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World Federation of Hemophilia Gene Therapy Registry
dc.contributor.authorKonkle, Barbara A.
dc.contributor.authorCoffin, Donna
dc.contributor.authorPierce, Glenn F.
dc.contributor.authorClark, Cary
dc.contributor.authorGeorge, Lindsey
dc.contributor.authorIorio, Alfonso
dc.contributor.authorMahlangu, Johnny
dc.contributor.authorNaccache, Mayss
dc.contributor.authorO’mahony, Brian
dc.contributor.authorPeyvandi, Flora
dc.contributor.authorPipe, Steve
dc.contributor.authorQuartel, Adrian
dc.contributor.authorSawyer, Eileen K.
dc.contributor.authorSkinner, Mark W.
dc.contributor.authorTortella, Bartholomew
dc.contributor.authorWatson, Crystal
dc.contributor.authorWinburn, Ian
dc.date.accessioned2020-08-10T20:52:53Z
dc.date.availableWITHHELD_12_MONTHS
dc.date.available2020-08-10T20:52:53Z
dc.date.issued2020-07
dc.identifier.citationKonkle, Barbara A.; Coffin, Donna; Pierce, Glenn F.; Clark, Cary; George, Lindsey; Iorio, Alfonso; Mahlangu, Johnny; Naccache, Mayss; O’mahony, Brian ; Peyvandi, Flora; Pipe, Steve; Quartel, Adrian; Sawyer, Eileen K.; Skinner, Mark W.; Tortella, Bartholomew; Watson, Crystal; Winburn, Ian (2020). "World Federation of Hemophilia Gene Therapy Registry." Haemophilia 26(4): 563-564.
dc.identifier.issn1351-8216
dc.identifier.issn1365-2516
dc.identifier.urihttps://hdl.handle.net/2027.42/156126
dc.publisherWiley Periodicals, Inc.
dc.titleWorld Federation of Hemophilia Gene Therapy Registry
dc.typeArticle
dc.rights.robotsIndexNoFollow
dc.subject.hlbsecondlevelOncology and Hematology
dc.subject.hlbtoplevelHealth Sciences
dc.description.peerreviewedPeer Reviewed
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/156126/2/hae14015_am.pdfen_US
dc.description.bitstreamurlhttp://deepblue.lib.umich.edu/bitstream/2027.42/156126/1/hae14015.pdfen_US
dc.identifier.doi10.1111/hae.14015
dc.identifier.sourceHaemophilia
dc.identifier.citedreferenceButterfield JSS, Hege KM, Herzog RW, Kaczmarek R. A Molecular Revolution in the Treatment of Hemophilia [published online ahead of print, 2019 Nov 13]. Mol Ther. 2019; S1525- 0016 ( 19 ): 30502 - 30507.
dc.identifier.citedreferencePeyvandi F, Garagiola I. Clinical advances in gene therapy updates on clinical trials of gene therapy in haemophilia. Haemophilia. 2019; 25: 738 - 746.
dc.identifier.citedreferenceWood WI, Capon DJ, Simonsen CC, et al. Expression of active human factor VIII from recombinant DNA clones. Nature. 1984; 312: 330 - 337.
dc.identifier.citedreferenceGitschier J, Wood WI, Goralka TM, et al. Characterization of the human factor VIII gene. Nature. 1984; 312: 326 - 330.
dc.identifier.citedreferenceNathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Prog. 2019; 6: 1 - 8.
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dc.identifier.citedreferencePierce GF, Coffin D, Members of the WFH Gene Therapy Round Table Program Committee and Organizing Committee. The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world. Haemophilia. 2019; 25: 189 - 194.
dc.identifier.citedreferenceGeorge LA, Sullivan SK, Rasko JEJ, et al. Efficacy and safety in 15 Hemophilia b patients treated with the AAV gene therapy vector Fidanacogene Elaparvovec and followed for at least 1 Year. Blood. 2019; 134 ( Supplement_1 ): 3347.
dc.identifier.citedreferenceNguyen GNEJ, Raymond H, Kafle S, et al. Long- term AAV- mediated factor viii expression in nine hemophilia a dogs: A 10 year follow- up analysis on durability, safety and vector integration. Blood. 2019; 134 ( Supplement_1 ): 611.
dc.identifier.citedreferenceNathwani AC, Reiss UM, Tuddenham EG, et al. Long- term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014; 371 ( 21 ): 1994 - 2004.
dc.identifier.citedreferencePasi KJ, Rangarajan S, Mitchell N, et al. Multiyear Follow- up of AAV5- hFVIII- SQ Gene Therapy for Hemophilia A. N Engl J Med. 2020; 382 ( 1 ): 29 - 40.
dc.identifier.citedreferenceVehar GA, Keyt B, Eaton D, et al. Structure of human factor VIII. Nature. 1984; 312: 337 - 342.
dc.identifier.citedreferenceUS Food and Drug Administration. Long Term Follow- Up After Administration of Human Gene Therapy Products. Available at: https://www.fda.gov/vaccines- blood- biologics/guidance- compliance- regulatory- information- biologics/biologics- guidances. Accessed March 3, 2020.
dc.identifier.citedreferenceHemophilia gene therapy Phase 3 clinical trials. Clinicaltrials.gov. Sourced February 19, 2020.
dc.identifier.citedreferencePierce GF, Iorio A. Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes. Haemophilia. 2018; 24: 60 - 67.
dc.identifier.citedreferenceAnson DS, Choo KH, Rees DJ, et al. The gene structure of human anti- haemophilic factor IX. EMBO J. 1984; 3: 1053 - 1060.
dc.identifier.citedreferenceChoo KH, Gould KG, Ress DJ, Brownlee GG. Molecular cloning of the gene for human anti- haemophilia factor IX. Nature. 1982; 299: 178 - 180.
dc.owningcollnameInterdisciplinary and Peer-Reviewed


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