PRPF8 Mutation-Induced Defects in Human iPSC-Derived RPE Are Rescued by Adenine Base Editing
PRPF8 mutations in patient-derived iPSC RPE cause functional and ultrastructural defects driven by splicing abnormalities. ABE correction of the PRPF8 mutation in iPSCs can restore PRPF8 expression and alleviate cellular and molecular defects in RPE and highlights the therapeutic potential of precise gene editing correction strategies for RP.
Authors
- H Xu
- Xiang Sun (ORCID: https://orcid.org/0000-0002-1144-1050)
- Hang Chen (ORCID: https://orcid.org/0000-0002-6356-3930)
- Yalan Zhou
- RX Zhang
- Wenwei Li
- Jiansu Chen
- Chengcheng Ding
- Yuan Liang
- Chunwen Duan
Institutions
- Central South University (CN)
- Jinan University (CN)
- He Eye Hospital (CN)
- Shandong Eye Hospital (CN)
- Xiangya Hospital Central South University (CN)
- Affiliated Hospital of Jining Medical University (CN)
- First Affiliated Hospital of Jinan University (CN)
Publication Details
- Journal
- Investigative Ophthalmology & Visual Science
- Published
- 2026-01-09
- DOI
- https://doi.org/10.1167/iovs.67.1.21
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.00