Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-associated X-linked Retinitis Pigmentosa

Michel Michaelides; Cagri G. Besirli; Yesa Yang; Thales A. C. de Guimarães; Sui Chien Wong; Rachel M. Huckfeldt; Jason Comander; José‐Alain Sahel; Syed Mahmood Shah; James Tee; Neruban Kumaran; Anastasios Georgiadis; PANSY MINNICK; ROBERT ZELDIN; Stuart Naylor; Jialin Xu; Michael Clark; Eddy Anglade; Peggy Wong; P. Fleck; Albert Fung; Colleen Peluso; Angelos Kalitzeos; Michalis Georgiou; Caterina Ripamonti; Alexander J. Smith; Robin R. Ali; Alexandria Forbes; James Bainbridge

doi:10.1016/j.ajo.2024.05.034

Abstract

To assess the safety and efficacy of AAV5-hRKp.RPGR in participants with retinitis pigmentosa GTPase regulator (RPGR)-associated X-linked retinitis pigmentosa (XLRP).

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Michaelides et al. (2024) studied this question.

synapsesocial.com/papers/68e64f8fb6db6435875e0673 https://doi.org/10.1016/j.ajo.2024.05.034

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Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-associated X-linked Retinitis Pigmentosa

Abstract

Discussion

Cite This Study

Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-associated X-linked Retinitis Pigmentosa

Abstract

Discussion

Cite This Study

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