Chengzu Long, Leonela Amoasii, Alex A Mireault, John R McAnally, Hui Li, Efrain Sanchez-Ortiz, Samadrita Bhattacharyya, John M Shelton, Rhonda Bassel-Duby, Eric N Olson
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), intramuscular at P12, and retro-orbital at P18...
January 22, 2016: Science