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Curr Opin Neurol. 2015 Oct;28(5):522-7. doi: 10.1097/WCO.0000000000000241.

Viral vector-mediated gene therapies.

Author information

1
Department of Neurology, Wellstone Muscular Dystrophy Research Center, University of Washington School of Medicine, Seattle, Washington, USA.

Abstract

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 transduction efficiency. Full-length dystrophin expression via gene editing with targeted nucleases may ultimately be an ideal treatment option. Also genes with homologues function may ameliorate many aspects of the DMD pathophysiology.

SUMMARY:

The work during the past year has increased our understanding of AAV vector-mediated therapy and has also validated new approaches to treat DMD. The results will aid in the design of both preclinical and clinical trials.

PMID:
26263476
PMCID:
PMC4608371
DOI:
10.1097/WCO.0000000000000241
[Indexed for MEDLINE]
Free PMC Article

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