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

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 r...

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Main Authors: Anya T. Joynt (Author), Erin W. Kavanagh (Author), Gregory A. Newby (Author), Shakela Mitchell (Author), Alice C. Eastman (Author), Kathleen C. Paul (Author), Alyssa D. Bowling (Author), Derek L. Osorio (Author), Christian A. Merlo (Author), Shivani U. Patel (Author), Karen S. Raraigh (Author), David R. Liu (Author), Neeraj Sharma (Author), Garry R. Cutting (Author)
Format: Book
Published: Elsevier, 2023-09-01T00:00:00Z.
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3rd Floor Main Library

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