title: Duchenne muscular dystrophy: CRISPR/Cas9 treatment.
abstract: A novel approach to gene correction by genome editing shows great promise as a treatment for Duchenne muscular dystrophy (DMD). CRISPR/Cas9 delivered by adeno-associated virus to a mouse model for DMD demonstrated improvement in function and histology.
authors.affiliation: Department of Pediatrics, Columbus, OH 43205, USA.|Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.|The Ohio State University, Columbus, OH 43205, USA.
authors.forename: Jerry R
authors.initials: JR
authors.lastname: Mendell
authors.affiliation: Department of Pediatrics, Columbus, OH 43205, USA.|Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.|The Ohio State University, Columbus, OH 43205, USA.
authors.forename: Louise R
authors.initials: LR
authors.lastname: Rodino-Klapac
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems; D016189:Dystrophin / Q000235:genetics; D015316:Genetic Therapy; D006801:Humans; D007231:Infant, Newborn; D051379:Mice; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics*; D015997:Neonatal Screening
chemical_list: D016189:Dystrophin