Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus
S. Vaidyanathan, R. Baik, L. Chen, D. T. Bravo, C. J. Suarez, S. M. Abazari, A. A. Salahudeen, A. M. Dudek, C. A. Teran, T. H. Davis, C. M. Lee, G. Bao, S. H. Randell, S. E. Artandi, J. J. Wine, , M. Porteus
This study corrects multiple CFTR mutations in human airway stem cells using CRISPR/Cas9 and AAV vectors, achieving functional restoration in differentiated epithelial cells.
bioRxiv · Feb 26, 2021Bioengineering