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CRISPR-Cas9-Mediated Correction of SLC12A3 Gene Mutation Rescues the Gitelman’s Disease Phenotype in a Patient-Derived Kidney Organoid System

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 CMCi0...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9917614/
https://www.ncbi.nlm.nih.gov/pubmed/36769335
http://dx.doi.org/10.3390/ijms24033019