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Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial by MacLaren, RE, Fischer, MD, Gow, JA, Lam, BL, Sankila, E-MK, Girach, A, Panda, S, Yoon, D, Zhao, G, Pennesi, ME
Published 2023Journal article -
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Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR by Cehajic-Kapetanovic, J, Xue, K, Martinez-Fernandez de la Camara, C, Nanda, A, Davies, A, Wood, LJ, Salvetti, AP, Fischer, MD, Aylward, JW, Barnard, AR, Jolly, JK, Luo, E, Lujan, BJ, Ong, T, Girach, A, Black, GCM, Gregori, NZ, Davis, JL, Rosa, PR, Lotery, AJ, Lam, BL, Stanga, PE, MacLaren, RE
Published 2020Journal article