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Article

Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors.

Nucleic acids research - 11 Oct 2021

Krishnamurthy Sateesh, Traore Soumba, Cooney Ashley L, Brommel Christian M, Kulhankova Katarina, Sinn Patrick L, Newby Gregory A, Liu David R, McCray Paul B

Abstract excerpt

Mutations in the CFTR gene that lead to premature stop codons or splicing defects cause cystic fibrosis (CF) and are not amenable to treatment by small-molecule modulators. Here, we investigate the use of adenine base editor (ABE) ribonucleoproteins (RNPs) that convert A•T to G•C base pairs as a therapeutic strategy for three CF-causing mutations. Using ABE RNPs, we corrected in human airway epithelial cells...

Topics

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Adenine
Cell Line
Cells, Cultured
Cystic Fibrosis
Cystic Fibrosis Transmembrane Conductance Regulator

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Respiratory Mucosa

Ribonucleoproteins

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Open in PubMed Open full text

Identifiers

PMID: 34520545
DOI: 10.1093/nar/gkab788
PMCID: PMC8501978

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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