Ontology highlight
ABSTRACT:
SUBMITTER: Amoasii L
PROVIDER: S-EPMC6205228 | biostudies-other | 2018 Oct
REPOSITORIES: biostudies-other
Science (New York, N.Y.) 20180830 6410
Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD harbors a mutation corresponding to a mutational "hotspot" in the human <i>DMD</i> gene. We used adeno-associated viruses to deliver CRISPR gene editing components to four dogs and examined dystrophin protein expression 6 weeks after intramuscular delivery (<i>n</i> = 2) or 8 weeks after systemic delivery (<i>n</i> = 2). Afte ...[more]