Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
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Publisher
United States: Elsevier Inc
Journal title
Language
English
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Publisher
United States: Elsevier Inc
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Scope and Contents
Contents
Efficient and widespread gene transfer is required for successful treatment of Duchenne muscular dystrophy (DMD). Here, we performed the first clinical trial using a chimeric adeno-associated virus (AAV) capsid variant (designated AAV2.5) derived from a rational design strategy. AAV2.5 was generated from the AAV2 capsid with five mutations from AAV...
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Full title
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3277234
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_3277234
Other Identifiers
ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1038/mt.2011.237
