Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant
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- Jenny McIntosh
- University College London Cancer Institute, London, United Kingdom;
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- Peter J. Lenting
- Institut National de la Santé et de la Recherche Médicale U770 and Unité Mixte de Recherche Université Paris Sud, Le Kremlin-Bicetre, France;
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- Cecilia Rosales
- University College London Cancer Institute, London, United Kingdom;
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- Doyoung Lee
- University College London Cancer Institute, London, United Kingdom;
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- Samira Rabbanian
- University College London Cancer Institute, London, United Kingdom;
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- Deepak Raj
- University College London Cancer Institute, London, United Kingdom;
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- Nishil Patel
- University College London Cancer Institute, London, United Kingdom;
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- Edward G. D. Tuddenham
- University College London Cancer Institute, London, United Kingdom;
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- Olivier D. Christophe
- Institut National de la Santé et de la Recherche Médicale U770 and Unité Mixte de Recherche Université Paris Sud, Le Kremlin-Bicetre, France;
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- John H. McVey
- Medical Research Council Centre for Transplantation, Kings College, London, United Kingdom;
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- Simon Waddington
- University College London Prenatal Cell and Gene Therapy Group, London, United Kingdom;
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- Arthur W. Nienhuis
- Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, TN;
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- John T. Gray
- Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, TN;
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- Paolo Fagone
- Division of Experimental Hematology, St. Jude Children's Research Hospital, Memphis, TN;
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- Federico Mingozzi
- Center for Cellular and Molecular Therapeutics and
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- Shang-Zhen Zhou
- Center for Cellular and Molecular Therapeutics and
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- Katherine A. High
- Center for Cellular and Molecular Therapeutics and
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- Maria Cancio
- Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN
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- Catherine Y. C. Ng
- Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN
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- Junfang Zhou
- Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN
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- Christopher L. Morton
- Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN
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- Andrew M. Davidoff
- Department of Surgery, St. Jude Children's Research Hospital, Memphis, TN
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- Amit C. Nathwani
- University College London Cancer Institute, London, United Kingdom;
抄録
<jats:title>Key Points</jats:title> <jats:p>Novel, more potent codon-optimized human FVIII variant (codop-hFVIII-V3). Codop-hFVIII-V3 is safe and efficacious in mice and nonhuman primates, thus improving the prospects of gene therapy for hemophilia A.</jats:p>
収録刊行物
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- Blood
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Blood 121 (17), 3335-3344, 2013-04-25
American Society of Hematology