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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.
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: george la. 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.
Hemophilia gene therapy comes of age.
George LA. George LA. Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):587-594. doi: 10.1182/asheducation-2017.1.587. Hematology Am Soc Hematol Educ Program. 2017. PMID: 29222308 Free PMC article. Review.
Hemophilia gene therapy comes of age.
George LA. George LA. Blood Adv. 2017 Dec 8;1(26):2591-2599. doi: 10.1182/bloodadvances.2017009878. eCollection 2017 Dec 12. Blood Adv. 2017. PMID: 29296912 Free PMC article. Review.
The national blueprint for future factor VIII inhibitor clinical trials: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors.
Ragni MV, George LA; Members of Working Group 1, the NHLBI State of the Science Workshop on factor VIII inhibitors: Generating a national blueprint for future research. Ragni MV, et al. Among authors: george la. Haemophilia. 2019 Jul;25(4):581-589. doi: 10.1111/hae.13717. Haemophilia. 2019. PMID: 31329364 Free PMC article.
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.
Activated protein C has a regulatory role in factor VIII function.
Wilhelm AR, Parsons NA, Samelson-Jones BJ, Davidson RJ, Esmon CT, Camire RM, George LA. Wilhelm AR, et al. Among authors: george la. Blood. 2021 May 6;137(18):2532-2543. doi: 10.1182/blood.2020007562. Blood. 2021. PMID: 33512448 Free PMC article.
111 results