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Article

Targeted correction and restored function of the CFTR gene in cystic fibrosis induced pluripotent stem cells.

Stem cell reports - 14 Apr 2015

Crane Ana M, Kramer Philipp, Bui Jacquelin H, Chung Wook Joon, Li Xuan Shirley, Gonzalez-Garay Manuel L, Hawkins Finn, Liao Wei, Mora Daniela, Choi Sangbum, Wang Jianbin, Sun Helena C, Paschon David E, Guschin Dmitry Y, Gregory Philip D, Kotton Darrell N, Holmes Michael C, Sorscher Eric J, Davis Brian R

Abstract excerpt

Recently developed reprogramming and genome editing technologies make possible the derivation of corrected patient-specific pluripotent stem cell sources-potentially useful for the development of new therapeutic approaches. Starting with skin fibroblasts from patients diagnosed with cystic fibrosis, we derived and characterized induced pluripotent stem cell (iPSC) lines. We then utilized zinc-finger nucleases...

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Share to Cystic Fibrosis
Share to Cystic Fibrosis Transmembrane Conductance Regulator

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 25772471
DOI: 10.1016/j.stemcr.2015.02.005
PMCID: PMC4400651

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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