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Gene Therapy for Inherited Muscle Diseases.
- Source :
-
American Journal of Physical Medicine & Rehabilitation . Nov2014 Supplement, Vol. 93, pS97-S107. 11p. 1 Black and White Photograph, 3 Diagrams, 2 Charts. - Publication Year :
- 2014
-
Abstract
- 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. [ABSTRACT FROM AUTHOR]
- Subjects :
- *GENE therapy
*MUSCLE diseases
*MUSCULAR dystrophy
*REHABILITATION
Subjects
Details
- Language :
- English
- ISSN :
- 08949115
- Volume :
- 93
- Database :
- Academic Search Index
- Journal :
- American Journal of Physical Medicine & Rehabilitation
- Publication Type :
- Academic Journal
- Accession number :
- 99098759
- Full Text :
- https://doi.org/10.1097/PHM.0000000000000138