Article
Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs.
Cell reports - 1 Sept 2015
Firth Amy L, Menon Tushar, Parker Gregory S, Qualls Susan J, Lewis Benjamin M, Ke Eugene, Dargitz Carl T, Wright Rebecca, Khanna Ajai, Gage Fred H, Verma Inder M
Abstract excerpt
Lung disease is a major cause of death in the United States, with current therapeutic approaches serving only to manage symptoms. The most common chronic and life-threatening genetic disease of the lung is cystic fibrosis (CF) caused by mutations in the cystic fibrosis transmembrane regulator (CFTR). We have generated induced pluripotent stem cells (iPSCs) from CF patients carrying a homozygous deletion of F508...
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