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Paper Details

Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus.
Mol Ther
25
2022
CF, CF gene, CF transmembrane conductance regulator, CFTR, CFTR cDNA, CFTR locus, Cas9, Cystic fibrosis, Differentiated epithelial monolayers, HBECs, UABCs, cDNA, endogenous locus, epithelial monolayers, full-length, human, human airway stem cells, human bronchial epithelial cells, monogenic disease, patients, stem cell, tCD19, tCD19+, tCD19+ UABCs, truncated CD19, upper airway basal stem cells
Author NameAffiliation
Scott H RandellThe University of North Carolina at Chapel Hill
Jayakar V Nayak
Matthew H PorteusStanford University
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Release 1.2