Title: | Structure-Based Designed Nano-Dysferlin Significantly Improves Dysferlinopathy in BLA/J Mice |
Journal : | Molecular therapy : the journal of the American Society of Gene Therapy, 25, 9 |
Authors: | Llanga T, Author ; Nagy N ; Conatser L ; Dial C ; Sutton RB ; Hirsch ML |
Material Type: | Article |
Publication Date: | 2017 |
Size: | p 2150 |
Languages: | English |
Keywords : | adenoassociated vectors ; cell cultures ; DYSF gene ; dysferlin ; dysferlinopathy ; gene therapy ; immunofluorescence ; intramuscular injection ; motor skills ; mouse model ; muscle histopathology ; myoblast ; preclinical trials ; protein structure ; skeletal muscle |
Keywords: | AAV;adeno-associated virus;dysferlin;dysferlinopathy;gene therapy;muscular dystrophy;nanodysferlin; |
Pubmed / DOI : | DOI : 10.1016/j.ymthe.2017.05.013 / Pubmed : 28629822 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/28629822 |
See also : |