Ontology highlight
ABSTRACT:
SUBMITTER: Kornegay JN
PROVIDER: S-EPMC2927072 | biostudies-literature | 2010 Aug
REPOSITORIES: biostudies-literature
Kornegay Joe N JN Li Juan J Bogan Janet R JR Bogan Daniel J DJ Chen Chunlian C Zheng Hui H Wang Bing B Qiao Chunping C Howard James F JF Xiao Xiao X
Molecular therapy : the journal of the American Society of Gene Therapy 20100601 8
Duchenne (DMD) and golden retriever (GRMD) muscular dystrophy are caused by genetic mutations in the dystrophin gene and afflict striated muscles. We investigated systemic gene delivery in 4-day-old GRMD dogs given a single intravenous injection of an AAV9 vector (1.5 x 10(14) vector genomes/kg) carrying a human codon-optimized human mini-dystrophin gene under control of the cytomegalovirus (CMV) promoter. One of the three treated dogs was euthanized 9 days later due to pre-existing conditions. ...[more]