Title: | Adeno-associated virus 9 mediated FKRP gene therapy restores functional glycosylation of alpha--dystroglycan and improves muscle functions |
Journal : | Molecular therapy, 21, 10 |
Authors: | Xu L ; Lu PJ ; Wang CH ; Keramaris E ; Qiao C ; Xiao B ; Blake DJ ; Xiao X ; Lu QL |
Material Type: | Article |
Publication Date: | 2013 |
Size: | p. 1832-1840 |
Languages: | English |
Keywords : | AAV9 vectors ; alpha-dystroglycan ; FKRP gene ; gene therapy ; glycosylation ; Golgi apparatus ; heart muscle ; mouse model ; muscle fibers ; muscle function ; skeletal muscle ; toxicity |
Abstract: |
Accès au résumé Pubmed/to pubmed abstract |
Pubmed / DOI : | DOI : 10.1038/mt.2013.156 / Pubmed : 23817215 |