This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies.
Keywords: AAV; Alpha1 Antitrypsin deficiency; CRISPR; Clinical trials; DMD; DNA damage; HDR; Myotonic Dystrophy; NHEJ; nanoparticles.