Ontology highlight
ABSTRACT:
SUBMITTER: Chicoine LG
PROVIDER: S-EPMC3916040 | biostudies-literature | 2014 Feb
REPOSITORIES: biostudies-literature
Chicoine L G LG Montgomery C L CL Bremer W G WG Shontz K M KM Griffin D A DA Heller K N KN Lewis S S Malik V V Grose W E WE Shilling C J CJ Campbell K J KJ Preston T J TJ Coley B D BD Martin P T PT Walker C M CM Clark K R KR Sahenk Z Z Mendell J R JR Rodino-Klapac L R LR
Molecular therapy : the journal of the American Society of Gene Therapy 20131023 2
Duchenne muscular dystrophy is a monogenic disease potentially treatable by gene replacement. Use of recombinant adeno-associated virus (AAV) will ultimately require a vascular approach to broadly transduce muscle cells. We tested the impact of preexisting AAV antibodies on microdystrophin expression following vascular delivery to nonhuman primates. Rhesus macaques were treated by isolated limb perfusion using a fluoroscopically guided catheter. In addition to serostatus stratification, the anim ...[more]