In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.

ABSTRACT:

Rationale

Duchenne muscular dystrophy is a severe inherited form of muscular dystrophy caused by mutations in the reading frame of the dystrophin gene disrupting its protein expression. Dystrophic cardiomyopathy is a leading cause of death in Duchenne muscular dystrophy patients, and currently no effective treatment exists to halt its progression. Recent advancement in genome editing technologies offers a promising therapeutic approach in restoring dystrophin protein expression. However, the impact of this approach on Duchenne muscular dystrophy cardiac function has yet to be evaluated. Therefore, we assessed the therapeutic efficacy of CRISPR (clustered regularly interspaced short palindromic repeats)-mediated genome editing on dystrophin expression and cardiac function in mdx/

SUBMITTER: El Refaey M 

PROVIDER: S-EPMC5623072 | biostudies-literature | 2017 Sep

REPOSITORIES: biostudies-literature