Article
CRISPR-Cas9-Mediated Correction of SLC12A3 Gene Mutation Rescues the Gitelman's Disease Phenotype in a Patient-Derived Kidney Organoid System.
International journal of molecular sciences - 3 Feb 2023
Lim Sun Woo, Fang Xianying, Cui Sheng, Lee Hanbi, Shin Yoo Jin, Ko Eun Jeong, Lee Kang In, Lee Jae Young, Chung Byung Ha, Yang Chul Woo
Abstract excerpt
The aim of this study is to explore the possibility of modeling Gitelman's disease (GIT) with human-induced pluripotent stem cell (hiPSC)-derived kidney organoids and to test whether gene correction using CRISPR/Cas9 can rescue the disease phenotype of GIT. To model GIT, we used the hiPSC line CMCi002 (CMC-GIT-001), generated using PBMCs from GIT patients with SLC12A3 gene mutation. Using the CRISPR-Cas9 system,...
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