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Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa.
Michaelides Michel, Besirli Cagri G, Yang Yesa, DE Guimaraes Thales A C, Wong Sui Chien, Huckfeldt Rachel M et al.
Am J OphthalmolJun 202427 citationsClinical Trial
This gene therapy for XLRP-RPGR was safe, well-tolerated, and improved retinal sensitivity and functional vision, supporting further study.