AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice

Hakim CH, Wasala NB, Nelson CE, et al. AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice. JCI insight 2018;3   Abstract Adeno-associated virus-mediated (AAV-mediated) CRISPR editing is a revolutionary approach for treating inherited diseases. Sustained, often life-long mutation correction is required for treating these diseases. Unfortunately, this has never […]