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Article

Viral vector-mediated gene therapies

15 Aug 2015

Abstract excerpt

PURPOSE OF REVIEW: Gene therapy as a treatment for neuromuscular disease has significantly advanced over the past decade. In the present review, the progress of adeno-associated viruses (AAV) vector-mediated gene therapy for Duchenne muscular dystrophy (DMD) during the past year is highlighted. RECENT FINDINGS: Modulating the immune response to AAV vector capsid or the transgene has helped to increase stable...

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PMID: 26263476
DOI: 10.1097/wco.0000000000000241

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