Cited 5 times in
Generation of a gene edited hemophilia A patient-derived iPSC cell line, YCMi001-B-1, by targeted insertion of coagulation factor FVIII using CRISPR/Cas9
DC Field | Value | Language |
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dc.contributor.author | 김동욱 | - |
dc.date.accessioned | 2020-12-01T17:46:04Z | - |
dc.date.available | 2020-12-01T17:46:04Z | - |
dc.date.issued | 2020-10 | - |
dc.identifier.issn | 1873-5061 | - |
dc.identifier.uri | https://ir.ymlib.yonsei.ac.kr/handle/22282913/180437 | - |
dc.description.abstract | Hemophilia A is an ideal target for cell or gene therapy because a mild increase in coagulation factor VIII (FVIII) improves symptoms in patients with severe hemophilia A. In this study, we used CRISPR/Cas9 to insert FVIII cDNA into exon 1 of the mutant FVIII locus in induced pluripotent stem cells (iPSCs) from a hemophilia A patient. This gene-modified YCMi001-B-1 line maintained its pluripotency, formed all three germ layers, and had a normal karyotype. In addition, FVIII expression was confirmed in YCMi001-B-1-derived endothelial cells. | - |
dc.description.statementOfResponsibility | open | - |
dc.language | English | - |
dc.publisher | Elsevier | - |
dc.relation.isPartOf | STEM CELL RESEARCH | - |
dc.rights | CC BY-NC-ND 2.0 KR | - |
dc.title | Generation of a gene edited hemophilia A patient-derived iPSC cell line, YCMi001-B-1, by targeted insertion of coagulation factor FVIII using CRISPR/Cas9 | - |
dc.type | Article | - |
dc.contributor.college | College of Medicine (의과대학) | - |
dc.contributor.department | Dept. of Physiology (생리학교실) | - |
dc.contributor.googleauthor | Jin Jea Sung | - |
dc.contributor.googleauthor | Sanghyun Park | - |
dc.contributor.googleauthor | Sang-Hwi Choi | - |
dc.contributor.googleauthor | Jongwan Kim | - |
dc.contributor.googleauthor | Myung Soo Cho | - |
dc.contributor.googleauthor | Dong-Wook Kim | - |
dc.identifier.doi | 10.1016/j.scr.2020.101948 | - |
dc.contributor.localId | A00406 | - |
dc.relation.journalcode | J02680 | - |
dc.identifier.eissn | 1876-7753 | - |
dc.identifier.pmid | 32798916 | - |
dc.contributor.alternativeName | Kim, Dong Wook | - |
dc.contributor.affiliatedAuthor | 김동욱 | - |
dc.citation.volume | 48 | - |
dc.citation.startPage | 101948 | - |
dc.identifier.bibliographicCitation | STEM CELL RESEARCH, Vol.48 : 101948, 2020-10 | - |
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