Highly Efficient Repair of the ΔF508 Mutation in Airway Stem Cells of Cystic Fibrosis Patients with Functional Rescue of the Differentiated Epithelia
S. Vaidyanathan, A. A. Salahudeen, Z. M. Sellers, D. T. Bravo, S. S. Choi, A. Batish, W. Le, S. De La O, M. P. Kaushik, N. Galper, C. M. Lee, G. Bao, E. H. Chang, J. J. Wine, C. E. Milla, , M. H. Porteus
This paper corrects the ΔF508 mutation in CF patient airway stem cells using CRISPR-Cas9 and AAV6, achieving high correction rates and functional rescue in differentiated epithelia.
bioRxiv · Feb 26, 2019 · PreprintBioengineering