Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.


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… (More)
DOI: 10.1126/science.aad5725


Blog articles referencing this paper