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Page 1
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: high ka. 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.
Identification of mouse AAV capsid-specific CD8+ T cell epitopes.
Sabatino DE, Mingozzi F, Hui DJ, Chen H, Colosi P, Ertl HC, High KA. Sabatino DE, et al. Among authors: high ka. Mol Ther. 2005 Dec;12(6):1023-33. doi: 10.1016/j.ymthe.2005.09.009. Epub 2005 Nov 2. Mol Ther. 2005. PMID: 16263332 Free 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: high ka. 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.
Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice.
Zou C, Vercauteren KOA, Michailidis E, Kabbani M, Zoluthkin I, Quirk C, Chiriboga L, Yazicioglu M, Anguela XM, Meuleman P, High KA, Herzog RW, de Jong YP. Zou C, et al. Among authors: high ka. Mol Ther Methods Clin Dev. 2020 Jun 2;18:189-198. doi: 10.1016/j.omtm.2020.05.033. eCollection 2020 Sep 11. Mol Ther Methods Clin Dev. 2020. PMID: 32637450 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: high ka. Sci Transl Med. 2013 Jul 17;5(194):194ra92. doi: 10.1126/scitranslmed.3005795. Sci Transl Med. 2013. PMID: 23863832 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. Among authors: high ka. 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.
239 results