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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.
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.
Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy.
Samelson-Jones BJ, Arruda VR. Samelson-Jones BJ, et al. Mol Ther Methods Clin Dev. 2018 Dec 31;12:184-201. doi: 10.1016/j.omtm.2018.12.007. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30705923 Free PMC article. Review.
Gene Therapy for Inherited Bleeding Disorders.
Arruda VR, Weber J, Samelson-Jones BJ. Arruda VR, et al. Semin Thromb Hemost. 2021 Mar;47(2):161-173. doi: 10.1055/s-0041-1722862. Epub 2021 Feb 26. Semin Thromb Hemost. 2021. PMID: 33636747 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.
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