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Page 1
Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps.
Brimble MA, Cheng PH, Winston SM, Reeves IL, Souquette A, Spence Y, Zhou J, Wang YD, Morton CL, Valentine M, Thomas PG, Nathwani AC, Gray JT, Davidoff AM. Brimble MA, et al. Among authors: spence y. Mol Ther Methods Clin Dev. 2022 Jan 19;24:280-291. doi: 10.1016/j.omtm.2022.01.008. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2022. PMID: 35211640 Free PMC article.
Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.
Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN, Tuddenham EG, Kemball-Cook G, McIntosh J, Boon-Spijker M, Mertens K, Davidoff AM. Nathwani AC, et al. Among authors: spence y. Blood. 2006 Apr 1;107(7):2653-61. doi: 10.1182/blood-2005-10-4035. Epub 2005 Dec 1. Blood. 2006. PMID: 16322469 Free PMC article.
Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates.
Nathwani AC, Gray JT, McIntosh J, Ng CY, Zhou J, Spence Y, Cochrane M, Gray E, Tuddenham EG, Davidoff AM. Nathwani AC, et al. Among authors: spence y. Blood. 2007 Feb 15;109(4):1414-21. doi: 10.1182/blood-2006-03-010181. Epub 2006 Nov 7. Blood. 2007. PMID: 17090654 Free PMC article.
Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.
Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: spence y. Mol Ther. 2011 May;19(5):876-85. doi: 10.1038/mt.2010.274. Epub 2011 Jan 18. Mol Ther. 2011. PMID: 21245849 Free PMC article.
18 results