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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: gray jt. 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.
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: gray jt, gray e. 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: gray jt. 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.
AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.
Binny C, McIntosh J, Della Peruta M, Kymalainen H, Tuddenham EG, Buckley SM, Waddington SN, McVey JH, Spence Y, Morton CL, Thrasher AJ, Gray JT, Castellino FJ, Tarantal AF, Davidoff AM, Nathwani AC. Binny C, et al. Among authors: gray jt. Blood. 2012 Jan 26;119(4):957-66. doi: 10.1182/blood-2011-09-377630. Epub 2011 Dec 1. Blood. 2012. PMID: 22134170 Free PMC article.
97 results