Efficacy and Safety of Gene Therapy for RPGR Gene-Associated X-Linked Retinitis Pigmentosa: A Systematic Review and Meta-Analysis
XLRP, a severe hereditary retinal dystrophy primarily caused by RPGR gene mutations, lacks curative treatments. AAV-mediated RPGR gene therapy shows promise in early trials, but systemic evaluation of its efficacy and safety is absent. This evaluates gene therapy as a novel approach, noting short-term efficacy but ocular AE risks.
Authors
- Qinghua Qiu
- Jiaqi Yang (ORCID: https://orcid.org/0009-0008-7495-7223)
- Dongwei Lai (ORCID: https://orcid.org/0000-0002-9568-8463)
- Yiou Lei
- Bingqing Xiao
- Ying Li
Institutions
- Central South University (CN)
- Shanghai Jiao Tong University (CN)
- Shanghai Eye Disease Prevention & Treatment Center (CN)
- Tongren Hospital (CN)
- Shanghai First People's Hospital (CN)
Publication Details
- Journal
- Translational Vision Science & Technology
- Published
- 2026-03-03
- DOI
- https://doi.org/10.1167/tvst.15.3.5
- Primary Topic
- Retinal Development and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.00