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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.
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.
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.
117 results