Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-associated X-linked Retinitis Pigmentosa.
Michaelides M, Besirli CG, Yang Y, de Guimaraes TAC, Sui Chien W, Huckfeldt RM, Comander JI, Sahel JA, Shah SM, Tee JJL, Kumaran N, Georgiadis A, Minnick P, Zeldin R, Naylor S, Xu J, Clark M, Anglade E, Wong P, Fleck PR, Fung A, Peluso C, Kalitzeos A, Georgiou M, Ripamonti C, Smith AJ, Ali RR, Forbes A, Bainbridge J.
Michaelides M, et al. Among authors: shah sm.
Am J Ophthalmol. 2024 Jun 11:S0002-9394(24)00244-7. doi: 10.1016/j.ajo.2024.05.034. Online ahead of print.
Am J Ophthalmol. 2024.
PMID: 38871269