...published concurrently in Science have shown CRISPR can treat a genetic disease in a postnatal mouse model...another approach. Since shorter forms of dystrophin can still be functional, exon skipping is a good option...culture, but Ran et al. have shown that CRISPR and AAV can be used together for postnatal genome editing in ...strategies for Duchenne muscular dystrophy. gRNAs can be designed to flank a single or multiple exons that...’d like to treat with CRISPR. As seen above, DMD can be treated with “one-size-fits-most” NHEJ-mediated...DMD. If the safety and efficacy of this approach can be optimized, DMD could become one of the first diseases...science.aae0169
2. Long C, Amoasii L, Mireault AA, McAnally JR, Li H, Sanchez-Ortiz E, Bhattacharyya S, Shelton...