Ontology highlight
ABSTRACT:
SUBMITTER: Iyer PS
PROVIDER: S-EPMC6079556 | biostudies-literature | 2018 Apr
REPOSITORIES: biostudies-literature
Iyer Pavithra S PS Mavoungou Lionel O LO Ronzoni Flavio F Zemla Joanna J Schmid-Siegert Emanuel E Antonini Stefania S Neff Laurence A LA Dorchies Olivier M OM Jaconi Marisa M Lekka Malgorzata M Messina Graziella G Mermod Nicolas N
Molecular therapy : the journal of the American Society of Gene Therapy 20180202 4
Duchenne muscular dystrophy (DMD) is a lethal muscle-wasting disease currently without cure. We investigated the use of the PiggyBac transposon for full-length dystrophin expression in murine mesoangioblast (MABs) progenitor cells. DMD murine MABs were transfected with transposable expression vectors for full-length dystrophin and transplanted intramuscularly or intra-arterially into mdx/SCID mice. Intra-arterial delivery indicated that the MABs could migrate to regenerating muscles to mediate d ...[more]