Article
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.
Molecular therapy : the journal of the American Society of Gene Therapy - 5 Jan 2022
Vaidyanathan Sriram, Baik Ron, Chen Lu, Bravo Dawn T, Suarez Carlos J, Abazari Shayda M, Salahudeen Ameen A, Dudek Amanda M, Teran Christopher A, Davis Timothy H, Lee Ciaran M, Bao Gang, Randell Scott H, Artandi Steven E, Wine Jeffrey J, Kuo Calvin J, Desai Tushar J, Nayak Jayakar V, Sellers Zachary M, Porteus Matthew H
Abstract excerpt
Cystic fibrosis (CF) is a monogenic disease caused by impaired production and/or function of the CF transmembrane conductance regulator (CFTR) protein. Although we have previously shown correction of the most common pathogenic mutation, there are many other pathogenic mutations throughout the CF gene. An autologous airway stem cell therapy in which the CFTR cDNA is precisely inserted into the CFTR locus may...
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