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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. Among authors: morton cl. 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.
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: morton cl. 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.
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.
McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC. McIntosh J, et al. Among authors: morton cl. Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20. Blood. 2013. PMID: 23426947 Free PMC article.
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: morton cl. 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.
Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.
Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA. Paulk NK, et al. Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25. Mol Ther. 2018. PMID: 29055620 Free PMC article.
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: morton cl. 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.
The role of histone demethylase KDM4B in Myc signaling in neuroblastoma.
Yang J, AlTahan AM, Hu D, Wang Y, Cheng PH, Morton CL, Qu C, Nathwani AC, Shohet JM, Fotsis T, Koster J, Versteeg R, Okada H, Harris AL, Davidoff AM. Yang J, et al. Among authors: morton cl. J Natl Cancer Inst. 2015 Apr 29;107(6):djv080. doi: 10.1093/jnci/djv080. Print 2015 Jun. J Natl Cancer Inst. 2015. PMID: 25925418 Free PMC article.
128 results