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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Russell S, et al. Among authors: raffini l. Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Lancet. 2017. PMID: 28712537 Free PMC article. Clinical Trial.
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.
Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, De Baere E, Shindler KS, Volpe NJ, Surace EM, Acerra C, Lyubarsky A, Redmond TM, Stone E, Sun J, McDonnell JW, Leroy BP, Simonelli F, Bennett J. Maguire AM, et al. Among authors: raffini l. Lancet. 2009 Nov 7;374(9701):1597-605. doi: 10.1016/S0140-6736(09)61836-5. Epub 2009 Oct 23. Lancet. 2009. PMID: 19854499 Free PMC article. Clinical Trial.
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.
George LA, Ragni MV, Rasko JEJ, Raffini LJ, Samelson-Jones BJ, Ozelo M, Hazbon M, Runowski AR, Wellman JA, Wachtel K, Chen Y, Anguela XM, Kuranda K, Mingozzi F, High KA. George LA, et al. Mol Ther. 2020 Sep 2;28(9):2073-2082. doi: 10.1016/j.ymthe.2020.06.001. Epub 2020 Jun 10. Mol Ther. 2020. PMID: 32559433 Free PMC article. Clinical Trial.
Pediatric transplantation: managing bleeding.
Raffini L, Witmer C. Raffini L, et al. J Thromb Haemost. 2015 Jun;13 Suppl 1:S362-9. doi: 10.1111/jth.12913. J Thromb Haemost. 2015. PMID: 26149048 Free article. Review.
98 results