Search Results - Amit C. Nathwani

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  1. 1
    Gene therapy for hemophilia

    Gene therapy for hemophilia by Amit C. Nathwani

    Published 2019
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  2. 2
    Gene therapy for hemophilia

    Gene therapy for hemophilia by Amit C. Nathwani

    Published 2022
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  3. 3
    Gene Therapy for Hemophilia

    Gene Therapy for Hemophilia by Arthur W. Nienhuis, Amit C. Nathwani, Andrew M. Davidoff

    Published 2017
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  4. 4
    Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

    Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway by Andrew M. Davidoff, Catherine Y. Ng, Junfang Zhou, Yunyu Spence, Amit C. Nathwani

    Published 2003
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  5. 5
    Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein

    Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline... by Patrick Kelly, Jody Vandergriff, Amit C. Nathwani, Arthur W. Nienhuis, Elio F. Vanin

    Published 2000
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  6. 6
    Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods

    Systemic Errors in Quantitative Polymerase Chain Reaction Titration of Self-Complementary Adeno-Associated Viral Vectors and Improved Alternative Methods by Paolo Fagone, J. Fraser Wright, Amit C. Nathwani, Arthur W. Nienhuis, Andrew M. Davidoff, John T. Gray

    Published 2012
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  7. 7
    Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA

    Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA by Amit C. Nathwani, Andrew M. Davidoff, Hideki Hanawa, Junfang Zhou, Elio F. Vanin, Arthur W. Nienhuis

    Published 2001
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  8. 8
    Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

    Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhu... by Andrew M. Davidoff, John T. Gray, Catherine Y. Ng, Youbin Zhang, Junfang Zhou, Yunyu Spence, Yusura Bakar, Amit C. Nathwani

    Published 2005
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  9. 9
    Comparison of Various Envelope Proteins for Their Ability to Pseudotype Lentiviral Vectors and Transduce Primitive Hematopoietic Cells from Human Blood

    Comparison of Various Envelope Proteins for Their Ability to Pseudotype Lentiviral Vectors and Transduce Primitive Hematopoietic Cells from Human Blood by Hideki Hanawa, Patrick Kelly, Amit C. Nathwani, Derek A. Persons, Jody Vandergriff, Phillip W. Hargrove, Elio F. Vanin, Arthur W. Nienhuis

    Published 2002
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  10. 10
    Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy

    Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy by Brandon K. Sack, Sherin N. Merchant, David M. Markusic, Amit C. Nathwani, Andrew M. Davidoff, Barry J. Byrne, Roland W. Herzog

    Published 2012
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  11. 11
    Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

    Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman prima... by Amit C. Nathwani, John T. Gray, Jenny McIntosh, Catherine Y. Ng, Junfang Zhou, Yunyu Spence, Melanie Cochrane, Elaine Gray, Edward G. D. Tuddenham, Andrew M. Davidoff

    Published 2006
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  12. 12
    Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

    Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial by James A. Allay, Susan Sleep, Scott Long, David M. Tillman, Rob Clark, Gael Carney, Paolo Fagone, Jenny H. McIntosh, Arthur W. Nienhuis, Andrew M. Davidoff, Amit C. Nathwani, John T. Gray

    Published 2011
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  13. 13
    Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

    Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman... by Amit C. Nathwani, John T. Gray, Catherine Y. Ng, Junfang Zhou, Yunyu Spence, Simon N. Waddington, Edward G. D. Tuddenham, Geoffrey Kemball‐Cook, Jenny McIntosh, Mariëtte Boon‐Spijker, Koen Mertens, Andrew M. Davidoff

    Published 2005
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  14. 14
    Codon optimization of human factor VIII cDNAs leads to high-level expression

    Codon optimization of human factor VIII cDNAs leads to high-level expression by Natalie J Ward, Suzanne M. K. Buckley, Simon N. Waddington, Thierry VandenDriessche, Marinee Chuah, Amit C. Nathwani, Jenny McIntosh, Edward G. D. Tuddenham, Christine Kinnon, Adrian J. Thrasher, John H. McVey

    Published 2010
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  15. 15
    In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

    In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model by Citra Nurfarah Zaini Mattar, Irene Gil-Fariña, Cecilia Rosales, Nuryanti Johana, Yvonne Tan, Jenny McIntosh, Christine Kaeppel, Simon N. Waddington, Arijit Biswas, Mahesh Choolani, Manfred Schmidt, Amit C. Nathwani, Jerry Kok Yen Chan

    Published 2017
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  16. 16
    The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

    The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective by Nancy S. Bolous, Yichen Chen, Huiqi Wang, Andrew M. Davidoff, Meenakshi Devidas, Timothy W. Jacobs, Michael M. Meagher, Amit C. Nathwani, Ellis J. Neufeld, Bryan A. Piras, Carlos Rodríguez‐Galindo, Ulrike M. Reiss, Nickhill Bhakta

    Published 2021
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  17. 17
    Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

    Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques by Amit C. Nathwani, Andrew M. Davidoff, Hideki Hanawa, Yunyu Hu, Fredric A. Hoffer, Alexander Y. Nikanorov, Clive A. Slaughter, Catherine Y. Ng, Junfang Zhou, Jay N. Lozier, Timothy D. Mandrell, Elio F. Vanin, Arthur W. Nienhuis

    Published 2002
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  18. 18
    GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

    GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant by Amit C. Nathwani, Edward G. D. Tuddenham, Pratima Chowdary, Jenny McIntosh, Doyoung Lee, Cecilia Rosales, Mark Phillips, Jun Pie, Junfang Zhou, Michael M. Meagher, Ulrike M. Reiss, Andrew M. Davidoff, Chris L Morton, Anne Riddell

    Published 2018
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  19. 19
    The Role of Histone Demethylase KDM4B in Myc Signaling in Neuroblastoma

    The Role of Histone Demethylase KDM4B in Myc Signaling in Neuroblastoma by Jun J. Yang, Alaa Altahan, Dongli Hu, Yingdi Wang, Pei-Hsin Cheng, Christopher L. Morton, Chunxu Qu, Amit C. Nathwani, Jason M. Shohet, Theodore Fotsis, Jan Köster, Rogier Versteeg, Hitoshi Okada, Adrian L. Harris, Andrew M. Davidoff

    Published 2015
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  20. 20
    Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B

    Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B by Pratima Chowdary, Susan Shapiro, Michael Makris, Gillian Evans, Sara Boyce, Kate Talks, Gerard Dolan, Ulrike M. Reiss, Mark Phillips, Anne Riddell, Maria R. Peralta, Michelle Quaye, David Patch, Edward G. D. Tuddenham, Allison Dane, Marie Watissée, Alison Long, Amit C. Nathwani

    Published 2022
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