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Page 1
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
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.
Roctavian gene therapy for hemophilia A.
Samelson-Jones BJ, Small JC, George LA. Samelson-Jones BJ, et al. Blood Adv. 2024 Oct 8;8(19):5179-5189. doi: 10.1182/bloodadvances.2023011847. Blood Adv. 2024. PMID: 38991118 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.
No CpGs for AAVs?
George LA. George LA. Blood. 2021 Feb 11;137(6):721-723. doi: 10.1182/blood.2020009285. Blood. 2021. PMID: 33570612 Free PMC article.
Coagulation factor VIII regulates von Willebrand factor homeostasis invivo.
Cao W, Trask AR, Bignotti AI, George LA, Doshi BS, Sabatino DE, Yada N, Zheng L, Camire RM, Zheng XL. Cao W, et al. J Thromb Haemost. 2023 Dec;21(12):3477-3489. doi: 10.1016/j.jtha.2023.09.004. Epub 2023 Sep 17. J Thromb Haemost. 2023. PMID: 37726033 Free PMC article.
Total Knee Arthroplasty after Gene Therapy for Hemophilia B.
Xue F, Wang P, Yuan Z, Shi C, Fang Y, Liu W, Wang Y, Xiao X, Yang R, George LA, Zhang L. Xue F, et al. N Engl J Med. 2022 Oct 27;387(17):1622-1624. doi: 10.1056/NEJMc2211173. N Engl J Med. 2022. PMID: 36306204 Free PMC article. No abstract available.
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 gene therapy: ushering in a new treatment paradigm?
George LA. George LA. Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):226-233. doi: 10.1182/hematology.2021000254. Hematology Am Soc Hematol Educ Program. 2021. PMID: 34889378 Free PMC article. Review.
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