Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

The overall goal of this in vivo cardiac gene editing protocol is to restore dystrophin expression in the heart of dystrophic mice using recombinant adeno-associated virus rh. 74 with CRISPR SaCas9 and guide RNA vectors. This method can help answer key questions in the muscular dystrophy field, such as whether gene editing is a reliable therapeutic strategy to treat the root cause of the cardiomyopathy associated with Duchenne's muscular dystrophy.

The main advantage of these techniques are the expression of the corrected dystrophin gene is under its endogenous regulatory control, and this rescue effect is permanent. The implication of this technique extends toward treatment of other genetic disease where correction of mutant gene can be achieved by CRISPR Cas9 gene editing technology. This method not only provides insight into dystrophin restoration in the dystrophic heart, but it can also be used to knock out the gene of interest for reverse genetic studies.