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Article

Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X.

Human molecular genetics - 17 Nov 2023

Mention Karen, Cavusoglu-Doran Kader, Joynt Anya T, Santos Lúcia, Sanz David, Eastman Alice C, Merlo Christian, Langfelder-Schwind Elinor, Scallan Martina F, Farinha Carlos M, Cutting Garry R, Sharma Neeraj, Harrison Patrick T

Abstract excerpt

Small molecule drugs known as modulators can treat ~90% of people with cystic fibrosis (CF), but do not work for premature termination codon variants such as W1282X (c.3846G>A). Here we evaluated two gene editing strategies, Adenine Base Editing (ABE) to correct W1282X, and Homology-Independent Targeted Integration (HITI) of a CFTR superexon comprising exons 23-27 (SE23-27) to enable expression of a CFTR mRNA...

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Identifiers

PMID: 37649273
DOI: 10.1093/hmg/ddad143
PMCID: PMC10656707

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