Article
Prime editing-mediated correction of the CFTR W1282X mutation in iPSCs and derived airway epithelial cells.
PloS one - 1 Jan 2023
Li Chao, Liu Zhong, Anderson Justin, Liu Zhongyu, Tang Liping, Li Yao, Peng Ning, Chen Jianguo, Liu Xueming, Fu Lianwu, Townes Tim M, Rowe Steven M, Bedwell David M, Guimbellot Jennifer, Zhao Rui
Abstract excerpt
A major unmet need in the cystic fibrosis (CF) therapeutic landscape is the lack of effective treatments for nonsense CFTR mutations, which affect approximately 10% of CF patients. Correction of nonsense CFTR mutations via genomic editing represents a promising therapeutic approach. In this study, we tested whether prime editing, a novel CRISPR-based genomic editing method, can be a potential therapeutic modality...
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