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Gene therapy for inherited muscle diseases: Where genetics meets rehabilitation medicine

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 (AAV) vectors are attractive for clinical use because (i) AAVs do not cause human disease, and (ii) the...

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Autors principals: Braun, Robynne, Wang, Zejing, Mack, David L., Childers, Martin K.
Format: Artigo
Idioma:Inglês
Publicat: 2014
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4197407/
https://ncbi.nlm.nih.gov/pubmed/25313664
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1097/PHM.0000000000000138
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