Title: | Muscle and Heart Function Restoration in a Limb Girdle Muscular Dystrophy 2I (LGMD2I) Mouse Model by Systemic FKRP Gene Delivery |
Journal : | Molecular therapy : the journal of the American Society of Gene Therapy, 22, 11 |
Authors: | Qiao C ; Wang CH ; Zhao C ; Lu P ; Awano H ; Xiao B ; Li J ; Yuan Z ; Dai Y ; Martin CB ; Li J ; Lu Q ; Xiao X |
Material Type: | Article |
Publication Date: | 2014 |
Size: | p 1890 |
Languages: | English |
Keywords : | AAV9 vectors ; cardiomyopathy ; cross-sectional study ; fibrosis ; FKRP gene ; FKRP protein ; FKRP-related limb-girdle muscular dystrophy R9 ; gene therapy ; heart muscle ; mouse model ; muscle fiber degeneration ; skeletal muscle |
Pubmed / DOI : | DOI : 10.1038/mt.2014.141 / Pubmed : 25048216 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/25048216 |