Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice.
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[1] M. Kay,et al. A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. , 2003, Blood.
[2] M. Chuah,et al. Gene therapy for the hemophilias , 2003, Journal of thrombosis and haemostasis : JTH.
[3] L. Naldini,et al. Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells , 2003, Nature Medicine.
[4] I. Miconnet,et al. In vivo administration of a lentiviral vaccine targets DCs and induces efficient CD8(+) T cell responses. , 2003, The Journal of clinical investigation.
[5] I. Verma,et al. Efficient production of human FVIII in hemophilic mice using lentiviral vectors. , 2003, Molecular therapy : the journal of the American Society of Gene Therapy.
[6] Yuqin Wang,et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. , 2003, The Journal of clinical investigation.
[7] Alan McClelland,et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. , 2003, Blood.
[8] D. Collen,et al. Humoral immune response in mice against a circulating antigen induced by adenoviral transfer is strictly dependent on expression in antigen-presenting cells. , 2003, Blood.
[9] S. Kochanek,et al. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. , 2003, Blood.
[10] L. Ailles,et al. Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient. , 2002, Blood.
[11] J. Neefjes,et al. Antigen degradation or presentation by MHC class I molecules via classical and non-classical pathways. , 2002, Molecular immunology.
[12] B. Brown,et al. Dangerous liaisons: the role of "danger" signals in the immune response to gene therapy. , 2002, Blood.
[13] L. Naldini,et al. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. , 2002, Blood.
[14] R. Herzog,et al. Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. , 2002, Human gene therapy.
[15] P. Romero,et al. Efficient transduction of dendritic cells and induction of a T-cell response by third-generation lentivectors. , 2002, Human gene therapy.
[16] M. Kay,et al. A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. , 2002, Blood.
[17] R. Herzog,et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. , 2002, Blood.
[18] P. Matzinger. The Danger Model: A Renewed Sense of Self , 2002, Science.
[19] L. Naldini,et al. Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. , 2002, Human gene therapy.
[20] T. Dull,et al. Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector , 2002, Nature Biotechnology.
[21] D. Skelton,et al. The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice , 2001, Gene Therapy.
[22] M. Denaro,et al. EGFP-transduced EL-4 cells from tumors in C57BL/6 mice , 2001, Gene Therapy.
[23] A. Suhrbier,et al. Cytotoxic T cell polyepitope vaccines delivered by ISCOMs. , 2001, Vaccine.
[24] R. Herzog,et al. Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.
[25] R. Herzog,et al. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.
[26] K. High,et al. Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice , 2001, Gene Therapy.
[27] I. Verma,et al. Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging. , 2001, Molecular therapy : the journal of the American Society of Gene Therapy.
[28] K. High. Gene transfer as an approach to treating hemophilia. , 2001, Circulation research.
[29] Luigi Naldini,et al. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics , 2001, Nature Medicine.
[30] Thomas N. Sato,et al. Universal GFP reporter for the study of vascular development , 2000, Genesis.
[31] M. Kay,et al. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. , 2000, Blood.
[32] H. Ertl,et al. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.
[33] Alan McClelland,et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector , 2000, Nature Genetics.
[34] I. Verma,et al. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. , 2000, Molecular therapy : the journal of the American Society of Gene Therapy.
[35] R. Zinkernagel,et al. Immunological Memory , 2006 .
[36] R. Morgan,et al. Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity. , 1999, Blood.
[37] A. Beaudet,et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. , 1999, Proceedings of the National Academy of Sciences of the United States of America.
[38] Sebastian Amigorena,et al. Selective transport of internalized antigens to the cytosol for MHC class I presentation in dendritic cells , 1999, Nature Cell Biology.
[39] A. Beaudet,et al. Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors. , 1999, Human gene therapy.
[40] D. Kohn,et al. Immune response to green fluorescent protein: implications for gene therapy , 1999, Gene Therapy.
[41] C. Caskey,et al. Expanded-capacity adenoviral vectors--the helper-dependent vectors. , 1999, Molecular medicine today.
[42] Katherine A. High,et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector , 1999, Nature Medicine.
[43] M. Kay,et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors , 1999, Nature Medicine.
[44] James M. Wilson,et al. Transduction of Dendritic Cells by DNA Viral Vectors Directs the Immune Response to Transgene Products in Muscle Fibers , 1998, Journal of Virology.
[45] A. Gown,et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors , 1997, Nature Genetics.
[46] M. Mehtali,et al. Adenovirus-mediated gene transfer: influence of transgene, mouse strain and type of immune response on persistence of transgene expression , 1997, Gene Therapy.
[47] K. Pasi. Gene therapy for haemophilia , 2001, Bailliere's clinical haematology.
[48] M. Kay,et al. Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains. , 1995, Gene therapy.