Generation of a homozygous GBA deletion human embryonic stem cell line
Summary
We describe the generation of a biallelic GBA deletion human embryonic stem cell line using zinc finger nuclease-mediated gene targeting. The homozygous targeting of exon 4 of the GBA locus leads to a complete loss of glucocerebrosidase (GCase) protein expression. Copyright © 2017 The Authors. Published by Elsevier B.V. All rights reserved.
| Authors | Gündner AL, Meyer CA, Aigner S, Christensen K, Patsch C, Jagasia R, Baumann K, Burcin M |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2017 Aug;23:122-126 |
| PubMed | 28925361 |
| DOI | 10.1016/j.scr.2017.07.009 |