Ontology highlight
ABSTRACT:
SUBMITTER: Long C
PROVIDER: S-EPMC4398027 | biostudies-literature | 2014 Sep
REPOSITORIES: biostudies-literature
Long Chengzu C McAnally John R JR Shelton John M JM Mireault Alex A AA Bassel-Duby Rhonda R Olson Eric N EN
Science (New York, N.Y.) 20140814 6201
Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. DMD is characterized by progressive muscle weakness and a shortened life span, and there is no effective treatment. We used clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9)-mediated genome editing to correct the dystrophin gene (Dmd) mutation in the germ line of mdx mice, a model for DMD, and then monitor ...[more]