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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: kay ma. 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.
Gene therapy for the hemophilias.
Kay MA, High K. Kay MA, et al. Proc Natl Acad Sci U S A. 1999 Aug 31;96(18):9973-5. doi: 10.1073/pnas.96.18.9973. Proc Natl Acad Sci U S A. 1999. PMID: 10468539 Free PMC article. Review. No abstract available.
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B. Manno CS, et al. Among authors: kay ma. Blood. 2003 Apr 15;101(8):2963-72. doi: 10.1182/blood-2002-10-3296. Epub 2002 Dec 19. Blood. 2003. PMID: 12515715 Free article.
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Manno CS, et al. Among authors: kay ma. Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12. Nat Med. 2006. PMID: 16474400 Clinical Trial.
Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.
Nichols TC, Raymer RA, Franck HW, Merricks EP, Bellinger DA, DeFriess N, Margaritis P, Arruda VR, Kay MA, High KA. Nichols TC, et al. Among authors: kay ma. Haemophilia. 2010 May;16 Suppl 3(Suppl 3):19-23. doi: 10.1111/j.1365-2516.2010.02255.x. Haemophilia. 2010. PMID: 20586797 Free PMC article. Review.
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: kay ma. 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. Among authors: kay ma. 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.
305 results