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Challenges for gene therapy for muscular dystrophy

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Abstract

Gene therapy for muscular dystrophy represents a promising avenue of pursuit for a disease with a limited repertoire of treatment. Recent successes in the research arena using adeno-associated viral vectors should accelerate the movement of gene-based therapeutics for muscle disorders into the clinic. Nevertheless, significant challenges remain before gene therapy can deliver on the promises avowed by early pioneers of the field. This review examines recent progress and the hurdles remaining to achieve gene-based treatment therapies for muscular dystrophy.

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Authors and Affiliations

  1. Center for Gene Therapy, Columbus Children’s Research Institute, 700 Children’s Drive, 43205, Columbus, OH, USA

    Jerry R. Mendell MD

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  1. Jerry R. Mendell MD
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  2. K. Reed Clark PhD
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Correspondence to Jerry R. Mendell MD.

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Mendell, J.R., Clark, K.R. Challenges for gene therapy for muscular dystrophy. Curr Neurol Neurosci Rep 6, 47–56 (2006). https://doi.org/10.1007/s11910-996-0009-8

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  • DOI: https://doi.org/10.1007/s11910-996-0009-8

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