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A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.
Nat Biotechnol. 2021 Jan;39(1):47-55. doi: 10.1038/s41587-020-0741-7. Epub 2020 Nov 16.
Nat Biotechnol. 2021.
PMID: 33199875
Free PMC article.
Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice.
Nguyen GN, Lindgren JR, Seleme MC, Kafle S, Zander CB, Zheng XL, Sabatino DE.
Nguyen GN, et al.
J Thromb Haemost. 2023 Aug;21(8):2101-2113. doi: 10.1016/j.jtha.2023.04.012. Epub 2023 Apr 18.
J Thromb Haemost. 2023.
PMID: 37080538
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Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A.
Nguyen GN, George LA, Siner JI, Davidson RJ, Zander CB, Zheng XL, Arruda VR, Camire RM, Sabatino DE.
Nguyen GN, et al.
J Thromb Haemost. 2017 Jan;15(1):110-121. doi: 10.1111/jth.13543. Epub 2016 Nov 25.
J Thromb Haemost. 2017.
PMID: 27749002
Free PMC article.
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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.
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.
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