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Page 1
Haemophilia care: the only constant is change.
Samelson-Jones BJ, George LA. Samelson-Jones BJ, et al. Br J Haematol. 2021 Sep;194(5):805-807. doi: 10.1111/bjh.17661. Epub 2021 Aug 2. Br J Haematol. 2021. PMID: 34340257 Free PMC article. No abstract available.
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.
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.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.
George LA, Monahan PE, Eyster ME, Sullivan SK, Ragni MV, Croteau SE, Rasko JEJ, Recht M, Samelson-Jones BJ, MacDougall A, Jaworski K, Noble R, Curran M, Kuranda K, Mingozzi F, Chang T, Reape KZ, Anguela XM, High KA. George LA, et al. N Engl J Med. 2021 Nov 18;385(21):1961-1973. doi: 10.1056/NEJMoa2104205. N Engl J Med. 2021. PMID: 34788507 Free PMC article. Clinical Trial.
Adeno-Associated Virus Gene Therapy for Hemophilia.
Samelson-Jones BJ, George LA. Samelson-Jones BJ, et al. Annu Rev Med. 2023 Jan 27;74:231-247. doi: 10.1146/annurev-med-043021-033013. Epub 2022 Sep 14. Annu Rev Med. 2023. PMID: 36103998 Free PMC article. Review.
42 results