Introduction of an RS1 mutation causative variant consistent with identified XLRS patient using CRISPR/Cas9 technology in normal iPSC
X-linked retinoschisis (XLRS) is a common retinal genetic disease that occurs in juvenile males and causes progressive visual impairment. This presents a schisis in the macula or peripheral retina of bilateral eyes, which has no effective treatment. Here, we introduced the RS1 (c.C304T, p.R102W) mutation into a normal induced pluripotent stem (iPS) cell line using CRISPR/Cas9 technology. This missense mutation was consistent with that observed in the XLRS patient-derived iPS cell line (CSUASOi001-A). Conclusively, establishing a directed gene mutation cell line (CSUi007-A) provides a useful cell resource to investigate XLRS pathogenesis.
Authors
- Xihao Sun (ORCID: https://orcid.org/0000-0002-3470-2006)
- Jiansu Chen (ORCID: https://orcid.org/0000-0001-7332-8427)
- Yuqin Liang (ORCID: https://orcid.org/0000-0002-2448-4746)
- Zekai Cui (ORCID: https://orcid.org/0000-0003-3670-6219)
- Shibo Tang (ORCID: https://orcid.org/0000-0003-2737-6780)
- Shengru Mao
- Chunwen Duan
- Jianing Gu (ORCID: https://orcid.org/0000-0003-2241-5533)
- Bing Jiang (ORCID: https://orcid.org/0000-0001-6594-4019)
- Chengcheng Ding
Institutions
- Central South University (CN)
- Jinan University (CN)
- Second Xiangya Hospital of Central South University (CN)
Publication Details
- Journal
- Stem Cell Research
- Published
- 2024-09-01
- DOI
- https://doi.org/10.1016/j.scr.2024.103549
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Natural Science Foundation of China