Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells

Canonical splice site variants affecting the 5′ GT and 3′ AG nucleotides of introns result in severe missplicing and account for about 10% of disease-causing genomic alterations. Treatment of such variants has proven challenging due to the unstable mRNA or protein isoforms that typically result from...

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Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Anya T. Joynt, Erin W. Kavanagh, Gregory A. Newby, Shakela Mitchell, Alice C. Eastman, Kathleen C. Paul, Alyssa D. Bowling, Derek L. Osorio, Christian A. Merlo, Shivani U. Patel, Karen S. Raraigh, David R. Liu, Neeraj Sharma, Garry R. Cutting
Format:	Article
Language:	English
Published:	Elsevier 2023-09-01
Subjects:	MT: RNA/DNA Editing canonical splice site variant CSSV CRISPR-Cas9-mediated adenine base editing ABE primary airway epithelial cells
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253123001701