Ontology highlight
ABSTRACT:
SUBMITTER: Long C
PROVIDER: S-EPMC4760628 | biostudies-literature | 2016 Jan
REPOSITORIES: biostudies-literature
Long Chengzu C Amoasii Leonela L Mireault Alex A AA McAnally John R JR Li Hui H Sanchez-Ortiz Efrain E Bhattacharyya Samadrita S Shelton John M JM Bassel-Duby Rhonda R Olson Eric N EN
Science (New York, N.Y.) 20151231 6271
CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. To correct DMD by skipping mutant dystrophin exons in postnatal muscle tissue in vivo, we used adeno-associated virus-9 (AAV9) to deliver gene-editing components to postnatal mdx mice, a model of DMD. Different modes of AAV9 delivery were systematically tested, including intraperitoneal at postnatal day 1 (P1), ...[more]