Key result
Gene therapy using adeno-associated virus vectors demonstrates feasibility, safety, and efficacy in preclinical models for long-term correction of inherited muscle disorders.
Why the study?
Does gene therapy with adeno-associated virus vectors improve muscle pathology and weakness in inherited muscle diseases?
Does gene therapy with adeno-associated virus vectors improve muscle pathology and weakness in inherited muscle diseases?
Gene therapy using adeno-associated virus vectors shows preclinical feasibility, safety, and efficacy for treating inherited muscle disorders like Duchenne muscular dystrophy.
Should not yet change practice; hypothesis-generating for AAV gene therapy in inherited muscle disorders.
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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Braun et al. (2014) conducted a review in Inherited muscle diseases. Gene therapy (Adeno-associated virus vectors) was evaluated. Gene therapy using adeno-associated virus vectors demonstrates feasibility, safety, and efficacy in preclinical models for long-term correction of inherited muscle disorders.
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