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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

Authors
 Jin Jea Sung  ;  Sanghyun Park  ;  Sang-Hwi Choi  ;  Jongwan Kim  ;  Myung Soo Cho  ;  Dong-Wook Kim 
Citation
 STEM CELL RESEARCH, Vol.48 : 101948, 2020-10 
Journal Title
 STEM CELL RESEARCH 
ISSN
 1873-5061 
Issue Date
2020-10
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.
Files in This Item:
T202004761.pdf Download
DOI
10.1016/j.scr.2020.101948
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Physiology (생리학교실) > 1. Journal Papers
Yonsei Authors
Kim, Dong Wook(김동욱) ORCID logo https://orcid.org/0000-0002-5025-1532
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/180437
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