Raffini, Leslie

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

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Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia b

George, L. A., Ragni, M. V., Rasko, J. E. J., Raffini, L. J., Samelson-Jones, B. J., Ozelo, M., Hazbon, M., Runowski, A. R., Wellman, J. A., Wachtel, K., et al. (2020) · Molecular Therapy

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Evolutionary Insights Into Coagulation Factor IX Padua and Other High-Specific-Activity Variants

Samelson-Jones, B. J., Finn, J. D., Raffini, L. J., Merricks, E. P., Camire, R. M., Nichols, T. C., & Arruda, V. R. (2021) · Blood Advances

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