Pavithra S Iyer, Lionel O Mavoungou, Flavio Ronzoni, Joanna Zemla, Emanuel Schmid-Siegert, Stefania Antonini, Laurence A Neff, Olivier M Dorchies, Marisa Jaconi, Malgorzata Lekka, Graziella Messina, Nicolas Mermod
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 dystrophin expression...
February 2, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy