Generation of a gene-corrected isogenic human iPS cell line (CSUASOi006-A-2) from a retinitis pigmentosa patient using CRISPR/Cas9 technology
Summary
Retinitis pigmentosa (RP) is a heterogeneous group of hereditary eye disorders characterized by a progressive degeneration of the light-sensing photoreceptor cells in the retina. Currently, there are no effective treatments. In a previous study, we generated a human induced pluripotent stem (iPS) cell line (CSUASOi006-A) from an RP patient carrying a PRPF8 (c.C5792T) mutation. In this study, we corrected the c.5792C > T mutation in the PRPF8 gene using CRISPR/Cas9 technology and generated an isogenic control cell line (CSUASOi006-A-2). This provides an important cellular resource for RP research. Copyright © 2025 The Author(s). Published by Elsevier B.V. All rights reserved.
| Authors | Chen H, Liang Y, Liang Y, Chen Y, Li X, Zhang R, Duan C, Li W, Cui Z, Gu J, Ding C, Sun X, Chen J |
|---|---|
| Journal | Stem cell research |
| Publication Date | 2025 Apr 26;86:103727 |
| PubMed | 40318521 |
| DOI | 10.1016/j.scr.2025.103727 |