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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: reiss um. 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.
Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
Chowdary P, Shapiro S, Makris M, Evans G, Boyce S, Talks K, Dolan G, Reiss U, Phillips M, Riddell A, Peralta MR, Quaye M, Patch DW, Tuddenham E, Dane A, Watissée M, Long A, Nathwani A. Chowdary P, et al. N Engl J Med. 2022 Jul 21;387(3):237-247. doi: 10.1056/NEJMoa2119913. N Engl J Med. 2022. PMID: 35857660 Free article. Clinical Trial.
Progress towards gene therapy for haemophilia B.
Patel N, Reiss U, Davidoff AM, Nathwani AC. Patel N, et al. Int J Hematol. 2014 Apr;99(4):372-6. doi: 10.1007/s12185-014-1523-0. Epub 2014 Feb 6. Int J Hematol. 2014. PMID: 24500177 Free PMC article. Review.
Gene Therapy and Hemophilia: Where Do We Go from Here?
Bolous NS, Bhatt N, Bhakta N, Neufeld EJ, Davidoff AM, Reiss UM. Bolous NS, et al. Among authors: reiss um. J Blood Med. 2022 Oct 6;13:559-580. doi: 10.2147/JBM.S371438. eCollection 2022. J Blood Med. 2022. PMID: 36226233 Free PMC article. Review.
Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural).
Shapiro AD, Ragni MV, Borhany M, Abajas YL, Tarantino MD, Holstein K, Croteau SE, Liesner R, Tarango C, Carvalho M, McGuinn C, Funding E, Kempton CL, Bidlingmaier C, Cohen A, Oldenburg J, Kearney S, Knoll C, Kuriakose P, Acharya S, Reiss UM, Kulkarni R, Witkop M, Lethagen S, Donfield S, LeBeau P, Berntorp E, Astermark J. Shapiro AD, et al. Among authors: reiss um. Haemophilia. 2021 Jan;27(1):49-59. doi: 10.1111/hae.14139. Epub 2020 Dec 5. Haemophilia. 2021. PMID: 33278853
Hemophilia gene therapy-New country initiatives.
Reiss UM, Zhang L, Ohmori T. Reiss UM, et al. Haemophilia. 2021 Feb;27 Suppl 3:132-141. doi: 10.1111/hae.14080. Epub 2020 Jul 7. Haemophilia. 2021. PMID: 32638467 Review.
58 results