Immune responses to AAV in clinical trials.
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[1] J. F. Wright,et al. Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. , 2007, Blood.
[2] C. Miao. Recent advances in immune modulation. , 2007, Current gene therapy.
[3] R. Mandel,et al. Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the cell biology of neuroimmune interactions. , 2007, Current gene therapy.
[4] Thomas Ferkol,et al. Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial. , 2007, Human gene therapy.
[5] R. Herzog,et al. Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer. , 2007, Blood.
[6] Aarati R. Ranade,et al. Robust expansion of human hepatocytes in Fah−/−/Rag2−/−/Il2rg−/− mice , 2007, Nature Biotechnology.
[7] D. DiMichele. Inhibitor development in haemophilia B: an orphan disease in need of attention , 2007, British journal of haematology.
[8] R. Samulski,et al. Adeno-Associated Virus Type 2 (AAV2) Capsid-Specific Cytotoxic T Lymphocytes Eliminate Only Vector-Transduced Cells Coexpressing the AAV2 Capsid In Vivo , 2007, Journal of Virology.
[9] Lili Wang,et al. Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets. , 2007, Human gene therapy.
[10] Z. Xiang,et al. Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[11] Marcela V Maus,et al. CD8+ T-cell responses to adeno-associated virus capsid in humans , 2007, Nature Medicine.
[12] J. F. Wright,et al. Separation of adeno-associated virus type 2 empty particles from genome containing vectors by anion-exchange column chromatography. , 2007, Journal of virological methods.
[13] J. Gray,et al. 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. , 2007, Blood.
[14] J. Wilson,et al. Efficacy and safety of adeno‐associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy , 2006, Journal of thrombosis and haemostasis : JTH.
[15] Sihong Song,et al. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. , 2006, Human gene therapy.
[16] J. Sommer,et al. Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy. , 2006, Blood.
[17] L. Naldini,et al. Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice. , 2006, The Journal of clinical investigation.
[18] Nicki Panoskaltsis,et al. Cytokine storm in a phase 1 trial of the anti-CD28 monoclonal antibody TGN1412. , 2006, The New England journal of medicine.
[19] M. Ozelo,et al. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[20] S. Akira,et al. Immunoprivileged status of the liver is controlled by Toll-like receptor 3 signaling. , 2006, The Journal of clinical investigation.
[21] Ravi A. Desai,et al. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid , 2006, Nature Medicine.
[22] L. Couto,et al. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. , 2006, Blood.
[23] R. Hebbel,et al. Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor. , 2006, Blood.
[24] J. Sommer,et al. Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[25] F. Bosch,et al. Reversal of Type 1 Diabetes by Engineering a Glucose Sensor in Skeletal Muscle , 2006, Diabetes.
[26] A. Varki,et al. Loss of Siglec expression on T lymphocytes during human evolution , 2006, Proceedings of the National Academy of Sciences of the United States of America.
[27] L. Naldini,et al. Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer , 2006, Nature Medicine.
[28] R. Herzog,et al. Treatment of human disease by adeno-associated viral gene transfer , 2006, Human Genetics.
[29] C. Norbury. Drinking a lot is good for dendritic cells , 2006, Immunology.
[30] J. Emerson,et al. Prevalence of neutralizing antibodies against adeno-associated virus (AAV) types 2, 5, and 6 in cystic fibrosis and normal populations: Implications for gene therapy using AAV vectors. , 2006, Human gene therapy.
[31] Geoffrey Kemball-Cook,et al. 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. , 2006, Blood.
[32] J. Rasko,et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response , 2006, Nature Medicine.
[33] D. Schaffer,et al. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors , 2006, Nature Biotechnology.
[34] Wenman Wu,et al. Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia. , 2006, Human gene therapy.
[35] A. Salvetti,et al. Evidence for encapsidation of prokaryotic sequences during recombinant adeno-associated virus production and their in vivo persistence after vector delivery. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.
[36] T. Flotte. Recent developments in recombinant AAV-mediated gene therapy for lung diseases. , 2005, Current gene therapy.
[37] P. Tak,et al. Enhanced gene transfer to arthritic joints using adeno-associated virus type 5: implications for intra-articular gene therapy , 2005, Annals of the rheumatic diseases.
[38] H. Stedman,et al. Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. , 2005, Blood.
[39] Luis Garcia,et al. Rescue of Dystrophic Muscle Through U7 snRNA-Mediated Exon Skipping , 2004, Science.
[40] R. Herzog,et al. Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector. , 2004, Human gene therapy.
[41] R. Herzog,et al. Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer. , 2004, Blood.
[42] A. Annoni,et al. Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. , 2004, Blood.
[43] Cameron S. Osborne,et al. LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1 , 2003, Science.
[44] 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.
[45] Alan McClelland,et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. , 2003, Blood.
[46] K. High. The risks of germline gene transfer. , 2003, The Hastings Center report.
[47] R. Herzog,et al. Improved Hepatic Gene Transfer by Using an Adeno-Associated Virus Serotype 5 Vector , 2002, Journal of Virology.
[48] G. Aguirre,et al. Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs , 2002, Proceedings of the National Academy of Sciences of the United States of America.
[49] Lili Wang,et al. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy , 2002, Proceedings of the National Academy of Sciences of the United States of America.
[50] Lung-Ji Chang,et al. RNA 3′ Readthrough of Oncoretrovirus and Lentivirus: Implications for Vector Safety and Efficacy , 2002, Journal of Virology.
[51] A. Mortellaro,et al. Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning , 2002, Science.
[52] A. Fischer,et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. , 2002, The New England journal of medicine.
[53] R. Herzog,et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. , 2002, Blood.
[54] N. Boyce. Trial halted after gene shows up in semen , 2001, Nature.
[55] K. High. AAV‐Mediated Gene Transfer for Hemophilia , 2001, Annals of the New York Academy of Sciences.
[56] D. McCarty,et al. Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis , 2001, Gene Therapy.
[57] Theresa A. Storm,et al. Extrachromosomal Recombinant Adeno-Associated Virus Vector Genomes Are Primarily Responsible for Stable Liver Transduction In Vivo , 2001, Journal of Virology.
[58] Jean Bennett,et al. Gene therapy restores vision in a canine model of childhood blindness , 2001, Nature Genetics.
[59] 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.
[60] J. Schlehofer,et al. Update on the prevalence of serum antibodies (IgG and IgM) to adeno‐associated virus (AAV) , 1999, Journal of medical virology.
[61] K. Propert,et al. Immune responses to adenovirus and adeno-associated virus in humans , 1999, Gene Therapy.
[62] T. Flotte,et al. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. , 1998, Proceedings of the National Academy of Sciences of the United States of America.
[63] Prerna Sharma,et al. Circular Intermediates of Recombinant Adeno-Associated Virus Have Defined Structural Characteristics Responsible for Long-Term Episomal Persistence in Muscle Tissue , 1998, Journal of Virology.
[64] L. Villarreal,et al. Adeno-associated virus vectors can be efficiently produced without helper virus , 1998, Gene Therapy.
[65] 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.
[66] James M. Wilson,et al. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. , 1997, Proceedings of the National Academy of Sciences of the United States of America.
[67] T. Palmer,et al. Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B. , 1989, Blood.
[68] R. Samulski,et al. Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells. , 1982, Proceedings of the National Academy of Sciences of the United States of America.
[69] D. Duan,et al. Efficient Whole-body Transduction with Trans-splicing Adeno-associated Viral Vectors. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[70] R. Herzog,et al. Posttranslational modifications of recombinant myotube-synthesized human factor IX. , 2001, Blood.
[71] 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.