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 Aug 5;48:101948
PubMed 32798916
DOI 10.1016/j.scr.2020.101948

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