Adeno-associated viral (AAV) vectors do not efficiently target muscle satellite cells.

ABSTRACT: Adeno-associated viral (AAV) vectors are becoming an important tool for gene therapy of numerous genetic and other disorders. Several recombinant AAV vectors (rAAV) have the ability to transduce striated muscles in a variety of animals following intramuscular and intravascular administration, and have attracted widespread interest for therapy of muscle disorders such as the muscular dystrophies. However, most studies have focused on the ability to transduce mature muscle cells, and have not examined the ability to target myogenic stem cells such as skeletal muscle satellite cells. Here we examined the relative ability of rAAV vectors derived from AAV6 to target myoblasts, myocytes and myotubes in culture and satellite cells and myofibers in vivo. AAV vectors are able to transduce prolifera

SUBMITTER: Arnett AL 

PROVIDER: S-EPMC4288464 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature