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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM. Nathwani AC, et al. N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10. N Engl J Med. 2011. PMID: 22149959 Free PMC article. Clinical Trial.
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM. Nathwani AC, et al. Among authors: basner tschakarjan e. N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309. N Engl J Med. 2014. PMID: 25409372 Free PMC article. Clinical Trial.
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: basner tschakarjan e. 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.
AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes.
Hui DJ, Edmonson SC, Podsakoff GM, Pien GC, Ivanciu L, Camire RM, Ertl H, Mingozzi F, High KA, Basner-Tschakarjan E. Hui DJ, et al. Among authors: basner tschakarjan e. Mol Ther Methods Clin Dev. 2015 Sep 30;2:15029. doi: 10.1038/mtm.2015.29. eCollection 2015. Mol Ther Methods Clin Dev. 2015. PMID: 26445723 Free PMC article.
25 results