Title: | Gene therapy in monogenic congenital myopathies |
Journal : | Methods, 99 |
Authors: | Guan X, Author ; Goddard MA ; Mack DL ; Childers MK |
Material Type: | Article |
Publication Date: | 2016 |
Size: | p 91 |
Languages: | English |
Keywords : | AAV8 vectors ; adenoassociated vectors ; alpha-sarcoglycan-related limb-girdle muscular dystrophy R3 ; cellular models ; clinical trials ; Duchenne muscular dystrophy ; exon skipping ; facioscapulohumeral muscular dystrophy ; gamma-sarcoglycan-related limb-girdle muscular dystrophy R5 ; gene therapy ; injection ; interfering RNA ; model dog ; mouse model ; MTM1 gene ; myotonic dystrophy type 1 ; myotonic dystrophy type 2 ; perfusion ; preclinical trials ; review article ; X-linked myotubular myopathy |
Keywords: | Animal models;Gene therapy;Muscle disease;Muscular dystrophy;Myopathy; |
Pubmed / DOI : | DOI : 10.1016/j.ymeth.2015.10.004 / Pubmed : 26454198 |