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Article

Comparison of Cas9 and Cas12a CRISPR editing methods to correct the W1282X-CFTR mutation.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2022

Santos Lúcia, Mention Karen, Cavusoglu-Doran Kader, Sanz David J, Bacalhau Mafalda, Lopes-Pacheco Miquéias, Harrison Patrick T, Farinha Carlos M

Abstract excerpt

BACKGROUND: W1282X-CFTR variant (c.3846G>A) is the second most common nonsense cystic fibrosis (CF)-causing mutation in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. Even though remarkable breakthroughs have been done towards CF treatment with the approval of four CFTR protein modulators, none of these are approved for patients with nonsense mutations. CRISPR gene editing tools can be of...

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PMID: 34103250
DOI: 10.1016/j.jcf.2021.05.014

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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