BioengineeringbioRxiv
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…·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.
DOI 10.1016/j.ymthe.2021.03.023