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

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dc.contributor.author강훈철-
dc.contributor.author김형범-
dc.contributor.author장지호-
dc.date.accessioned2022-03-11T05:57:43Z-
dc.date.available2022-03-11T05:57:43Z-
dc.date.issued2022-03-
dc.identifier.issn1873-5061-
dc.identifier.urihttps://ir.ymlib.yonsei.ac.kr/handle/22282913/187881-
dc.description.abstractX-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.-
dc.description.statementOfResponsibilityopen-
dc.formatapplication/pdf-
dc.languageEnglish-
dc.publisherElsevier-
dc.relation.isPartOfSTEM CELL RESEARCH-
dc.rightsCC BY-NC-ND 2.0 KR-
dc.titleGeneration of mutation-corrected induced pluripotent stem cell lines derived from adrenoleukodystrophy patient by using homology directed repair-
dc.typeArticle-
dc.contributor.collegeCollege of Medicine (의과대학)-
dc.contributor.departmentDept. of Pediatrics (소아과학교실)-
dc.contributor.googleauthorEul Sik Jung-
dc.contributor.googleauthorJi Hun Kim-
dc.contributor.googleauthorMi-Yoon Chang-
dc.contributor.googleauthorWonjun Hong-
dc.contributor.googleauthorZhejiu Quan-
dc.contributor.googleauthorSeung Hyun Kim-
dc.contributor.googleauthorSeungkwon You-
dc.contributor.googleauthorDae-Sung Kim-
dc.contributor.googleauthorJiho Jang-
dc.contributor.googleauthorSang-Hun Lee-
dc.contributor.googleauthorHyongbum Henry Kim-
dc.contributor.googleauthorHoon Chul Kang-
dc.identifier.doi10.1016/j.scr.2022.102664-
dc.contributor.localIdA00102-
dc.contributor.localIdA01148-
dc.relation.journalcodeJ02680-
dc.identifier.eissn1876-7753-
dc.identifier.pmid35042083-
dc.subject.keywordCRISPR/Cas9-
dc.subject.keywordGenome editing-
dc.subject.keywordInduced pluripotent stem cell-
dc.subject.keywordX-linked adrenoleukodystrophy-
dc.contributor.alternativeNameKang, Hoon Chul-
dc.contributor.affiliatedAuthor강훈철-
dc.contributor.affiliatedAuthor김형범-
dc.citation.volume59-
dc.citation.startPage102664-
dc.identifier.bibliographicCitationSTEM CELL RESEARCH, Vol.59 : 102664, 2022-03-
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

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