SynthesisTranslational vision science & technology2026
Efficacy and Safety of Gene Therapy for RPGR Gene-Associated X-Linked Retinitis Pigmentosa: A Systematic Review and Meta-Analysis.
Synthesis in Translational vision science & technology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Functional Outcomes and Ocular Safety of Subretinal AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: A Systematic Review and Meta-Analysis.Journal of clinical medicine · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Purpose: This study aimed to conduct a systematic review and meta-analysis to synthesize clinical evidence on adeno-associated viral (AAV)-based RPGR gene therapy for X-linked retinitis pigmentosa (XLRP), guiding clinical decisions and future research. Methods: Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines and Prospective Register of Systematic Reviews (PROSPERO; CRD420251008337) registration, we searched PubMed, Embase, and other databases through March 2025, including controlled clinical trials evaluating AAV gene therapy for RPGR-related XLRP. Two reviewers independently screened literature, extracted data, and assessed bias using the Cochrane tool. Meta-analyses were performed in RevMan version 5.4, calculating risk ratios (RRs) for dichotomous outcomes and mean difference /standardized mean difference (MD / SMD) for continuous outcomes, with random/fixed-effects models based on heterogeneity (I² > 50%). Results: Five articles from three studies (205 patients) were included. Gene therapy significantly improved low-luminance visual acuity (LLVA) ≥10 Early Treatment Diabetic Retinopathy Study (ETDRS) letters at 6 months (RR = 3.79, P = 0.03) and retinal sensitivity at 6 months (MD = 1.06, P = 0.001) and 12 months (MD = 2.47, P < 0.00001), but not best-corrected visual acuity (VA) or LLVA ≥15 letters. Ocular treatment-emergent adverse event (TEAE) risk increased significantly (RR = 5.52, P < 0.00001), with common events including anterior chamber inflammation and intraocular inflammation. A trend toward higher serious adverse events (SAEs) was observed (odds ratio [OR] = 3.36, P = 0.05). Conclusions: Gene therapy demonstrates short-term efficacy for XLRP, with 6 months as a critical evaluation time point, but requires vigilance for ocular TEAE risks. Heterogeneity and small sample sizes highlight the need for large-scale, long-term multicenter trials to optimize protocols and advance safe clinical application. Translational Relevance: 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.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.