Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa
Topics
AAV5-hRKp.RPGR · botaretigene sparoparvovec · RPGR · X-linked retinitis pigmentosa · XLRP · gene therapy · dose escalation · Subretinal injection · static perimetry · Microperimetry · vision-guided behavior · Retinal sensitivity · Retinal detachment · uveitis · corticosteroid treatment · functional vision outcomes · pediatric patients · randomized immediate versus deferred treatment · Phase 1/2 clinical trial
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Pepkio Research Index (PRI). Topics and Trends in Most Cited Retinal Development and Disorders Papers, Class of 2026. https://pri.pepkio.com/top-papers/retinal-development-and-disorders/2026. Accessed 2026-10-24. Methodology Zheng Su, Tinsley Li, Thematic Shifts in Early-High-Impact Cancer Genomics and Diagnostics Research: A Bibliometric and Semantic Analysis. bioRxiv 2026.07.04.736459; doi: https://doi.org/10.64898/2026.07.04.736459

