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Page 1
In vivo genome editing of the albumin locus as a platform for protein replacement therapy.
Sharma R, Anguela XM, Doyon Y, Wechsler T, DeKelver RC, Sproul S, Paschon DE, Miller JC, Davidson RJ, Shivak D, Zhou S, Rieders J, Gregory PD, Holmes MC, Rebar EJ, High KA. Sharma R, et al. Among authors: anguela xm. Blood. 2015 Oct 8;126(15):1777-84. doi: 10.1182/blood-2014-12-615492. Epub 2015 Aug 21. Blood. 2015. PMID: 26297739 Free PMC article.
In vivo genome editing restores haemostasis in a mouse model of haemophilia.
Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L, Murphy SL, Finn JD, Khazi FR, Zhou S, Paschon DE, Rebar EJ, Bushman FD, Gregory PD, Holmes MC, High KA. Li H, et al. Among authors: anguela xm. Nature. 2011 Jun 26;475(7355):217-21. doi: 10.1038/nature10177. Nature. 2011. PMID: 21706032 Free PMC article.
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.
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.
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.
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A.
Elkouby L, Armour SM, Toso R, DiPietro M, Davidson RJ, Nguyen GN, Willet M, Kutza S, Silverberg J, Frick J, Crosariol M, Wang Y, Wang C, High KA, Sabatino DE, Anguela XM. Elkouby L, et al. Among authors: anguela xm. Mol Ther Methods Clin Dev. 2021 Nov 24;24:20-29. doi: 10.1016/j.omtm.2021.11.005. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 34977269 Free PMC article.
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.
24 results