Article
Generation of human induced pluripotent stem cells from cystic fibrosis patient carrying nonsense mutation (p.S308X) in CFTR gene.
Stem cell research - 1 Apr 2022
Khor Winnie, Hwang Tzyh-Chang, Wang Chih-Chien, Yarmishyn Aliaksandr A, Yeh Jiunn-Tyng, Chiou Shih-Hwa, Chou Shih-Jie
Abstract excerpt
Cystic fibrosis (CF) is a genetic disease affects CFTR channel synthesis. While 90 percent of the CF patients now benefit from small molecule target therapies, this treatment has yet to extend to those bearing nonsense mutations. Studies of these rare mutations using cell lines with native pathological signatures of the disease may lead to breakthroughs in therapeutic development. Here, we report the generation...
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