Title: | Efficacy of Gene Therapy Is Dependent on Disease Progression in Dystrophic Mice with Mutations in the FKRP Gene |
Journal : | Molecular therapy. Methods & clinical development, 5 |
Authors: | Vannoy CH, Author ; Xiao W ; Lu P ; Xiao X ; Lu QL |
Material Type: | Article |
Publication Date: | 2017 |
Size: | p 31 |
Languages: | English |
Keywords : | AAV9 vectors ; alpha-dystroglycanopathy ; FKRP gene ; FKRP-related limb-girdle muscular dystrophy R9 ; gene therapy ; glycosylation ; mouse model ; muscle histopathology ; skeletal muscle |
Keywords: | adeno-associated virus;dystroglycanopathy;fukutin-related protein;gene therapy;muscular dystrophy; |
Pubmed / DOI : | DOI : 10.1016/j.omtm.2017.02.002 / Pubmed : 28480302 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28480302 |