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Am J Phys Med Rehabil. 2014 Nov;93(11 Suppl 3):S97-107. doi: 10.1097/PHM.0000000000000138.

Gene therapy for inherited muscle diseases: where genetics meets rehabilitation medicine.

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From the Department of Rehabilitation Medicine and Institute for Stem Cell and Regenerative Medicine, University of Washington (RB, DLM, MKC); and Fred Hutchinson Cancer Research Center (ZW), Seattle, Washington.


The development of clinical vectors to correct genetic mutations that cause inherited myopathies and related disorders of skeletal muscle is advancing at an impressive rate. Adeno-associated virus vectors are attractive for clinical use because (1) adeno-associated viruses do not cause human disease and (2) these vectors are able to persist for years. New vectors are now becoming available as gene therapy delivery tools, and recent preclinical experiments have demonstrated the feasibility, safety, and efficacy of gene therapy with adeno-associated virus for long-term correction of muscle pathology and weakness in myotubularin-deficient canine and murine disease models. In this review, recent advances in the application of gene therapies to treat inherited muscle disorders are presented, including Duchenne muscular dystrophy and x-linked myotubular myopathy. Potential areas for therapeutic synergies between rehabilitation medicine and genetics are also discussed.

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