Generation of a homozygous DNAJC19 knockout human embryonic stem cell line by CRISPR/Cas9 system
Summary
The DNAJC19 gene, a member of DNAJ heat shock protein (Hsp40) family, is localized within the inner mitochondrial membrane (IMM) and plays a crucial role in regulating the function and localization of mitochondrial Hsp70 (MtHsp70). Mutations in the DNAJC19 gene cause Dilated Cardiomyopathy with Ataxia Syndrome (DCMA). The precise mechanisms underlying the DCMA phenotype caused by DNAJC19 mutations remain poorly understood, and effective treatment modalities were lacking unitl recently. By using CRISPR-Cas9 gene editing technology, this study generated a DNAJC19-knockout (DNAJC19-KO) human embryonic stem cell line (hESC), which will be a useful tool in studying the pathogenesis of DCMA. Copyright © 2024 The Authors. Published by Elsevier B.V. All rights reserved.
| Authors | Chen G, Bai R, Huang P, Liu X, Ni J, Chen L |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2024 Jun;77:103427 |
| PubMed | 38696852 |
| DOI | 10.1016/j.scr.2024.103427 |