Article
CRISPR-Cas9 genome editing in the parental iPSC line PCIi033-A to introduce the homozygous mutation p.F508del (c.1521_1523del) in the CFTR gene.
Stem cell research - 1 Apr 2026
Simonneau Benjamin, Baghdoyan Sandrine, Cailleret Michel, Simon Stéphanie, Ruckebusch Odile, Vrablikova Barbora, Giraud-Triboult Karine, Kassar Lina El, Fanen Pascale, Duriez Bénédicte
Abstract excerpt
Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene. Patients carrying the most common mutation, p.F508del, benefit from the triple therapy Kaftrio®. We genome-edited the commercially available iPSC line PCIi033-A (wild-type CFTR) to generate the subclone PCIi033-A-5, which is homozygous for the mutation c.1521_1523del (p.F508del), using CRISPR-SpCas9 tools. PCIi033-A-5 has...
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