Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

Nelson CE, Wu Y, Gemberling MP, et al. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy. Nature medicine 2019;25:427-432   Abstract Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for therapeutic genome editing. There have been several recent reports of genome editing in preclinical models of Duchenne muscular dystrophy(1-6), however, […]