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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.
Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid.
Vercauteren K, Hoffman BE, Zolotukhin I, Keeler GD, Xiao JW, Basner-Tschakarjan E, High KA, Ertl HC, Rice CM, Srivastava A, de Jong YP, Herzog RW. Vercauteren K, et al. Among authors: high ka. Mol Ther. 2016 Jun;24(6):1042-1049. doi: 10.1038/mt.2016.61. Epub 2016 Mar 29. Mol Ther. 2016. PMID: 27019999 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: 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.
An edible switch for gene therapy.
Anguela XM, High KA. Anguela XM, et al. Among authors: high ka. Nat Biotechnol. 2016 Aug 9;34(8):824-5. doi: 10.1038/nbt.3645. Nat Biotechnol. 2016. PMID: 27504775 No abstract available.
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.
239 results