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 Guimaraes,Sui Chien Wong,Rachel M. Huckfeldt,Jason I. Comander,Jose-Alain Sahel,Syed Mahmood Shah,James J. L. Tee,Neruban Kumaran,Anastasios Georgiadis, Pansy Minnick, Robert Zeldin,Stuart Naylor, Jialin Xu,Michael Clark,Eddy Anglade,Peggy Wong, Penny R. Fleck,Albert Fung, Colleen Peluso,Angelos Kalitzeos,Michalis Georgiou,Caterina Ripamonti,Alexander J. Smith,Robin R. Ali,Alexandria Forbes,James Bainbridge American journal of ophthalmology（2024）
关键词
retinitis pigmentosa,X-linked retinitis pigmentosa,retinitis pigmentosa GTPase regulator,gene therapy, clinical trial
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