[Report] In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

In this study, adeno-associated virus was used to deliver the clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9 system to the mdx mouse model of DMD to remove the mutated exon 23 from the dystrophin gene. This includes local and systemic delivery to adult mice and systemic delivery to neonatal mice. Exon 23 deletion by CRISPR-Cas9 resulted in expression of the modified dystrophin gene, partial recovery of functional dystrophin protein in skeletal myofibers and cardiac muscle, improvement of muscle biochemistry, and significant enhancement of muscle force. This work establishes CRISPR-Cas9–based genome editing as a potential therapy to treat DMD. Authors: Christopher E. Nelson, Chady H. Hakim, David G. Ousterout, Pratiksha I. Thakore, Eirik A. Moreb, Ruth M. Castellanos Rivera, Sarina Madhavan, Xiufang Pan, F. Ann Ran, Winston X. Yan, Aravind Asokan, Feng Zhang, Dongsheng Duan, Charles A. Gersbach
Source: ScienceNOW - Category: Science Authors: Source Type: news