Meeting Report : 2022 Muscular Dystrophy Association Summit on 'Safety and Challenges in Gene Transfer Therapy'
Muscular Dystrophy Association (MDA) has invested over $125M in the development of gene therapy for neuromuscular disorders (NMD) over the past 20 years. As a lead initiator of progress in this important field of medicine and to help ensure continued progress towards therapies for patients, MDA organized a dedicated summit in January 2022 to address emerging challenges in safely delivering adeno-associated virus (AAV) mediated gene therapies with a focus on their application in NMD. In this meeting, chaired by Carsten Bönnemann (NINDS, NIH) and Barry Byrne (University of Florida), academic and industry experts and stakeholders convened to openly discuss adverse events linked to clinical trials, as well as other challenges emerging in preclinical studies associated with difficulties in the translation of AAV-mediated gene therapies.
| Medienart: |
E-Artikel |
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| Erscheinungsjahr: |
2023 |
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| Erschienen: |
2023 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:10 |
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| Enthalten in: |
Journal of neuromuscular diseases - 10(2023), 3 vom: 07., Seite 327-336 |
| Sprache: |
Englisch |
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| Beteiligte Personen: |
Lek, Angela [VerfasserIn] |
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| Links: |
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| Themen: |
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| Anmerkungen: |
Date Completed 09.05.2023 Date Revised 09.05.2023 published: Print Citation Status MEDLINE |
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| doi: |
10.3233/JND-221639 |
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| funding: |
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| Förderinstitution / Projekttitel: |
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| 520 | |a Muscular Dystrophy Association (MDA) has invested over $125M in the development of gene therapy for neuromuscular disorders (NMD) over the past 20 years. As a lead initiator of progress in this important field of medicine and to help ensure continued progress towards therapies for patients, MDA organized a dedicated summit in January 2022 to address emerging challenges in safely delivering adeno-associated virus (AAV) mediated gene therapies with a focus on their application in NMD. In this meeting, chaired by Carsten Bönnemann (NINDS, NIH) and Barry Byrne (University of Florida), academic and industry experts and stakeholders convened to openly discuss adverse events linked to clinical trials, as well as other challenges emerging in preclinical studies associated with difficulties in the translation of AAV-mediated gene therapies | ||
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| 700 | 1 | |a Bönnemann, Carsten G |e verfasserin |4 aut | |
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