Virus-mediated gene delivery for human gene therapy.
暂无分享,去创建一个
[1] R. Samulski,et al. Infectious Entry Pathway of Adeno-Associated Virus and Adeno-Associated Virus Vectors , 2000, Journal of Virology.
[2] M. Kay,et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors , 1999, Nature Medicine.
[3] T. VandenDriessche,et al. Clinical gene transfer studies for hemophilia A. , 2004, Seminars in thrombosis and hemostasis.
[4] A. Fattaey,et al. An Adenovirus Mutant That Replicates Selectively in p53- Deficient Human Tumor Cells , 1996, Science.
[5] H. Kaufman,et al. OPTIM trial: a Phase III trial of an oncolytic herpes virus encoding GM-CSF for unresectable stage III or IV melanoma. , 2010, Future oncology.
[6] J. Schlehofer,et al. Update on the prevalence of serum antibodies (IgG and IgM) to adeno‐associated virus (AAV) , 1999, Journal of medical virology.
[7] E Marshall,et al. Gene Therapy Death Prompts Review of Adenovirus Vector , 1999, Science.
[8] N. Brunetti‐Pierri,et al. Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors , 2008, Gene Therapy.
[9] Christof von Kalle,et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. , 2010, The New England journal of medicine.
[10] W. Russell,et al. Adenoviruses: update on structure and function. , 2009, The Journal of general virology.
[11] 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.
[12] F. Gage,et al. In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector , 1996, Science.
[13] E. Galanis,et al. Clinical trial results with oncolytic virotherapy: a century of promise, a decade of progress , 2007, Nature Clinical Practice Oncology.
[14] Keyun Qing,et al. Adeno-associated virus 2 co-receptors?-first reply , 1999, Nature Medicine.
[15] M. Weitzman,et al. Processing of recombinant AAV genomes occurs in specific nuclear structures that overlap with foci of DNA-damage-response proteins , 2008, Journal of Cell Science.
[16] R. Kotin,et al. Adeno-associated virus type 5: transduction efficiency and cell-type specificity in the primate retina. , 2003, Human gene therapy.
[17] M. Gulley,et al. A phase I study of Onyx-015, an E1B attenuated adenovirus, administered intratumorally to patients with recurrent head and neck cancer. , 2000, Clinical cancer research : an official journal of the American Association for Cancer Research.
[18] Luca Biasco,et al. Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. , 2007, The Journal of clinical investigation.
[19] J. Burns,et al. Generation of high-titer pseudotyped retroviral vectors with very broad host range. , 1994, Methods in cell biology.
[20] J. Agudo,et al. In vivo genetic engineering of murine pancreatic beta cells mediated by single-stranded adeno-associated viral vectors of serotypes 6, 8 and 9 , 2011, Diabetologia.
[21] R. Kotin,et al. Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles , 1997, Journal of virology.
[22] A. Epstein. Progress and prospects: Biological properties and technological advances of herpes simplex virus type 1-based amplicon vectors , 2009, Gene Therapy.
[23] N. Mazarakis,et al. Enhanced pseudotyping efficiency of HIV-1 lentiviral vectors by a rabies/vesicular stomatitis virus chimeric envelope glycoprotein , 2011, Gene Therapy.
[24] K. High,et al. Immune responses to AAV in clinical trials. , 2007, Current gene therapy.
[25] A. Epstein,et al. HSV as a vector in vaccine development and gene therapy. , 2008, Advances in experimental medicine and biology.
[26] M. Imperiale,et al. Production of first generation adenovirus vectors: a review , 2000, Gene Therapy.
[27] R. Samulski,et al. αVβ5 integrin: a co-receptor for adeno-associated virus type 2 infection , 1999, Nature Medicine.
[28] M. Yudkoff,et al. Recombinant adenovirus gene transfer in adults with partial ornithine transcarbamylase deficiency (OTCD). , 1999, Human gene therapy.
[29] Alessandro Aiuti,et al. Hot spots of retroviral integration in human CD34+ hematopoietic cells. , 2007, Blood.
[30] A. Epstein,et al. Efficient and non-toxic gene transfer to cardiomyocytes using novel generation amplicon vectors derived from HSV-1. , 2005, Journal of molecular and cellular cardiology.
[31] J. Petrini,et al. Rad50 Is Dispensable for the Maintenance and Viability of Postmitotic Tissues , 2008, Molecular and Cellular Biology.
[32] Alessandro Guffanti,et al. High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors. , 2010, Blood.
[33] D. Muruve,et al. The innate immune response to adenovirus vectors. , 2004, Human gene therapy.
[34] Cameron S. Osborne,et al. LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1 , 2003, Science.
[35] M. Hallek,et al. AAV-based gene transfer. , 2003, Current opinion in molecular therapeutics.
[36] A. Miller,et al. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection , 1990, Molecular and cellular biology.
[37] Theresa A. Storm,et al. Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[38] Kazuto Kobayashi,et al. Neuron-specific gene transfer through retrograde transport of lentiviral vector pseudotyped with a novel type of fusion envelope glycoprotein. , 2011, Human gene therapy.
[39] R. Samulski,et al. Cross-Dressing the Virion: the Transcapsidation of Adeno-Associated Virus Serotypes Functionally Defines Subgroups , 2004, Journal of Virology.
[40] Nick Tyler,et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. , 2008, The New England journal of medicine.
[41] Yan Cui,et al. Lentiviral vectors with CMV or MHCII promoters administered in vivo: immune reactivity versus persistence of expression. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[42] C. Kao,et al. Gene Therapy for Prostate Cancer by Controlling Adenovirus E1a and E4 Gene Expression with PSES Enhancer , 2005 .
[43] J. Pessin,et al. Dynamin Is Required for Recombinant Adeno-Associated Virus Type 2 Infection , 1999, Journal of Virology.
[44] R. Friedman. Expression of human adenosine deaminase using a transmissable murine retrovirus vector system. , 1985, Proceedings of the National Academy of Sciences of the United States of America.
[45] M. Weitzman,et al. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis , 1996, Journal of virology.
[46] R. Daniel,et al. Integration site selection by retroviral vectors: molecular mechanism and clinical consequences. , 2008, Human gene therapy.
[47] Youichi Suzuki,et al. The road to chromatin — nuclear entry of retroviruses , 2007, Nature Reviews Microbiology.
[48] Qiang Liu,et al. Molecular basis of the inflammatory response to adenovirus vectors , 2003, Gene Therapy.
[49] S. Snyder,et al. Increased apoptosis of Huntington disease lymphoblasts associated with repeat length-dependent mitochondrial depolarization , 1999, Nature Medicine.
[50] B. Hub,et al. Endocytosis of Adeno-Associated Virus Type 5 Leads to Accumulation of Virus Particles in the Golgi Compartment , 2002, Journal of Virology.
[51] K. High,et al. AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects , 2008, Gene Therapy.
[52] D. Sze,et al. Phase I/II study of oncolytic herpes simplex virus NV1020 in patients with extensively pretreated refractory colorectal cancer metastatic to the liver. , 2010, Human gene therapy.
[53] U. Dietrich,et al. Pseudotyping Vesicular Stomatitis Virus with Lymphocytic Choriomeningitis Virus Glycoproteins Enhances Infectivity for Glioma Cells and Minimizes Neurotropism , 2011, Journal of Virology.
[54] M. Chillón,et al. Gutless adenovirus: last-generation adenovirus for gene therapy , 2005, Gene Therapy.
[55] M. Kay,et al. The 37/67-Kilodalton Laminin Receptor Is a Receptor for Adeno-Associated Virus Serotypes 8, 2, 3, and 9 , 2006, Journal of Virology.
[56] D. Descamps,et al. Two key challenges for effective adenovirus-mediated liver gene therapy: innate immune responses and hepatocyte-specific transduction. , 2009, Current gene therapy.
[57] M. Giacca,et al. Integration site selection by retroviruses. , 2004, AIDS reviews.
[58] J. Simons,et al. Prostate attenuated replication competent adenovirus (ARCA) CN706: a selective cytotoxic for prostate-specific antigen-positive prostate cancer cells. , 1997, Cancer research.
[59] H. Yang,et al. Dendritic cell-directed lentivector vaccine induces antigen-specific immune responses against murine melanoma , 2011, Cancer Gene Therapy.
[60] D. Kirn,et al. Gene therapy progress and prospects cancer: oncolytic viruses , 2008, Gene Therapy.
[61] M. Sadelain,et al. The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[62] P. Kaleebu,et al. Adenovirus-Based Vaccines: Comparison of Vectors from Three Species of Adenoviridae , 2010, Journal of Virology.
[63] Manfred Schmidt,et al. Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy , 2009, Science.
[64] H. Wakimoto,et al. Oncolytic herpes simplex virus vectors and chemotherapy: are combinatorial strategies more effective for cancer? , 2010, Future oncology.
[65] 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.
[66] R. Ali,et al. Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising , 2008, Gene Therapy.
[67] R. Linden,et al. Adeno-associated virus site-specifically integrates into a muscle-specific DNA region. , 2000, Proceedings of the National Academy of Sciences of the United States of America.
[68] J. Glorioso,et al. Herpes vector-mediated gene transfer in treatment of diseases of the nervous system. , 2004, Annual review of microbiology.
[69] Macgregor Rr. Clinical protocol. A phase 1 open-label clinical trial of the safety and tolerability of single escalating doses of autologous CD4 T cells transduced with VRX496 in HIV-positive subjects. , 2001 .
[70] L. Zentilin,et al. Involvement of Cellular Double-Stranded DNA Break Binding Proteins in Processing of the Recombinant Adeno-Associated Virus Genome , 2001, Journal of Virology.
[71] Gianluigi Zanetti,et al. Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection. , 2011, Blood.
[72] J. Couzin-Frankel. Genetics. The promise of a cure: 20 years and counting. , 2009, Science.
[73] Roberto Cattaneo,et al. Reprogrammed viruses as cancer therapeutics: targeted, armed and shielded , 2008, Nature Reviews Microbiology.
[74] K. Nakano,et al. HSV trafficking and development of gene therapy vectors with applications in the nervous system , 2005, Gene Therapy.
[75] E. Lehtonen,et al. Evaluation of risks related to the use of adeno-associated virus-based vectors. , 2003, Current gene therapy.
[76] G. Trobridge. Genotoxicity of retroviral hematopoietic stem cell gene therapy , 2011, Expert opinion on biological therapy.
[77] T. Samulski,et al. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors , 1996, Journal of virology.
[78] A. Schambach,et al. Clinical application of lentiviral vectors - concepts and practice. , 2008, Current gene therapy.
[79] B. Davidson,et al. What does it take to bind CAR? , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.
[80] J. Cody,et al. Armed replicating adenoviruses for cancer virotherapy , 2009, Cancer Gene Therapy.
[81] Jérôme Larghero,et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia , 2010, Nature.
[82] J. Kornegay,et al. Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.
[83] Y. Chérel,et al. Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery. , 2003, Molecular therapy : the journal of the American Society of Gene Therapy.
[84] Pin Wang,et al. Pseudotyping lentiviral vectors with aura virus envelope glycoproteins for DC-SIGN-mediated transduction of dendritic cells. , 2011, Human gene therapy.
[85] R. Linden,et al. The cryptic life style of adenoassociated virus , 1995, BioEssays : news and reviews in molecular, cellular and developmental biology.
[86] N. Bessis,et al. Immune responses to gene therapy vectors: influence on vector function and effector mechanisms , 2004, Gene therapy.
[87] J. Glorioso,et al. Replication-defective genomic HSV gene therapy vectors: design, production and CNS applications. , 2005, Current opinion in molecular therapeutics.
[88] R. Ali,et al. AAV-mediated gene therapy for retinal disorders: from mouse to man , 2008, Gene Therapy.
[89] S. Bhide,et al. Phase I/II Study of Oncolytic HSVGM-CSF in Combination with Radiotherapy and Cisplatin in Untreated Stage III/IV Squamous Cell Cancer of the Head and Neck , 2010, Clinical Cancer Research.
[90] C. Aiken. Pseudotyping human immunodeficiency virus type 1 (HIV-1) by the glycoprotein of vesicular stomatitis virus targets HIV-1 entry to an endocytic pathway and suppresses both the requirement for Nef and the sensitivity to cyclosporin A , 1997, Journal of virology.
[91] A. Miller. Retrovirus packaging cells. , 1990, Human gene therapy.
[92] R. Kotin,et al. Characterization of a preferred site on human chromosome 19q for integration of adeno‐associated virus DNA by non‐homologous recombination. , 1992, The EMBO journal.
[93] R. Samulski,et al. Adeno-associated virus serotypes: vector toolkit for human gene therapy. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[94] T. Rabbitts,et al. Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency. , 2004, The New England journal of medicine.
[95] D. Nettelbeck,et al. Novel oncolytic adenoviruses targeted to melanoma: specific viral replication and cytolysis by expression of E1A mutants from the tyrosinase enhancer/promoter. , 2002, Cancer research.
[96] I. Tannock,et al. A controlled trial of intratumoral ONYX-015, a selectively-replicating adenovirus, in combination with cisplatin and 5-fluorouracil in patients with recurrent head and neck cancer , 2000, Nature Medicine.
[97] Hoguen Kim,et al. Ad-mTERT-delta19, a conditional replication-competent adenovirus driven by the human telomerase promoter, selectively replicates in and elicits cytopathic effect in a cancer cell-specific manner. , 2003, Human gene therapy.
[98] W. Yung,et al. Δ24-hyCD adenovirus suppresses glioma growth in vivo by combining oncolysis and chemosensitization , 2005, Cancer Gene Therapy.
[99] M. Gulley,et al. Erratum: A phase I study of Onyx-015, an E1B attenuated adenovirus, administered intratumorally to patients with recurrent head and neck cancer (Clinical Cancer Research (2000) 6 (798-806)) , 2000 .
[100] A. Epstein,et al. HSV-1-derived helper-independent defective vectors, replicating vectors and amplicon vectors, for the treatment of brain diseases. , 2010, Current opinion in drug discovery & development.
[101] J. Huard,et al. Gene transfer to skeletal muscle using herpes simplex virus-based vectors. , 2004, Methods in molecular biology.
[102] Lung-Ji Chang,et al. Efficient and persistent transduction of exocrine and endocrine pancreas by adeno-associated virus type 8. , 2007, Journal of biomedical science.
[103] Christof von Kalle,et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. , 2003, The New England journal of medicine.
[104] Yang Du,et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 , 2006, Nature Medicine.
[105] S. W. Kim,et al. Evolution of oncolytic adenovirus for cancer treatment. , 2012, Advanced drug delivery reviews.
[106] R. H. Smith,et al. Adeno-associated virus integration: virus versus vector , 2008, Gene Therapy.
[107] D. Grimm,et al. Novel tools for production and purification of recombinant adenoassociated virus vectors. , 1998, Human gene therapy.
[108] Alessandro Aiuti,et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. , 2009, The New England journal of medicine.
[109] R. Samulski,et al. Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair , 2010, Gene Therapy.
[110] P. Sinn,et al. Gene Therapy Progress and Prospects: Development of improved lentiviral and retroviral vectors – design, biosafety, and production , 2005, Gene Therapy.
[111] Theresa A. Storm,et al. The host response to adenovirus, helper-dependent adenovirus, and adeno-associated virus in mouse liver. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.
[112] O. Merten,et al. Adeno-associated viruses. , 2011, Methods in molecular biology.
[113] Sally Lehrman,et al. Virus treatment questioned after gene therapy death , 1999, Nature.
[114] 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.
[115] W. Xiao,et al. Characterization of Tissue Tropism Determinants of Adeno-Associated Virus Type 1 , 2003, Journal of Virology.
[116] D. Trono,et al. Self-Inactivating Lentivirus Vector for Safe and Efficient In Vivo Gene Delivery , 1998, Journal of Virology.
[117] M. Finer,et al. Second–generation adenovirus vectors , 1996, Nature Medicine.
[118] Edward B. Miller,et al. α2,3 and α2,6 N-Linked Sialic Acids Facilitate Efficient Binding and Transduction by Adeno-Associated Virus Types 1 and 6 , 2006, Journal of Virology.
[119] Heartening results: the CUPID gene therapy trial for heart failure. , 2011, Molecular therapy : the journal of the American Society of Gene Therapy.
[120] N. Copeland,et al. Gene Therapy Insertional Mutagenesis Insights , 2004, Science.
[121] M. Sadelain,et al. Occurrence of leukaemia following gene therapy of X-linked SCID , 2003, Nature Reviews Cancer.
[122] D. Mccarty. Self-complementary AAV vectors; advances and applications. , 2008, Molecular therapy : the journal of the American Society of Gene Therapy.
[123] F. Bushman,et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. , 2008, The Journal of clinical investigation.
[124] M. Rudnicki,et al. A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal. , 1996, Proceedings of the National Academy of Sciences of the United States of America.
[125] Alan McClelland,et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. , 2003, Blood.
[126] R. Buckley. Gene therapy for SCID—a complication after remarkable progress , 2002, The Lancet.
[127] B. Levine,et al. Regulatory considerations for novel gene therapy products: a review of the process leading to the first clinical lentiviral vector. , 2005, Human gene therapy.
[128] S. Bernasconi,et al. A human immunodeficiency virus type 1 pol gene-derived sequence (cPPT/CTS) increases the efficiency of transduction of human nondividing monocytes and T lymphocytes by lentiviral vectors. , 2002, Human gene therapy.
[129] K. Propert,et al. Immune responses to adenovirus and adeno-associated virus in humans , 1999, Gene Therapy.
[130] A. Epstein,et al. HSV-1-derived recombinant and amplicon vectors for gene transfer and gene therapy. , 2005, Current gene therapy.
[131] R. Manservigi,et al. Replication-competent herpes simplex vectors: design and applications , 2005, Gene Therapy.
[132] R. Linden,et al. The recombination signals for adeno-associated virus site-specific integration. , 1996, Proceedings of the National Academy of Sciences of the United States of America.
[133] L. Zentilin,et al. Adeno-associated virus vectors: versatile tools for in vivo gene transfer. , 2008, Contributions to nephrology.
[134] M. Simonatto,et al. DNA damage and cellular differentiation: More questions than responses , 2007, Journal of cellular physiology.