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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. Among authors: anguela xm. 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. Among authors: anguela xm. 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. Among authors: anguela xm. 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.
An edible switch for gene therapy.
Anguela XM, High KA. Anguela XM, et al. Nat Biotechnol. 2016 Aug 9;34(8):824-5. doi: 10.1038/nbt.3645. Nat Biotechnol. 2016. PMID: 27504775 No abstract available.
Entering the Modern Era of Gene Therapy.
Anguela XM, High KA. Anguela XM, et al. Annu Rev Med. 2019 Jan 27;70:273-288. doi: 10.1146/annurev-med-012017-043332. Epub 2018 Nov 26. Annu Rev Med. 2019. PMID: 30477394 Review.
Robust ZFN-mediated genome editing in adult hemophilic mice.
Anguela XM, Sharma R, Doyon Y, Miller JC, Li H, Haurigot V, Rohde ME, Wong SY, Davidson RJ, Zhou S, Gregory PD, Holmes MC, High KA. Anguela XM, et al. Blood. 2013 Nov 7;122(19):3283-7. doi: 10.1182/blood-2013-04-497354. Epub 2013 Oct 1. Blood. 2013. PMID: 24085764 Free PMC article.
Overcoming preexisting humoral immunity to AAV using capsid decoys.
Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, Wright JF, High KA. Mingozzi F, et al. Among authors: anguela xm. Sci Transl Med. 2013 Jul 17;5(194):194ra92. doi: 10.1126/scitranslmed.3005795. Sci Transl Med. 2013. PMID: 23863832 Free PMC article.
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