MD, University of Pennsylvania<br /> &nbsp;</div> </li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>15. Wearable Device Outcomes From EMBARK, a Pivotal Phase 3 Study in Patients With Duchenne Muscular Dystrophy Treated With Delandistrogene Moxeparvovec&nbsp;</strong><br /> Paul Strijbos, PhD, F. Hoffmann-La Roche Ltd&nbsp;<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>16. Turbo-Charging Gene Therapy for Genetic Muscle Disorders using Novel Transcriptional Cis-Regulatory Elements&nbsp;</strong><br /> Thierry Vandendriessche, PhD, Free University of Brussels (VUB)&nbsp;<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>18. Development of Pilot Potency Assessments for MyoDys<sup>45-55</sup>, a Gene Editing Therapy for Duchenne Muscular Dystrophy&nbsp;</strong><br /> Courtney Young, PhD, MyoGene Bio&nbsp;<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>19. (Virtual) A Transformative DMD Cytosine Base Editing Drug</strong><br /> Chunyan He, PhD, Suzhou GenAssist Thx<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>20. (Virtual) Drug Metabolism and Pharmacokinetics in Mice Systemically Administrated with a Base Editing Drug for Duchenne Muscular Dystrophin (DMD)</strong><br /> Chunyan He, PhD, Suzhou GenAssist Thx<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>21. Screening of Treatment-Emergent Anti-Dystrophin Antibodies: A Validated, Regulatory-Compliant LIPS Assay Covering 100% of Dystrophin</strong><br /> Eric Hoffman, PhD, Binghamton University<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>22. Development of a 248 Kilodalton Midlength-Dystrophin Gene Therapy for DMD using an AAV-Based RNA End-Joining Approach</strong><br /> Gretchen Thomsen, PhD, Insmed, Inc.<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>23. Optidys, a New Gene Therapy Product for Duchenne Muscular Dystrophy, Shows Therapeutic Potential and Advantage over Current Treatments</strong><br /> Evelyne Gicquel, PhD, Genethon<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>24. AUF1 Gene Therapy for DMD Increases Durable Endogenous Utrophin Expression, Muscle Regeneration, Muscle Function Performance and Synergizes With Microdystrophin Therapy</strong><br /> Dounia Abbadi, PhD, NYU School of Medicine<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>25. AUF1 Gene Therapy Restores Muscle Morphology and Function in Limb Girdle Muscular Dystrophy Recessive 1 (LGMDR1, calpainopathy)</strong><br /> Dounia Abbadi, PhD, NYU School of Medicine<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>26. An Engineered AAV Targeting Integrin Alpha V Beta 6 Presents Improved Myotropism Across Species</strong><br /> Anthony Brureau, PhD, GENETHON<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>27. Overcoming Phenotypic Variability in Dystrophic Models: A Machine Learning Method for Refined Gene Therapy Evaluation</strong><br /> Anthony Brureau, PhD, GENETHON<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>28. A Universal Approach to Potency Assay Development for AAVs Delivering Micro-RNAs</strong><br /> Jason McCoy, PhD, Nationwide Children&#39;s Hospital<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>29. Developing a Second Generation Proviral Plasmid to Reduce Aberrant Cross Packaging and ITR Promoter Activity in AAV Vector Preparations</strong><br /> Pranali Mistry, Nationwide Children&#39;s Hospital<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>30. CRISPR-Cas13d Gene Therapy for Facioscapulohumeral Muscular Dystrophy</strong><br /> Kate Neal, Nationwide Children&#39;s Hospital<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>31. Development of KT323 as a Potent and Safe Gene Therapy Candidate for Facioscapulohumeral Muscular Dystrophy (FSHD) Through High Throughput AAV Capsid and Cargo Engineering</strong><br /> Saurav Seshadri, PhD, Kate Therapeutics<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>32. Efficient Expression of Full-Length SMCHD1 in Muscles Using Split Inteins</strong><br /> Merlin Premalatha Thangaraj, PhD, Nationwide Children&#39;s Hospital<br /> &nbsp;</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"><strong>33. (Virtual) The Prevalence of Sleep Disorders among Individuals with Muscular Dystrophy: A Review of the Literature</strong><br /> Nedra Whitehead, PHD, CGC, RTI International</li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"> <p><strong>34. (Virtual) BIN1 Gene Replacement Reverts BIN1-Related Centronuclear Myopathy in a Preclinical Model</strong><br /> Jocelyn Laporte, PhD, CERBM IGBMC</p> </li> <li paraeid="{06108988-5e69-437a-bb17-80e68e0266dd}{189}" paraid="1809488388"> <p><strong>35. Functional Benefit of CRISPR/Cas9-Induced Deletion for RYR1 Dominant Mutation</strong><br /> Isabelle Marty, PhD, Inserm</p> </li> </ul> <p><strong>Poster Hall 2: Ontario and Erie Rooms</strong></p> <ul> <li><strong>36. Correcting Mutations in the DYSF Gene Using Prime Editing&nbsp;</strong><br /> Jacques Tremblay, PhD, Université Laval and CRCHUQ<br /> &nbsp;</li> <li><strong>37. Multiplex Base Editing in Primary Human Muscle Stem Cells to Address Compound Heterozygous Muscular Dystrophy</strong><br /> Supriya Krishna, MDC Berlin<br /> &nbsp;</li> <li><strong>38. Functional Efficacy of SRP-9004 in Young and Aged LGMD2D/R3 Mice</strong><br /> Luke Lemmerman, PhD, Sarepta Therapeutics, Inc.<br /> &nbsp;</li> <li><strong>39. Patient-derived stem cells for in vitro modeling of LAMA2-related Congenital Muscular Dystrophy</strong><br /> Jaquelyn Villalba, Cedars-Sinai Medical Center<br /> &nbsp;</li> <li><strong>40. AAV Delivery of ADAR Mediated RNA Editing for Treatment of Collagen VI Related Muscular Dystrophy</strong><br /> Russell Butterfield, MD, PhD, University of Utah<br /> &nbsp;</li> <li><strong>41. Etiology of a Mouse Model of CHKB mediated muscular dystrophy - A Platform for Gene Therapy for Disorders of Lipid Synthesis</strong><br /> Christopher McMaster, PhD, Dalhousie University<br /> &nbsp;</li> <li><strong>42. Treatment of a Severe DM1 Mouse Model with Verapamil, Amlodipine, and Ranolazine</strong><br /> Lily Cisco, University of Rochester<br /> &nbsp;</li> <li><strong>43. Splicing is improved using a novel AAV-microRNA delivery platform as a treatment for Myotonic Dystrophy Type 1</strong><br /> Martin Goulet, PhD, Sanofi<br /> &nbsp;</li> <li><strong>44. Enhancing Antisense Oligonucleotide Efficacy Using Small Molecule Oligonucleotide Activity Enhancers (OAEs)&nbsp;</strong><br /> Saeed Anwar, University of Alberta<br /> &nbsp;</li> <li><strong>45. Development of a New DUX4-Responsive Reporter Mouse</strong><br /> Jessica Camp, Nationwide Children&#39;s Hospital<br /> &nbsp;</li> <li><strong>46. Spatial Transcriptomic Characterization of an AAV-DUX4 Mouse Model of FSHD Identifies Focal and Widespread Cell Type Expression Changes in Muscle</strong><br /> Joel Chamberlain, PhD, University of Washington School of Medicine<br /> &nbsp;</li> <li><strong>47. Development and Characterization of a Minipig Model of Facioscapulohumeral Muscular Dystrophy (FSHD)</strong><br /> Chris Rogers, PhD, Exemplar Genetics<br /> &nbsp;</li> <li><strong>48. Human Stem Cell-derived Engineered Skeletal Muscle Tissues to Test Epigenetic Modulators and Deimmunized Gene Transfer Strategies for Duchenne Muscular Dystrophy&nbsp;</strong><br /> David Mack, PhD, University of Washington Medicine&nbsp;<br /> &nbsp;</li> <li><strong>49. Delandistrogene Moxeparvovec Promoter Activity in Human Cardiomyocytes and Cardiac Safety Assessment in Mice and Nonhuman Primates&nbsp;</strong><br /> Stephen Baine, PhD, Sarepta Therapeutics&nbsp;<br /> &nbsp;</li> <li><strong>50. Reduction of Dystrophin-Targeting microRNAs Increases Expression of Clinically Relevant Dystrophin Isoforms</strong><br /> Alyson Fiorillo, PhD, Virginia Commonwealth University<br /> &nbsp;</li> <li><strong>51. Template-Assisted Sequence Knock-In Rescues a Deletion Model of Duchenne Muscular Dystrophy</strong><br /> Sina Fatehi Someeh, The Hospital for Sick Children (SickKids)<br /> &nbsp;</li> <li><strong>52. Correction of DMD Exon 45 Deletion Using Homology-Independent Targeted Integration</strong><br /> Stefan Nicolau, MD, Nationwide Children&#39;s Hospital<br /> &nbsp;</li> <li><strong>53. Ultrasound-Mediated Delivery of Non-Viral Gene Therapy Payloads to Skeletal and Cardiac Muscles in Mice and Non-Human Primates</strong><br /> Ivan Krivega, PhD, SonoThera<br /> &nbsp;</li> <li><strong>54. Bioengineered Synthetic Chromosomes as Therapeutics for Muscular Dystrophies</strong><br /> Edward Perkins, PhD, CarryGenes Bioengineering<br /> &nbsp;</li> <li><strong>55. Exon 45 Skipping and Dystrophin Production with ENTR-601-45 in Preclinical Models of Duchenne Muscular Dystrophy</strong><br /> Amy Hicks, PhD, Entrada Therapeutics<br /> &nbsp;</li> <li><strong>56. A Synergistic Oligonucleotide Therapy to Improve Muscle Health and Dystrophin Restoration in Duchenne Muscular Dystrophy</strong><br /> James Novak, PhD, Children&#39;s National Hospital<br /> &nbsp;</li> <li><strong>57. A Novel IP-Mass Spectrometry Method to Characterize the Dystrophin Associated Protein Complex in Skeletal Muscle</strong><br /> Emily Canessa, Binghamton University<br /> &nbsp;</li> <li><strong>59.&nbsp;(Virtual) Cellular Mapping of DMD Transcript in Human Myogenic Cells and Skeletal Muscle by RNA in Situ Hybridization and&nbsp;Multiphoton Imaging</strong><br /> Alessandra Ferlini, MD, PhD, University of Ferrara<br /> &nbsp;</li> <li><a><strong>60</strong></a><strong>.&nbsp;Gene Transfer to Muscle Using HSV Vectors</strong><br /> Selene Ingusci, PhD, University of Pittsburgh<br /> &nbsp;</li> <li><strong>61. Correction of Nonsense Variants in DMD Using AAV-Compatible Base and Prime Editing</strong><br /> Ryan Marks, The Hospital for Sick Children<br /> &nbsp;</li> <li><strong>62. Extensor Digitorum Brevis Feasibility for Cell-Based Therapy for Duchenne Muscular Dystrophy</strong><br /> John Martone, University of Minnesota<br /> &nbsp;</li> <li> <div class="ql-block" data-block-id="block-d7eac4f0-a8f6-4acd-8f4f-bb38174a2987"><strong>63. (Virtual) Transcriptomics Reveals DMD Driven Cell Dynamics and Mechanisms of Myofiber Death and Innate Immune Memory in Human Dystrophic Muscle; Potential Barriers to Microdystrophin Gene Therapy</strong></div> <div class="ql-block" data-block-id="block-4049c0c8-ec5a-407a-8374-dbc78b20ab8a">Stanley Nelson, MD, UCLA<br /> &nbsp;</div> </li> <li><strong>64. Development of Functionalized Lipid Nanoparticles for Enhanced Micro-Dystrophin Delivery in Duchenne Muscular Dystrophy Therapy</strong><br /> Andrea Pirrottina, University of Rome</li> <li> <p><strong>65. A Novel Engineered Membrane Repair Protein Therapeutic Increases Sarcolemma Repair by Producing Phosphatidylserine Binding in Models of Duchenne Muscular Dystrophy</strong><br /> Noah Weisleder, PhD, University of Kentucky</p> </li> <li> <p><strong>66. Prime Editing Permits to Correct Point Mutations Responsible for Many Muscular Dystrophies</strong><br /> Jacques Tremblay, PhD, Université Laval and CRCHUQ</p> </li> <li> <p><strong>67. Combinatorial in vitro and in vivo Phage Display Methodology to Identify Skeletal Myocyte Targeting Cell Penetrating Peptides</strong><br /> Maliha Zahid, MD, PhD, Mayo Clinic</p> </li> </ul> <h3>Wenesday, Novemeber 20</h3> <p><strong>Attendee Breakfast</strong><br /> 7:30 - 8:30 a.m.</p> <p><strong>RNA Targeted Therapies</strong><br /> 8:30&nbsp;- 10:15 a.m.<br /> <strong>Chair:&nbsp;</strong>Scott Harper, Ohio State University &amp; Nationwide Children&#39;s Hospital</p> <ul> <li><strong>Enhancing the Delivery of Antisense Oligonucleotide Therapies: Innovative Approaches for Muscular Dystrophy</strong><br /> Toshifumi Yokota, PhD, University of Alberta, Edmonton, AB</li> <li><strong>Translating RNAi-based Gene Therapies for Dominant Neuromuscular Disorders</strong><br /> Lindsay Wallace, PhD, Nationwide Children&#39;s Research Institute, Columbus, OH</li> <li><strong>RNA Therapies to Investigate the Impact of Dystrophin Restoration in the CNS of Dystrophic Mouse Models</strong><br /> Aurelie Goyenvalle, PhD, University of Versailles Saint Quentin En Yvelines</li> <li><strong>Panel Discussion</strong></li> </ul> <p><strong>Coffee Break and Paid Presentation</strong><br /> 10:15 - 10:45 a.m.</p> <p><img alt="" src="/getmedia/4eb016dc-4075-444b-8742-f70a4bb0f44e/catalyst.aspx?width=200&amp;height=100" style="width: 200px; height: 100px;" /></p> <p><strong>Panel: Future of Genetic Therapies for Muscular Dystrophies</strong><br /> 10:45 a.m.&nbsp;- 12 p.m.<br /> <strong>Chair:&nbsp;</strong>Sharon Hesterlee, Chief Research Officer, Muscular Dystrophy Association</p> <ul> <li><strong>Panelists</strong><br /> Jeffrey Chamberlain, PhD, University of Washington<br /> Carsten Bönnemann, MD, NINDS/NIH<br /> Katherine High, MD, Rockefeller University<br /> Barry Byrne, MD, PhD, University of Florida<br /> Melissa Spencer, PhD, UCLA</li> </ul> <h2>Ready to Register?</h2> <p><a class="btn btn-accent3" href="https://www.xcdsystem.com/asgct/attendee/index.cfm?ID=4Pa6Ppp" target="_blank">Register now!</a></p> <h2>Thank You to the Organizing Committee</h2> <ul> <li><strong>Jeffrey Chamberlain, PhD</strong><br /> University of Washington</li> <li><strong>Angela Lek, PhD</strong><br /> Muscular Dystrophy Association</li> <li><strong>Evrim Atas, PhD</strong><br /> Muscular Dystrophy Association</li> <li><strong>Charlie Gersbach, PhD</strong><br /> Duke University</li> <li><strong>Toshifumi Yokota, PhD</strong><br /> University of Alberta</li> <li><strong>Manuela Corti, PhD</strong><br /> University of Florida</li> <li><strong>Monkol Lek, PhD</strong><br /> Yale University</li> <li><strong>Julie Crudele, PhD</strong><br /> University of Washington</li> <li><strong>Melissa Spencer, PhD</strong><br /> UCLA</li> <li><strong>Carsten Bönnemann, MD</strong><br /> NIH</li> <li><strong>Scott Harper, PhD</strong><br /> The Ohio State University/Nationwide Children&#39;s Hospital</li> </ul> </main> <aside> <div class="asideBox"> <h4>Related Pages</h4> <ul> <li><a href="/events/breakthroughs-in-muscular-dystrophy/registration">Registration</a> </li> <li><a href="/events/breakthroughs-in-muscular-dystrophy/abstracts">Abstracts</a> </li> <li class="active"><a href="/events/breakthroughs-in-muscular-dystrophy/program">Program</a> </li class="active"> <li><a href="/events/breakthroughs-in-muscular-dystrophy/speaker-and-attendee-resources">Speaker + Attendee Resources</a> </li> <li><a href="/events/breakthroughs-in-muscular-dystrophy/sponsorship">Sponsorship</a> </li> </ul> </div> </aside> </div> </section> <div class="event-row"> <div class="year">2024</div> <div class="content"> <h2>Breakthroughs in Muscular Dystrophy</h2> <p>November 19-20, 2024 | Chicago, IL</p> </div> <div class="action"><a class="btn btn-white" href="/events/breakthroughs-in-muscular-dystrophy">Learn More</a></div> </div> <footer> <div class="container"> <div class="footerColContainer"> <div class="footerCol"> <p><img alt="logo-footer.png" class="footerLogo" src="/getmedia/c77b7df1-12a2-4998-9809-0a880c107341/ASGCT_Web_Horz_White.aspx" title="logo-footer.png" /></p> <p>20800 Swenson Dr.<br /> Suite 300<br /> Waukesha, WI 53186</p> </div> <div class="footerCol"> <ul> <li><a href="/about">About</a></li> <li><a href="/membership">Membership</a></li> <li><a href="https://annualmeeting.asgct.org" target="_blank">Annual Meeting</a></li> <li><a href="http://www.cell.com/molecular-therapy-family/home" target="_blank">Journal</a></li> </ul> </div> <div class="footerCol"> <ul> <li><a href="/education">Education</a></li> <li><a href="/advocacy">Advocacy</a></li> <li><a href="https://asgct.org/awards" target="_blank">Awards</a></li> <li><a href="https://asgct.org/about/privacy-policy" target="_blank">Privacy Policy</a></li> </ul> </div> <div class="footerCol"> <p class="altStyle">Connect with ASGCT</p> <p><strong>Phone:</strong> 414.278.1341<br /> <a href="mailto:info@asgct.org">info@asgct.org</a><div class="footerSocial"><div id="ctl00_bkSiteFooter_rptFooterBlocks_ctl04_widget1_ctl00_divSocialLinks" class="socialLinks footerSocial"> <ul> <li><a href="https://twitter.com/ASGCTherapy" target="_blank"><i class="bk-icon-twitter"></i></a></li> <li><a href="https://www.facebook.com/asgctherapy" target="_blank"><i class="bk-icon-facebook"></i></a></li> <li><a href="https://www.instagram.com/asgctherapy" target="_blank"><i class="bk-icon-instagram"></i></a></li> <li><a href="https://www.youtube.com/user/ASGCT" target="_blank"><i class="bk-icon-youtube"></i></a></li> <li><a href="https://www.linkedin.com/company/asgct/" target="_blank"><i class="bk-icon-linkedin"></i></a></li> </ul> </div></div></p> </div> </div> </div> </footer> <div class="postFooter"> <div class="container"> <p>&copy; 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