Generation of a human SLC12A3 knock-in human induced pluripotent stem cell line (CMCi014-A-82) using CRISPR-Cas9 system.

Autor:	Woo Lim S; Transplant Research Center, College of Medcine, The Catholic University of Korea, Seoul, Republic of Korea., In Lee K; R&D Center, ToolGen, Inc., Seoul, Republic of Korea., Cui S; Transplant Research Center, College of Medcine, The Catholic University of Korea, Seoul, Republic of Korea., Fang X; Transplant Research Center, College of Medcine, The Catholic University of Korea, Seoul, Republic of Korea., Jin Shin Y; Transplant Research Center, College of Medcine, The Catholic University of Korea, Seoul, Republic of Korea., Lee H; Transplant Research Center, College of Medcine, The Catholic University of Korea, Seoul, Republic of Korea; Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea., Woo Yang C; Transplant Research Center, College of Medcine, The Catholic University of Korea, Seoul, Republic of Korea; Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea., Young Lee J; R&D Center, ToolGen, Inc., Seoul, Republic of Korea. Electronic address: jy.lee2@toolgen.com., Ha Chung B; Transplant Research Center, College of Medcine, The Catholic University of Korea, Seoul, Republic of Korea; Department of Internal Medicine, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea. Electronic address: chungbh@catholic.ac.kr.
Jazyk:	angličtina
Zdroj:	Stem cell research [Stem Cell Res] 2024 Dec; Vol. 81, pp. 103522. Date of Electronic Publication: 2024 Jul 31.
DOI:	10.1016/j.scr.2024.103522
Abstrakt:	Gitelman's disease is caused by a genetic mutation in the solute carrier family 12 member 3 (SLC12A3) gene, which encodes the sodium chloride cotransporter. In this study, we generated a stable human induced pluripotent stem cell (hiPSC) line, WTC11-SLC12A3 (CMCi014-A-82), by knocking in the entire SLC12A3 gene at the SHS231 locus in healthy wild-type control hiPSCs (WTC11). We verified that WTC11-SLC12A3 expressed pluripotency markers and exhibited normal stem cell morphology. Furthermore, this cell line maintains a normal karyotype and can differentiate into the three germ layers. Therefore, this cell line may provide a basis for gene therapy for Gitelman's disease. Competing Interests: Declaration of competing interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper. (Copyright © 2024 The Authors. Published by Elsevier B.V. All rights reserved.)
Databáze:	MEDLINE
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