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George, Lindsey A (2017) Hemophilia gene therapy comes of age. Blood Adv 1:2591-2599
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Russell, Stephen; Bennett, Jean; Wellman, Jennifer A et al. (2017) Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet 390:849-860
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