Therapeutic base and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa
S.-a. Hong, S.-E. Kim, A.-y. Lee, G.-h. Hwang, J. H. Kim, H. Iwata, S.-C. Kim, S. Bae, S. E. Lee
This study corrects COL7A1 mutations in RDEB patient fibroblasts using base and prime editing, restoring C7 expression and function in vitro and in vivo.
bioRxiv · Jul 12, 2021Bioengineering