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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. Among authors: george la. 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.
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.
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.
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.
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. 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.
111 results