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Generation of mutation-corrected induced pluripotent stem cell lines derived from adrenoleukodystrophy patient by using homology directed repair

Authors
 Eul Sik Jung  ;  Ji Hun Kim  ;  Mi-Yoon Chang  ;  Wonjun Hong  ;  Zhejiu Quan  ;  Seung Hyun Kim  ;  Seungkwon You  ;  Dae-Sung Kim  ;  Jiho Jang  ;  Sang-Hun Lee  ;  Hyongbum Henry Kim  ;  Hoon Chul Kang 
Citation
 STEM CELL RESEARCH, Vol.59 : 102664, 2022-03 
Journal Title
STEM CELL RESEARCH
ISSN
 1873-5061 
Issue Date
2022-03
Keywords
CRISPR/Cas9 ; Genome editing ; Induced pluripotent stem cell ; X-linked adrenoleukodystrophy
Abstract
X-linked adrenoleukodystrophy (ALD) caused by the ABCD1 mutation, is the most common inherited peroxisomal disease. Previously, we generated an ALD patient-derived SCHi001-A iPSC model. In this study, we have performed the first genome editing of ALD patient-derived SCHi001-A iPSCs using homology-directed repair (HDR). The mutation site, c.1534G > A [GenBank: NM_000033.4], was corrected by introducing ssODN and the CRISPR/Cas9 system. The cell line exhibited normal iPSC plulipotency marker expression following genome editing. Mutation-corrected iPSCs from SCHi001-A iPSC line can be used in research into the pathophysiology of and therapeutics for ALD.
Files in This Item:
T202200207.pdf Download
DOI
10.1016/j.scr.2022.102664
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pediatrics (소아과학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Pharmacology (약리학교실) > 1. Journal Papers
Yonsei Authors
Kang, Hoon Chul(강훈철) ORCID logo https://orcid.org/0000-0002-3659-8847
Kim, Hyongbum(김형범) ORCID logo https://orcid.org/0000-0002-4693-738X
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/187881
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