Michaelides M, Besirli CG, Yang Y, de Guimaraes TAC, et al. Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-associated X-linked Retinitis Pigmentosa. Am J Ophthalmol 2024 Jun 11:S0002-9394(24)00244.
PMID: 38871269