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
Summary
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. Copyright © 2020 The Authors. Published by Elsevier B.V. All rights reserved.
| Authors | Sung JJ, Park S, Choi SH, Kim J, Cho MS, Kim DW |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2020 Oct;48:101948 |
| PubMed | 32798916 |
| DOI | 10.1016/j.scr.2020.101948 |