Ontology highlight
ABSTRACT:
SUBMITTER: Hayashita-Kinoh H
PROVIDER: S-EPMC4395797 | biostudies-other | 2015 Apr
REPOSITORIES: biostudies-other
Hayashita-Kinoh Hiromi H Yugeta Naoko N Okada Hironori H Nitahara-Kasahara Yuko Y Chiyo Tomoko T Okada Takashi T Takeda Shin'ichi S
Molecular therapy : the journal of the American Society of Gene Therapy 20150114 4
Duchenne muscular dystrophy (DMD) is a severe congenital disease due to mutations in the dystrophin gene. Supplementation of dystrophin using recombinant adenoassociated virus vector has promise as a treatment of DMD, although therapeutic benefit of the truncated dystrophin still remains to be elucidated. Besides, host immune responses against the vector as well as transgene products have been denoted in the clinical gene therapy studies. Here, we transduced dystrophic dogs fetuses to investigat ...[more]