lentivirus

Summary

Summary: A genus of the family RETROVIRIDAE consisting of non-oncogenic retroviruses that produce multi-organ diseases characterized by long incubation periods and persistent infection. Lentiviruses are unique in that they contain open reading frames (ORFs) between the pol and env genes and in the 3' env region. Five serogroups are recognized, reflecting the mammalian hosts with which they are associated. HIV-1 is the type species.

Top Publications

  1. pmc A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors
    Fang Zhang
    Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, University College London, London, UK
    Mol Ther 18:1640-9. 2010
  2. ncbi Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors
    Carlos Lois
    Division of Biology, California Institute of Technology, Pasadena, CA 91125, USA
    Science 295:868-72. 2002
  3. pmc Transgenesis by lentiviral vectors: lack of gene silencing in mammalian embryonic stem cells and preimplantation embryos
    Alexander Pfeifer
    The Salk Institute for Biological Studies, La Jolla, CA 92037, USA
    Proc Natl Acad Sci U S A 99:2140-5. 2002
  4. ncbi Induced pluripotent stem cell generation using a single lentiviral stem cell cassette
    Cesar A Sommer
    Department of Medicine, Boston University School of Medicine, Massachusetts 02118, USA
    Stem Cells 27:543-9. 2009
  5. pmc A third-generation lentivirus vector with a conditional packaging system
    T Dull
    Cell Genesys, Foster City, California 94404, USA
    J Virol 72:8463-71. 1998
  6. pmc Lentivirus-delivered stable gene silencing by RNAi in primary cells
    Sheila A Stewart
    Whitehead Institute for Biomedical Research, Cambridge, Massachusetts 02142, USA
    RNA 9:493-501. 2003
  7. ncbi High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter
    Christophe Demaison
    Molecular Immunology Unit, Institute of Child Health, UCL, 30 Guilford Street, London, WC1N 1EH, UK
    Hum Gene Ther 13:803-13. 2002
  8. ncbi Production and purification of lentiviral vectors
    Gustavo Tiscornia
    The Salk Institute for Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, California 92037, USA
    Nat Protoc 1:241-5. 2006
  9. ncbi A lentiviral RNAi library for human and mouse genes applied to an arrayed viral high-content screen
    Jason Moffat
    Broad Institute of MIT and Harvard, Cambridge, MA 02139, USA
    Cell 124:1283-98. 2006
  10. ncbi Effective gene therapy with nonintegrating lentiviral vectors
    Rafael J Yáñez-Muñoz
    Molecular Immunology Unit, Institute of Child Health, University College London, 30 Guilford Street, London WC1N 1EH, UK
    Nat Med 12:348-53. 2006

Detail Information

Publications337 found, 100 shown here

  1. pmc A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors
    Fang Zhang
    Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, University College London, London, UK
    Mol Ther 18:1640-9. 2010
    ..The A2UCOE therefore has considerable utility for gene therapy applications where reliable and sustained gene expression is desirable...
  2. ncbi Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors
    Carlos Lois
    Division of Biology, California Institute of Technology, Pasadena, CA 91125, USA
    Science 295:868-72. 2002
    ..We have also generated transgenic rats that express GFP at high levels, suggesting that this technique can be used to produce other transgenic animal species...
  3. pmc Transgenesis by lentiviral vectors: lack of gene silencing in mammalian embryonic stem cells and preimplantation embryos
    Alexander Pfeifer
    The Salk Institute for Biological Studies, La Jolla, CA 92037, USA
    Proc Natl Acad Sci U S A 99:2140-5. 2002
    ..Ability to transfer foreign genes into human ES cells has potential relevance for the development of gene and cell-based therapies...
  4. ncbi Induced pluripotent stem cell generation using a single lentiviral stem cell cassette
    Cesar A Sommer
    Department of Medicine, Boston University School of Medicine, Massachusetts 02118, USA
    Stem Cells 27:543-9. 2009
    ..The use of a single lentiviral vector for reprogramming represents a powerful laboratory tool and a significant step toward the application of iPS technology for clinical purposes...
  5. pmc A third-generation lentivirus vector with a conditional packaging system
    T Dull
    Cell Genesys, Foster City, California 94404, USA
    J Virol 72:8463-71. 1998
    ..However, their biosafety is of major concern. Here we exploit the complexity of the HIV genome to provide lentivirus vectors with novel biosafety features...
  6. pmc Lentivirus-delivered stable gene silencing by RNAi in primary cells
    Sheila A Stewart
    Whitehead Institute for Biomedical Research, Cambridge, Massachusetts 02142, USA
    RNA 9:493-501. 2003
    ..expressing hairpin RNA targeting green fluorescent protein (GFP) using Moloney leukemia virus-based and lentivirus-based retroviral vectors...
  7. ncbi High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter
    Christophe Demaison
    Molecular Immunology Unit, Institute of Child Health, UCL, 30 Guilford Street, London, WC1N 1EH, UK
    Hum Gene Ther 13:803-13. 2002
    ....
  8. ncbi Production and purification of lentiviral vectors
    Gustavo Tiscornia
    The Salk Institute for Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, California 92037, USA
    Nat Protoc 1:241-5. 2006
    ....
  9. ncbi A lentiviral RNAi library for human and mouse genes applied to an arrayed viral high-content screen
    Jason Moffat
    Broad Institute of MIT and Harvard, Cambridge, MA 02139, USA
    Cell 124:1283-98. 2006
    ..This work provides a widely applicable resource for loss-of-function screens, as well as a roadmap for its application to biological discovery...
  10. ncbi Effective gene therapy with nonintegrating lentiviral vectors
    Rafael J Yáñez-Muñoz
    Molecular Immunology Unit, Institute of Child Health, University College London, 30 Guilford Street, London WC1N 1EH, UK
    Nat Med 12:348-53. 2006
    ..For therapeutic application to postmitotic tissues, this system substantially reduces the risk of insertional mutagenesis...
  11. ncbi Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease
    J H Kordower
    Department of Neurological Sciences, Rush Presbyterian St Luke s Medical Center, Chicago, IL 60612, USA
    Science 290:767-73. 2000
    ..These data indicate that GDNF delivery using a lentiviral vector system can prevent nigrostriatal degeneration and induce regeneration in primate models of PD and might be a viable therapeutic strategy for PD patients...
  12. ncbi Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency
    Akitsu Hotta
    Developmental and Stem Cell Biology Program, Toronto, Ontario, Canada
    Nat Methods 6:370-6. 2009
    ..Finally, we used EOS selection to establish Rett syndrome-specific mouse and human iPS cell lines with known mutations in MECP2...
  13. ncbi Prevention strategies against small ruminant lentiviruses: an update
    Ramses Reina
    Institute of Agrobiotechnology CSIC UPNA Government of Navarra, 31192 Mutilva Baja, Spain
    Vet J 182:31-7. 2009
    ..This review updates the knowledge on diagnosis, risk of infection, immunisation approaches and criteria for selecting the different strategies to control the spread of SRLVs...
  14. pmc The cargo-binding domain of transportin 3 is required for lentivirus nuclear import
    Eric C Logue
    Department of Microbiology, New York University School of Medicine, 550 First Avenue, New York, NY 10016, USA
    J Virol 85:12950-61. 2011
    ..The mutated TNPO3 proteins maintained their ability to localize to the nucleus, suggesting that their inability to restore lentivirus infection resulted from an inability to bind to a host or viral cargo protein.
  15. pmc Lentivirus-based genetic manipulations of cortical neurons and their optical and electrophysiological monitoring in vivo
    Tanjew Dittgen
    Department of Molecular Neurobiology, Max Planck Institute for Medical Research, Jahnstrasse 29, 69120 Heidelberg, Germany
    Proc Natl Acad Sci U S A 101:18206-11. 2004
    ..This approach is ideally suited for analysis of gene functions in individual neurons in the intact brain...
  16. ncbi Excision of reprogramming transgenes improves the differentiation potential of iPS cells generated with a single excisable vector
    Cesar A Sommer
    Section of Gastroenterology, Department of Medicine, Boston University School of Medicine, Boston, Massachusetts 02118, USA
    Stem Cells 28:64-74. 2010
    ..In addition, our results underscore the consequences of long-term culture that will need to be taken into account for the clinical application of iPS cells...
  17. pmc Nonintegrating lentivector vaccines stimulate prolonged T-cell and antibody responses and are effective in tumor therapy
    Katarzyna Karwacz
    Division of Infection and Immunity, University College London, London, United Kingdom
    J Virol 83:3094-103. 2009
    ..In this case, both the vector genome and the immune response were maintained for at least 2 months. Together, our data indicate that nonintegrating lentivectors can be employed to generate effective vaccines...
  18. pmc LEDGF hybrids efficiently retarget lentiviral integration into heterochromatin
    Rik Gijsbers
    Division of Molecular Medicine, Katholieke Universiteit Leuven, Leuven, Belgium
    Mol Ther 18:552-60. 2010
    ..During normal lentivirus infection, the host cell-encoded transcriptional coactivator lens epithelium-derived growth factor/p75 (LEDGF/..
  19. ncbi Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model
    A Auricchio
    Institute for Human Gene Therapy, Department of Molecular and Cellular Engineering, The Wistar Institute, Philadelphia, PA, USA
    Hum Mol Genet 10:3075-81. 2001
    Recombinant vectors based on adeno-associated virus (AAV) or human immunodeficiency 1 (lentivirus) are promising tools for long term in vivo gene delivery...
  20. ncbi Hot spots of retroviral integration in human CD34+ hematopoietic cells
    Claudia Cattoglio
    Italian Institute of Technology, Unit of Molecular Neuroscience, Istituto Scientifico H San Raffaele, Milan, Italy
    Blood 110:1770-8. 2007
    ..The lower propensity of LV vectors for integrating in potentially dangerous regions of the human genome may be a factor determining a better safety profile for gene therapy applications...
  21. ncbi Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
    Angelo Lombardo
    San Raffaele Telethon Institute for Gene Therapy, Via Olgettina, 58, 20132 Milan, Italy
    Nat Biotechnol 25:1298-306. 2007
    ....
  22. pmc Efficient gene targeting mediated by a lentiviral vector-associated meganuclease
    Araksya Izmiryan
    Hopital Necker Enfants Malades, Universite Paris Descartes, 75743 Paris, France
    Nucleic Acids Res 39:7610-9. 2011
    ..These integration patterns were dependent upon the mode of meganuclease delivery, suggesting distinct recombination processes...
  23. ncbi Codon optimization of human factor VIII cDNAs leads to high-level expression
    Natalie J Ward
    Molecular Immunology Unit, Institute of Child Health, University College London, London, UK
    Blood 117:798-807. 2011
    ..These significant findings demonstrate that shorter FVIII constructs that can be more easily accommodated in viral vectors can result in increased therapeutic efficacy and may deliver effective gene therapy for hemophilia A...
  24. pmc Systematic comparison of constitutive promoters and the doxycycline-inducible promoter
    Jane Yuxia Qin
    School of Life Sciences, Northeast Normal University, Changchun, China
    PLoS ONE 5:e10611. 2010
    ..At maximal induction, the TRE promoter is comparable to a strong constitutive promoter. These results should facilitate more rational choices of promoters in ectopic gene expression studies...
  25. ncbi Induction of an interferon response by RNAi vectors in mammalian cells
    Alan J Bridge
    Swiss Institute for Experimental Cancer Research, CH 1066 Epalinges, Switzerland
    Nat Genet 34:263-4. 2003
    ..Although siRNAs are thought to be too short to induce interferon expression, we report here that a substantial number of shRNA vectors can trigger an interferon response...
  26. pmc Vaccines delivered by integration-deficient lentiviral vectors targeting dendritic cells induces strong antigen-specific immunity
    Biliang Hu
    Mork Family Department of Chemical Engineering and Materials Science, University of Southern California, Los Angeles, CA 90089, USA
    Vaccine 28:6675-83. 2010
    ..Thus, this DC-directed IDLV system represents a promising and efficient vector platform with remarkably improved safety for the future development of DC-based immunotherapy...
  27. pmc Distinct subtypes of cholecystokinin (CCK)-containing interneurons of the basolateral amygdala identified using a CCK promoter-specific lentivirus
    Aaron M Jasnow
    Department of Psychiatry and Behavioral Sciences, Center for Behavioral Neuroscience, Yerkes Research Center, Emory University School of Medicine, Atlanta, Georgia, USA
    J Neurophysiol 101:1494-506. 2009
    ..To address this question, we generated a lentivirus-expressing GFP under the control of the CCK promoter to identify CCK neurons in vivo...
  28. pmc Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors
    Ute Modlich
    Department of Experimental Hematology, Hannover Medical School, Hannover, Germany
    Mol Ther 17:1919-28. 2009
    ..Mechanistic studies support the conclusion that enhancer-mediated gene activation is the major cause for insertional transformation of hematopoietic cells, opening rational strategies for risk prevention...
  29. pmc Silencing of OB-RGRP in mouse hypothalamic arcuate nucleus increases leptin receptor signaling and prevents diet-induced obesity
    Cyril Couturier
    Departments of Cell Biology and Hematology, Institut Cochin, Unité Mixte de Recherche 8104, Centre National de la Recherche Scientifique, Universite Paris Descartes, 75014 Paris, France
    Proc Natl Acad Sci U S A 104:19476-81. 2007
    ..This finding could serve as the basis for an approach to identifying potential new therapeutic targets for a variety of diseases, including obesity...
  30. ncbi Highly efficient multicistronic lentiviral vectors with peptide 2A sequences
    Abdelilah Ibrahimi
    Molecular Medicine, Katholieke Universiteit Leuven, B 3000 Leuven, Flanders, Belgium
    Hum Gene Ther 20:845-60. 2009
    ..Last, using various peptide 2A sequences, we engineered the triple reporter LV-3R (i.e., eGFP, fLuc, HSV1-sr39tk), enabling efficient multimodality readouts in vivo...
  31. pmc Preferential labeling of inhibitory and excitatory cortical neurons by endogenous tropism of adeno-associated virus and lentivirus vectors
    J L Nathanson
    Systems Neurobiology Laboratories, Salk Institute for Biological Studies, 10010 North Torrey Pines Road, La Jolla, CA 92037, USA
    Neuroscience 161:441-50. 2009
    ..cortical neuron types by recombinant AAV pseudotyped with serotype 1 capsid (rAAV2/1) and by recombinant lentivirus pseudotyped with the vesicular stomatitis virus (VSV) glycoprotein...
  32. pmc Development of optimal bicistronic lentiviral vectors facilitates high-level TCR gene expression and robust tumor cell recognition
    S Yang
    Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Gene Ther 15:1411-23. 2008
    ..Such optimal vectors may have immediate applications in cancer gene therapy...
  33. ncbi High-level sustained transgene expression in human embryonic stem cells using lentiviral vectors
    Yue Ma
    National Primate Research Center, School of Medicine, University of Wisconsin, Madison, Wisconsin, USA
    Stem Cells 21:111-7. 2003
    ..These results also have important implications for the possible future use of gene-modified human ES cells in transplantation and tissue regeneration applications...
  34. ncbi A multicolor panel of novel lentiviral "gene ontology" (LeGO) vectors for functional gene analysis
    Kristoffer Weber
    Clinic for Stem Cell Transplantation, University Medical Center Hamburg Eppendorf, Hamburg, Germany
    Mol Ther 16:698-706. 2008
    ..LeGO vectors thus represent a valuable tool for investigating gene networks using conditional ectopic expression and knock-down approaches simultaneously...
  35. ncbi Single-vector inducible lentiviral RNAi system for oncology target validation
    Dmitri Wiederschain
    Oncology Research, Novartis Institutes for BioMedical Research, Cambridge, Massachusetts 02139, USA
    Cell Cycle 8:498-504. 2009
    ..The versatile and robust inducible lentiviral RNAi system reported herein can therefore serve as a powerful tool to rapidly reveal tumor cell dependence...
  36. ncbi A lentivirus-based system to functionally silence genes in primary mammalian cells, stem cells and transgenic mice by RNA interference
    Douglas A Rubinson
    Department of Biology, Massachusetts Institute of Technology, 77 Massachusetts Avenue, Cambridge, Massachusetts 02139, USA
    Nat Genet 33:401-6. 2003
    ..We show that lentivirus-delivered shRNAs are capable of specific, highly stable and functional silencing of gene expression in a variety ..
  37. pmc Integration-deficient lentiviral vectors: a slow coming of age
    Klaus Wanisch
    School of Biological Sciences, Royal Holloway, University of London, Egham, Surrey, UK
    Mol Ther 17:1316-32. 2009
    ..IDLVs can be converted into replicating episomes, suggesting that if a clinically applicable system can be developed they would also become highly appropriate for stable transduction of proliferating tissues in therapeutic applications...
  38. pmc Engineered lentivector targeting of dendritic cells for in vivo immunization
    Lili Yang
    Division of Biology, California Institute of Technology, 1200 E California Blvd, Pasadena, California 91125, USA
    Nat Biotechnol 26:326-34. 2008
    ..Thus, lentiviral vectors targeting dendritic cells provide a simple method of producing effective immunity and may provide an alternative route for immunization with protein antigens...
  39. pmc Recent advances in lentiviral vector development and applications
    Janka Matrai
    Flanders Institute for Biotechnology, VIB, Vesalius Research Center, University of Leuven, Leuven, Belgium
    Mol Ther 18:477-90. 2010
    ..These recent advances underscore the improved safety and efficacy of LVs with important implications for clinical trials...
  40. pmc HIV-1 lentiviral vector immunogenicity is mediated by Toll-like receptor 3 (TLR3) and TLR7
    Karine Breckpot
    Division of Infection and Immunity, University College London, 46 Cleveland Street, London W1T 4JF, United Kingdom
    J Virol 84:5627-36. 2010
    ..In conclusion, we demonstrated TLR-dependent DC activation by lentiviral vectors, explaining their immunogenicity. These data allow the rational development of strategies to manipulate the host's immune response to the transgene...
  41. ncbi Systematic determination of the packaging limit of lentiviral vectors
    M Kumar
    Department of Molecular Virology and Microbiology, Baylor College of Medicine, Houston, TX 77030, USA
    Hum Gene Ther 12:1893-905. 2001
    ..These results suggest that HIV-based vectors may have a secondary advantage over oncoretroviral vectors because of their greater packaging limit, although the very low titers of the larger vectors will be of limited utility...
  42. pmc Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk
    Janka Matrai
    Free University of Brussels, Brussels, Belgium
    Hepatology 53:1696-707. 2011
    ..Deep sequencing of IDLV-transduced livers showed only rare genomic integrations that had no preference for gene coding regions and occurred mostly by a mechanism inconsistent with residual integrase activity...
  43. ncbi Lentiviral vectors: regulated gene expression
    T Kafri
    Laboratory of Genetics, The Salk Institute, La Jolla, California 92037, USA
    Mol Ther 1:516-21. 2000
    ..These studies show that an inducible lentiviral vector can deliver and regulate transgene expression in vivo. We believe that regulated gene expression is an essential tool for successful gene therapy approaches...
  44. ncbi Dual-promoter lentiviral vectors for constitutive and regulated gene expression in neurons
    Sergio Gascón
    Instituto de Investigaciones Biomedicas Alberto Sols, CSIC UAM, Arturo Duperier, 4, 28029 Madrid, Spain
    J Neurosci Methods 168:104-12. 2008
    ..The viral systems we developed here provide neuron-specific and regulated expression mediated by single lentiviral vectors and will prove valuable tools for the study of neuronal function...
  45. ncbi Gene delivery by lentivirus vectors
    Adam S Cockrell
    Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA
    Mol Biotechnol 36:184-204. 2007
    ..Concluding remarks address some of the successes in preclinical animals, and the recent transition of lentiviral vectors to human clinical trials as therapy for a variety of infectious and genetic diseases...
  46. ncbi Silencing and variegation of gammaretrovirus and lentivirus vectors
    James Ellis
    Developmental Biology Program, Hospital for Sick Children, Toronto, ON, Canada M5G 1L7
    Hum Gene Ther 16:1241-6. 2005
    ..In contrast, lentivirus vectors based on human immunodeficiency virus type 1 appear to express more efficiently, although other reports ..
  47. ncbi Lentivirus vectors encoding both central polypurine tract and posttranscriptional regulatory element provide enhanced transduction and transgene expression
    S C Barry
    Department of Pediatrics, University of Washington, Seattle, WA 98195, USA
    Hum Gene Ther 12:1103-8. 2001
    Incorporation of a central polypurine tract (cPPT) and a posttranscriptional regulatory element (PRE) into lentivirus vectors provides increased transduction efficiency and transgene expression...
  48. ncbi Integrase-defective lentiviral vectors: progress and applications
    M B Banasik
    Department of Pediatrics, Program in Gene Therapy, Carver College of Medicine, The University of Iowa, Iowa City, IA, USA
    Gene Ther 17:150-7. 2010
    ..Several recent publications outline the development and initial biological characterization of such vectors. Here, we discuss the potential applications and new directions for the development of integration-defective LVs...
  49. ncbi Secreted frizzled-related protein-1 enhances mesenchymal stem cell function in angiogenesis and contributes to neovessel maturation
    Pascale Dufourcq
    Institut National de la Santé et de la Recherche Médicale U828, Universite Victor Segalen, Bordeaux2, Pessac, France
    Stem Cells 26:2991-3001. 2008
    ..Disclosure of potential conflicts of interest is found at the end of this article...
  50. ncbi Generation of multi-functional antigen-specific human T-cells by lentiviral TCR gene transfer
    M Perro
    Department of Immunology, Division of Infection and Immunity, University College London, Royal Free Hospital, London, UK
    Gene Ther 17:721-32. 2010
    ..The data demonstrate that the combination of lentiviral TCR gene transfer together with IL15/IL21 stimulation can efficiently redirect the antigen specificity of resting primary human T-cells and generate multi-functional T-cells...
  51. ncbi An improved tet-on system for gene expression in neurons delivered by a single lentiviral vector
    Xuebi Tian
    Department of Anesthesiology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430030, China
    Hum Gene Ther 20:113-23. 2009
    ..Transgene expression from the NSE promoter also provided tightly regulated gene expression in neurons in vivo...
  52. pmc Efficient reprogramming of human cord blood CD34+ cells into induced pluripotent stem cells with OCT4 and SOX2 alone
    Xianmei Meng
    Division of Regenerative Medicine MC1528B, Department of Medicine, Loma Linda University, Loma Linda, California 92350, USA
    Mol Ther 20:408-16. 2012
    ..This report is the first to describe the generation of transgene-free iPSCs with the use of OCT4 and SOX2 alone. These findings have important implications for the clinical applications of iPSCs...
  53. pmc Decreased striatal RGS2 expression is neuroprotective in Huntington's disease (HD) and exemplifies a compensatory aspect of HD-induced gene regulation
    Tamara Seredenina
    Laboratory of Functional Neurogenomics, Ecole Polytechnique Federale de Lausanne, Lausanne, Switzerland
    PLoS ONE 6:e22231. 2011
    ..Here we test whether individual changes in striatal gene expression are capable of mitigating HD-related neurotoxicity...
  54. ncbi Genetic analysis of small ruminant lentiviruses following lactogenic transmission
    Giuliano Pisoni
    Universita degli Studi di Milano, Dipartimento di Patologia Animale, Igiene e Sanita Pubblica Veterinaria, Via Celoria 10, 20133 Milano, Italy
    Virology 407:91-9. 2010
    ..that the physiology of the ruminant neonatal intestine, which permits the adsorption of infected maternal cells, shaped the evolution of these particular lentiviruses that represent a valid model of lactogenic lentivirus transmission.
  55. ncbi Viral evolution in deep time: lentiviruses and mammals
    Robert J Gifford
    Aaron Diamond AIDS Research Center, 455 1st Avenue, New York, NY 10016, USA
    Trends Genet 28:89-100. 2012
    ....
  56. ncbi Phylogenetic analysis of small ruminant lentiviruses from Southern Brazil
    A P Ravazzolo
    Biotechnology Center and Veterinary Faculty, UFRGS Universidade Federal do Rio Grande do Sul, Av Bento Goncalves 9500, CEP 91501 970, Porto Alegre, Brazil
    Virus Res 79:117-23. 2001
    The first lentivirus isolated from sheep in Brazil was analysed phylogenetically...
  57. pmc Investigating antibody neutralization of lyssaviruses using lentiviral pseudotypes: a cross-species comparison
    Edward Wright
    MRC UCL Centre for Medical Molecular Virology, Division of Infection and Immunity, University College London, 46 Cleveland Street, London W1T 4JF, UK
    J Gen Virol 89:2204-13. 2008
    ....
  58. ncbi Transduction of human islets with pseudotyped lentiviral vectors
    Gary P Kobinger
    Gene Therapy Program, Division of Medical Genetics, Department of Medicine, University of Pennsylvania Health System, Philadelphia, PA 19104, USA
    Hum Gene Ther 15:211-9. 2004
    ..Overall, our study describes an improved methodology for achieving safe and efficient gene transfer into cells of human islets...
  59. ncbi Lentivirus-mediated overexpression of microRNA-199a inhibits cell proliferation of human hepatocellular carcinoma
    Xiao Qin Jia
    Cancer Center and Department of Pathology, Nanjing Medical University, Nanjing, China
    Cell Biochem Biophys 62:237-44. 2012
    ..Thus, these studies provide an important new insight into the pathogenesis of human HCC and it may open a new perspective for the development of effective gene therapy for human HCC...
  60. ncbi Development of novel efficient SIN vectors with improved safety features for Wiskott-Aldrich syndrome stem cell based gene therapy
    Inés Avedillo Díez
    Department of Pediatric Hematology Oncology, Hannover Medical School, Hannover, Germany
    Mol Pharm 8:1525-37. 2011
    ..Therefore, the described SIN vectors appear to be good candidates for potential use in a safer new gene therapy protocol for WAS, with decreased risk of insertional mutagenesis...
  61. pmc Lentiviral gene transfer into the dorsal root ganglion of adult rats
    Hongwei Yu
    Department of Anesthesiology, Medical College of Wisconsin, 8100 Watertown Plank Rd, Milwaukee, Wisconsin 53226, USA
    Mol Pain 7:63. 2011
    ....
  62. pmc Amelioration of emphysema in mice through lentiviral transduction of long-lived pulmonary alveolar macrophages
    Andrew A Wilson
    Pulmonary Center and Department of Medicine, Boston University School of Medicine, 715 Albany Street, Boston, Massachusetts 02118, USA
    J Clin Invest 120:379-89. 2010
    ..Our results challenge the dogma that AMs are short lived and suggest that these differentiated cells may be a possible target cell population for in vivo gene therapy applications, including the sustained correction of hAAT deficiency...
  63. ncbi Novel integrase-defective lentiviral episomal vectors for gene transfer
    J Vargas
    Division of Infectious Disease, Department of Medicine, Mount Sinai School of Medicine, New York, NY 10029, USA
    Hum Gene Ther 15:361-72. 2004
    ..Lentiviral episomal vectors may serve as specific tools for therapeutic approaches to diseases, particularly those associated with episomal replication of DNA viruses including papillomaviruses, polyomaviruses, and herpesviruses...
  64. ncbi Quantitative analysis of lentiviral transgene expression in mice over seven generations
    Yong Wang
    Department of Laboratory Animal Science, College of Basic Medical Sciences, Third Military Medical University, Chongqing, China
    Transgenic Res 19:775-84. 2010
    ..Taken together, these data suggested that transgenic lines with long term stable expression and no position effect can be established by lentiviral transgenesis...
  65. pmc Lentiviral vectors transduce proliferating dendritic cell precursors leading to persistent antigen presentation and immunization
    Frederick Arce
    Division of Infection and Immunity, University College London, London, UK
    Mol Ther 17:1643-50. 2009
    ..These results show that LVs transduce DC precursors, which maintain transduced DCs in spleen for at least 2 months, leading to prolonged antigen presentation and effective T-cell memory...
  66. ncbi Characterization of new small ruminant lentivirus subtype B3 suggests animal trade within the Mediterranean Basin
    L Bertolotti
    Dipartimento di Produzioni Animali, Universita degli Studi di Torino, Grugliasco, Italy
    J Gen Virol 92:1923-9. 2011
    ....
  67. pmc Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression
    Samantha L Ginn
    Gene Therapy Research Unit of the Children s Medical Research Institute and The Children s Hospital at Westmead, Westmead, New South Wales, Australia
    Mol Ther 18:965-76. 2010
    ....
  68. ncbi Targeting l1 cell adhesion molecule using lentivirus-mediated short hairpin RNA interference reverses aggressiveness of oral squamous cell carcinoma
    Shiao Chen Hung
    Graduate Institute of Cancer Biology and Center for Molecular Medicine, China Medical University and Hospital, Taichung, Taiwan
    Mol Pharm 7:2312-23. 2010
    ..Targeting L1CAM gene expression in SCC4 cells overexpressing L1CAM using a lentivirus-mediated small hairpin RNA (shRNA) led to a significant reduction in cell proliferation in vitro via retardation ..
  69. ncbi Lentiviral vectors: their molecular design, safety, and use in laboratory and preclinical research
    Boro Dropulic
    Lentigen Corporation, Gaithersburg, MD 20878, USA
    Hum Gene Ther 22:649-57. 2011
    ..These include functional genomics, target validation, protein manufacturing, in vivo imaging, transgenic animals, and stem cell research...
  70. ncbi Production of influenza pseudotyped lentiviral particles and their use in influenza research and diagnosis: an update
    Jean Michel Garcia
    HKU Pasteur Research Centre, Dexter HC Man Building, 8 Sassoon Road, Pokfulam, Hong Kong
    Expert Rev Anti Infect Ther 9:443-55. 2011
    ..The second part focuses on the applications that such a flexible tool can provide in modern influenza research, in particular in the fields of drug discovery, molecular biology and diagnosis...
  71. ncbi Assaying beta-catenin/TCF transcription with beta-catenin/TCF transcription-based reporter constructs
    Travis L Biechele
    Howard Hughes Medical Institute and Department of Pharmacology, University of Washington School of Medicine, Seattle, WA, USA
    Methods Mol Biol 468:99-110. 2008
    ..Its enhanced sensitivity, increased dynamic range, and lentiviral platform provide a reporter system that will keep pace with the needs of scientists in the field...
  72. ncbi A dual promoter lentiviral vector for the in vivo evaluation of gene therapeutic approaches to axon regeneration after spinal cord injury
    K Low
    Department of Neurosciences, University of California San Diego, La Jolla, CA, USA
    Gene Ther 17:577-91. 2010
    ..This expression system is therefore an accurate and efficient means of screening candidate genes in vivo for enhancement of axonal growth...
  73. pmc Lentivirus-mediated overexpression of angiotensin-(1-7) attenuated ischaemia-induced cardiac pathophysiology
    Yanfei Qi
    Department of Pharmadocynamics, University of Florida, SW 1600 Archer Road, Gainesville, FL 32610, USA
    Exp Physiol 96:863-74. 2011
    ..Angiotensin-(1-7) [Ang-(1-7)] has been shown to provide cardioprotective effects. We hypothesize that lentivirus-mediated overexpression of Ang-(1-7) would protect the myocardium from ischaemic injury. A single bolus of 3...
  74. ncbi Generation of transgenic cattle by lentiviral gene transfer into oocytes
    Andreas Hofmann
    Department of Pharmacy, Institute for Pharmacology, Center for Drug Research, Ludwig Maximilians University, Butenandstrasse 5 C, 81377 Munich, Germany
    Biol Reprod 71:405-9. 2004
    ..Taken together, after adjusting lentiviral transgenesis to cattle, unprecedented high transgenesis and expression rates were achieved...
  75. ncbi Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow
    Dao Pan
    Department of Pediatrics and Institute of Human Genetics, University of Minnesota, Minneapolis, Minnesota 55455, USA
    Mol Ther 6:19-29. 2002
    ..The observation of bone marrow transduction after intravenous vector administration suggests the possibility of an in vivo approach to stem cell gene therapy...
  76. pmc Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5
    Xiao Feng Qin
    Division of Biology, California Institute of Technology, Pasadena 91125, USA
    Proc Natl Acad Sci U S A 100:183-8. 2003
    ..Here, we describe success in constructing a lentivirus-based vector to introduce siRNAs against the HIV-1 coreceptor, CCR5, into human peripheral blood T lymphocytes...
  77. ncbi Lentivirus-mediated gene transfer induces long-term transgene expression of BMP-2 in vitro and new bone formation in vivo
    Osamu Sugiyama
    Department of Orthopaedic Surgery, David Geffen School of Medicine, University of California at Los Angeles, Los Angeles, CA 90095, USA
    Mol Ther 11:390-8. 2005
    ..ELISA results demonstrated that lentivirus-mediated gene transfer into BMSCs induced stable BMP-2 production in vitro for 8 weeks...
  78. ncbi Neprilysin gene transfer reduces human amyloid pathology in transgenic mice
    Robert A Marr
    Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, California 92037, USA
    J Neurosci 23:1992-6. 2003
    ..These data further support a role for neprilysin in regulating cerebral amyloid deposition and suggest that gene transfer approaches might have potential for the development of alternative therapies for Alzheimer's disease...
  79. pmc Bone marrow stem cells expressing keratinocyte growth factor via an inducible lentivirus protects against bleomycin-induced pulmonary fibrosis
    Susana Aguilar
    Centre for Respiratory Research, Rayne Institute, University College London, London, United Kingdom
    PLoS ONE 4:e8013. 2009
    ..Taken together, our data indicates that bone marrow transplantation of lentivirus-transduced HSCs can attenuate lung damage, and shows for the first time the potential of using an inducible Tet-..
  80. pmc MECP2 isoform-specific vectors with regulated expression for Rett syndrome gene therapy
    Mojgan Rastegar
    Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
    PLoS ONE 4:e6810. 2009
    ..Our objective is to develop viral vectors for MECP2 gene transfer into Neural Stem Cells (NSC) and neurons suitable for gene therapy of Rett Syndrome...
  81. pmc HIV-1-based defective lentiviral vectors efficiently transduce human monocytes-derived macrophages and suppress replication of wild-type HIV-1
    Lingbing Zeng
    Pacific Biosciences Research Center, University of Hawaii at Manoa, Honolulu, Hawaii, USA
    J Gene Med 8:18-28. 2006
    ..In this study we established a lentiviral vector-based technique for improved gene transfer into human MDM cultures in vitro and demonstrated significant protection of transduced MDM from super-infection with wild-type HIV-1...
  82. pmc Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells
    Fang Zhang
    Centre for Immunodeficiency, Molecular Immunology Unit, Institute of Child Health, University College London, London, United Kingdom
    Blood 110:1448-57. 2007
    ..These properties are achieved in the absence of classic enhancer activity and therefore may confer a high safety profile...
  83. ncbi Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer
    Brian D Brown
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Via Olgettina 58, 20132 Milano, Italy
    Nat Med 12:585-91. 2006
    ..Our results provide novel in situ evidence of miRNA regulation and demonstrate a new paradigm in vector design with applications for genetic engineering and therapeutic gene transfer...
  84. pmc Lentiviral vector design for optimal T cell receptor gene expression in the transduction of peripheral blood lymphocytes and tumor-infiltrating lymphocytes
    Stephanie Jones
    Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
    Hum Gene Ther 20:630-40. 2009
    ..This paper emphasizes the need to optimize both promoter function and protein synthesis in constructs that require stoichiometric production of multiple protein subunits...
  85. pmc Mechanism of reduction in titers from lentivirus vectors carrying large inserts in the 3'LTR
    Fabrizia Urbinati
    Division of Experimental Hematology and Cancer Biology, Cincinnati Children s Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio 45229 3039, USA
    Mol Ther 17:1527-36. 2009
    ..These studies have important implications in design of integrating vectors...
  86. pmc A versatile approach to multiple gene RNA interference using microRNA-based short hairpin RNAs
    Xiaocui Zhu
    The Alliance for Cellular Signaling, Division of Biology, California Institute of Technology, Pasadena, CA 91125, USA
    BMC Mol Biol 8:98. 2007
    ..Effective and stable knockdown of multiple gene targets by RNA interference is often necessary to overcome isoform redundancy, but it remains a technical challenge when working with intractable cell systems...
  87. ncbi Lentiviral vectors with CMV or MHCII promoters administered in vivo: immune reactivity versus persistence of expression
    Takahiro Kimura
    Department of Medicine, Division of Digestive Diseases, David Geffen School of Medicine at University of California Los Angeles, Los Angeles, California, USA
    Mol Ther 15:1390-9. 2007
    ..These studies reveal that APC transduction by LVs could lead to immune reactivity (LV-CMV) or persistence of transgene expression (LV-MHCII), providing a relevant paradigm for vaccination and gene replacement approaches...
  88. ncbi A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
    Brian D Brown
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy
    Blood 110:4144-52. 2007
    ..This work, which is among the first applications to exploit the microRNA regulatory pathway, provides the basis for a promising new therapy for the treatment of hemophilia B...
  89. ncbi Polycistronic lentiviral vector for "hit and run" reprogramming of adult skin fibroblasts to induced pluripotent stem cells
    Chia Wei Chang
    Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham, Birmingham, AL, USA
    Stem Cells 27:1042-9. 2009
    ..These results suggest that a single, polycistronic "hit and run" vector can safely and effectively reprogram adult dermal fibroblasts into iPS cells...
  90. pmc Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
    Marina Cavazzana-Calvo
    Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm Assistance Publique Hôpitaux de Paris, Paris 75015, France
    Nature 467:318-22. 2010
    ..The clonal dominance that accompanies therapeutic efficacy may be coincidental and stochastic or result from a hitherto benign cell expansion caused by dysregulation of the HMGA2 gene in stem/progenitor cells...
  91. ncbi In vitro proliferation of cells derived from adult human beta-cells revealed by cell-lineage tracing
    Holger A Russ
    Department of Human Molecular Genetics and Biochemistry, Sackler School of Medicine, Tel Aviv University, Ramat Aviv, Tel Aviv, Israel
    Diabetes 57:1575-83. 2008
    ..A major obstacle to development of effective expansion protocols is the rapid loss of beta-cell markers in the cultured cells. Here, we report a genetic cell-lineage tracing approach for following the fate of cultured beta-cells...
  92. ncbi Gene transfer into rat mesenchymal stem cells: a comparative study of viral and nonviral vectors
    J M McMahon
    Regenerative Medicine Institute, National Centre for Biomedical Engineering Science and Department of Medicine, National University of Ireland, Galway, Republic of Ireland
    Stem Cells Dev 15:87-96. 2006
    ..Adenovirus, adeno-associated virus (AAV; serotypes 1, 2, 4, 5, and 6), lentivirus, and nonviral vectors were compared...
  93. ncbi Restraining influence of A2 neurons in chronic control of arterial pressure in spontaneously hypertensive rats
    Hanad Duale
    Department of Physiology, Bristol Heart Institute, School of Medical Sciences, University Walk, University of Bristol, Bristol, BS8 1TD, UK
    Cardiovasc Res 76:184-93. 2007
    ..We aimed to genetically target A2 neurons and induce expression of a potassium channel to reduce their electrical excitability and study how this impacts on long-term blood pressure control...
  94. pmc Targeted delivery of proteins across the blood-brain barrier
    Brian J Spencer
    Laboratory of Genetics, The Salk Institute for Biological Studies, La Jolla, CA 92037, USA
    Proc Natl Acad Sci U S A 104:7594-9. 2007
    ..Here, we report the use of the lentivirus vector system to deliver the lysosomal enzyme glucocerebrosidase and a secreted form of GFP to the neurons and ..
  95. pmc Delivery of the Cre recombinase by a self-deleting lentiviral vector: efficient gene targeting in vivo
    A Pfeifer
    The Salk Institute, La Jolla, CA 92037, USA
    Proc Natl Acad Sci U S A 98:11450-5. 2001
    ..Such a self-deleting Cre vector is a promising tool for the induction of conditional gene modifications with minimal Cre toxicity in vivo...
  96. pmc Characterization of a novel simian immunodeficiency virus from guereza colobus monkeys (Colobus guereza) in Cameroon: a new lineage in the nonhuman primate lentivirus family
    V Courgnaud
    , Montpellier, France
    J Virol 75:857-66. 2001
    ..In this study, we describe a novel lentivirus, named SIVcol, prevalent in guereza colobus monkeys...
  97. pmc Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses
    Evelyn Abordo-Adesida
    Gene Therapeutics Research Institute, Cedars Sinai Medical Center, Los Angeles, CA 90048, USA
    Hum Gene Ther 16:741-51. 2005
    ..is responsible for providing antigenic epitopes to the activated immune system, on systemic immunization with lentivirus. Low immunogenicity and prolonged transgene expression in the presence of preexisting lentiviral immunity are ..
  98. pmc Generation of tissue-specific transgenic birds with lentiviral vectors
    Benjamin B Scott
    Picower Institute for Learning and Memory, Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA 02139, USA
    Proc Natl Acad Sci U S A 102:16443-7. 2005
    ..Tissue-specific transgene expression at high levels provides a powerful tool for biological research and opens new avenues for genetic manipulation in birds...
  99. ncbi Lentivirally transduced dendritic cells as a tool for cancer immunotherapy
    Karine Breckpot
    Department of Physiology and Immunology, Medical School of the Vrije Universiteit Brussel VUB, Laarbeeklaan 103 E, 1090 Brussels, Belgium
    J Gene Med 5:654-67. 2003
    ..Dendritic cells (DC) are the professional antigen-presenting cells of the immune system, fully equipped to prime naive T cells and thus essential components for cancer immunotherapy...
  100. pmc Efficient transgenesis in farm animals by lentiviral vectors
    Andreas Hofmann
    Department of Pharmacy, Institute for Pharmacology, Center for Drug Research, Butenandtstrasse 5 C, Ludwig Maximilians University, 81377 Munich, Germany
    EMBO Rep 4:1054-60. 2003
    ..Finally, infection of bovine oocytes after and before in vitro fertilization with LV-PGK resulted in transgene expression in 45% and 92% of the infected embryos, respectively...
  101. pmc Differentiation stage determines potential of hematopoietic cells for reprogramming into induced pluripotent stem cells
    Sarah Eminli
    Massachusetts General Hospital Cancer Center and Center for Regenerative Medicine, Boston, Massachusetts, USA
    Nat Genet 41:968-76. 2009
    ..Moreover, we identify hematopoietic progenitors as an attractive cell type for applications of iPS cell technology in research and therapy...

Research Grants76

  1. NIH Director's Pioneer Award
    ERICH JARVIS; Fiscal Year: 2009
    ..Repairing the pathway in vocal learners, when damaged, would have profound impact for correcting neurological disorders of speech. ..
  2. Vitamin D3, Interference with Androgen Signal and Inhibition of Prostate Cancer
    Bandana Chatterjee; Fiscal Year: 2013
    ..Methods: We will use Co-IP, chromatin IP, transfection, ShRNAs, inducible protein expression, lentivirus infection, cell proliferation, xenograft tumor growth in SCID mice, protein enrichment and mass spectrometry...
  3. Inhibition of bone formation by Prx1
    Mark S Nanes; Fiscal Year: 2013
    ..Expression vectors will be used in primary cell culture via lentivirus constructs to evaluate the effect of Osx or RUNX2 expression on Prx1 inhibition of OB differentiation...
  4. The Role of Adipocyte-Induced Inflammation in Prostate Tumor Progression
    AIMALIE LYNNETTE HARDAWAY; Fiscal Year: 2012
    ..system, in vivo knockout model of bone metastasis, immunoblotting, ELISA assays, cytokine arrays, RT PCR and Lentivirus knockdown technology...
  5. Neonatal Chemoselection Following Ex Vivo Gene Transfer For Hereditary Disorders
    KARIN L GAENSLER; Fiscal Year: 2012
    ..Fetal liver and bone marrow-derived HSC will be transduced ex vivo by lentivirus-mediated gene transfer of P140K-methylguanine methytransferase (MGMT)...
  6. Chronic Granulomatous Lung Inflammation Elicited by Carbon Nanotubes
    Mary Jane Thomassen; Fiscal Year: 2013
    ..size and numbers in: (a) macrophage-specific PPAR?-null versus wild-type mice;and (b) mice treated with lentivirus-PPAR? plasmids, PPAR? agonist rosiglitazone;or PPAR? antagonist BADGE versus untreated mice...
  7. A Novel Model of, and Pre-clinical Therapy for, Scleroderma Lung Disease
    Sergei P Atamas; Fiscal Year: 2012
    ..We propose, in Specific Aim 1, to establish and investigate a chronic model of mIL-442 expression utilizing lentivirus-mediated gene delivery...
  8. INSULIN ACTION IN MUSCLE AND FAT CELLS
    JAMES CARLTON GARRISON; Fiscal Year: 2010
    ..b>Lentivirus will be used to overexpress rictor or to express shRNA to knockdown rictor in 3T3-L1 adipocytes...
  9. Dysfunction of neurofilaments in disease: an iPS model of Giant Axonal Neuropathy
    BETHANY LESLIE JOHNSON-KERNER; Fiscal Year: 2012
    ..A rescue lentivirus will be utilized to determine if replacement of gigaxonin can reverse any GAN-related phenotypes, as a first ..
  10. Role of microRNAs in Coronavirus-induced lethal encephalitis
    Sonia Navas-Martin; Fiscal Year: 2012
    ..Aim 1) in IL-1Ra and IFN? induction by macrophages and glial cells ex vivo, as well as in vivo using antagomir-lentivirus vectors...
  11. ANDV Induced Responses of Hypoxic Endothelial Cells
    Erich R Mackow; Fiscal Year: 2012
    ..A Lentivirus-puromycin selection system will be used to express ANDV proteins within primary human ECs and used to define the ..
  12. Mammary Gland Morphogenesis and Breast Cancer
    Ian G Macara; Fiscal Year: 2013
    ..To manipulate gene expression in mammary glands, stem cells are transduced with lentivirus, and mammary glands are regenerated in host mice from these cells...
  13. Mechanisms of trauma and hemorrhage-induced impairment of innate immunity
    Xiao Di Tan; Fiscal Year: 2013
    ..knockdown of Atg12 results in attenuation of eliminating bacteria by lung epithelial cells in vitro using a lentivirus-based gene silencing approach...
  14. Emerging Functions of Mitochondrial Fission in Postischemic Endothelial Cells
    BARBARA RITA ALEVRIADOU; Fiscal Year: 2012
    ..Transfection with a lentivirus that expresses green fluorescent protein in mitochondria will be used to analyze their dynamic behavior during ..
  15. Platelet-Derived FVIII Gene Therapy of Hemophilia A
    Qizhen Shi; Fiscal Year: 2013
    ..Another study using 2bF8 lentivirus-mediated bone marrow (BM) transduction and syngeneic transplantation resulted in therapeutic levels of FVIII ..
  16. Nicotine Modulation of Parasympathetic Cardiac Neurons
    David Mendelowitz; Fiscal Year: 2013
    ..include the identification of the synaptic terminals of sensory neurons originating in the nasal mucosa using lentivirus expression of enhanced yellow fluorescent protein (eYFP), as well as the expression of channelrhodopsin-2 (ChR2),..
  17. Antigen presentation by epithelial stem cells to promote life long immunity
    MARIE CLAIRE ELISABETH GAUDUIN; Fiscal Year: 2013
    ..Our concept is to target epithelial stem cells with a lentivirus vector to introduce an antigen construct under the control of the involucrin promoter...
  18. RTP801 and ATF4 in Neuronal Death in PD
    OREN ABRAHAM LEVY; Fiscal Year: 2013
    ..g. primary neuronal cultures, the use of lentivirus for high efficiency transduction, and RNA interference...
  19. Defining the role of the tumor-initiating cell niche in breast cancer
    Mei Zhang; Fiscal Year: 2011
    ..By applying both in vitro genetic approaches (such as short hairpin RNA (shRNA) knockdown and lentivirus transduction) as well as in vivo functional assays (limiting dilution transplantation), we should be able to ..
  20. Phase 1 Study of Umbilical Cord Blood-Derived T Cells in Malignant B Cells
    Elizabeth J Shpall; Fiscal Year: 2013
    ..which (i) avoids current problems other investigators are currently experiencing manufacturing clinical-grade lentivirus for gene transfer of the chimeric antigen receptor (CAR) transgene, and (ii) by using a non-viral system reduces ..
  21. Imaging of gene delivery in the central nervous system
    Assaf A Gilad; Fiscal Year: 2010
    ..Due to the capacity of the lentivirus to carry therapeutic genes in addition to the reporter gene, we anticipate that our approach is applicable for ..
  22. Optimized Adaptation of Simian-tropic R5 HIV Clade C to Pig-tailed Macaques
    RUTH MARGRIT RUPRECHT; Fiscal Year: 2013
    ..A primate lentivirus model based mostly upon HIV-1 but replication-competent in macaques would greatly facilitate the preclinical ..
  23. Infant immunoprophylaxis against a primate lentivirus
    RUTH MARGRIT RUPRECHT; Fiscal Year: 2012
    ..abstract_text> ..
  24. Hippocampal plasticity in co-morbid diabetes and depression
    LAWRENCE P contact REAGAN; Fiscal Year: 2010
    ..to more selectively asses the role of insulin receptor signaling, we recently developed a novel and innovative lentivirus vector that contains an antisense sequence selective for IR (IRAS) that examines the effects of decreasing IR ..
  25. Lentivirus-based positive/negative selection in minimally ablative transplants
    KARIN L GAENSLER; Fiscal Year: 2013
    ....
  26. Mitochondrial Kv1.3 potassium channels and lymphocyte apoptosis in sepsis
    Jay Yang; Fiscal Year: 2011
    ..The innovative hypothesis will be tested using a wide range state-of-art techniques including lentivirus-based ex vivo engineering of primary T lymphocytes, ex vivo and in vitro assessment of apoptosis, organelle-..
  27. Protective immunity against HIV: the role of IgG subclass and glycosylation
    Donald Forthal; Fiscal Year: 2009
    ..on natural killer cells, monocytes, macrophages, or dendritic cells may play a critical role in preventing lentivirus infection...
  28. Functional study of novel transcriptional regulators of hematopoietic stem cells
    Shannon L McKinney-Freeman; Fiscal Year: 2012
    ..Functionally evaluate prioritized gene candidates via lentiviral delivery to HSC. Here, we will use lentivirus to delivery genes to enriched WBM HSC prior to their transplantation into lethally irradiated mice and assess ..
  29. The influence of genotype on the outcome of gene transfer in beta-thalassemia
    Stefano Rivella; Fiscal Year: 2013
    ..approach for the treatment of beta-thalassemia is the introduction of non-mutant mRNAs through gene therapy (lentivirus)...
  30. Regulation of Angiogenesis by Kininogen
    Keith R McCrae; Fiscal Year: 2013
    ..These studies will employ lentivirus-produced murine HK, as well as a mutant HK resistant to cleavage by kallikrein...
  31. PERIPHERAL NEUROPATHY IN LENTIVIRUS INFECTIONS: EARLY VIRAL AND HOST DETERMINANTS
    Christopher Power; Fiscal Year: 2010
    ..Our overall working hypothesis is that the early events resulting in lentivirus-induced distal sensory polyneuropathy represent convergent effects of infection/activation of macrophages and ..
  32. MR Spectroscopy to Evaluate Liver Repopulation by Transplanted Hepatocytes
    Chandan Guha; Fiscal Year: 2009
    ..of the MRS methodology would allow us to proceed to the R33 phase (Year 3-4) involving the construction of lentivirus vectors expressing CK-B for ex vivo gene therapy of donor hepatocytes in Gunn rats, a rodent model of Crigler-..
  33. MECHANOTRANSDUCTION IN INTESTINAL SMOOTH MUSCLE CELLS
    Gianrico Farrugia; Fiscal Year: 2013
    ..delivery, high resolution patch imaging, immunohistochemistry, Western blots, single cell PCR, quantitative PCR, lentivirus RNA knock down techniques, organotypic and single cell cultures, total internal reflection fluorescence (TIRF) ..
  34. Elucidating the role of TIN2L at the telomere and in dyskeratosis congenita
    Nya D Nelson; Fiscal Year: 2013
    ..role of the two TIN2 isoforms, TRF2, proteins identified via MS and telomerase in the DC phenotype, we will use lentivirus to stably overexpress these proteins in DC patient derived lymphoblastic cell lines, and the effects on telomere ..
  35. The role of the deacetylase Sirt1 in the maintenance of adult neural stem cells
    VICTORIA RAFALSKI; Fiscal Year: 2010
    ..the molecular mechanisms of Sirt1 action in adult NSC maintenance I will use a combination of pharmacological, lentivirus-mediated shRNA knockdown, and mouse knockout approaches to assess how Sirt1 loss and gain of function influence ..
  36. Physical and Functional Systems Analysis of Mammalian Mitochondria
    JODI M NUNNARI; Fiscal Year: 2013
    ..Using an optimized lentivirus-delivered tagging system coupled with mass spectrometry, a mammalian mitochondrial physical interactome map will ..
  37. Development of Novel AIDS Gene Therapy Strategies in the Macaque SHIV Model
    Hans Peter Kiem; Fiscal Year: 2012
    ..We will study these cassettes in lentivirus and foamy virus vectors to determine the safety of these vectors systems first in NOD/SCID mice and then in the ..
  38. Decreased CNS leptin activity in co-morbid depression and obesity
    Lawrence P Reagan; Fiscal Year: 2013
    ..experimental models of obesity: a novel antagonist that selectively blocks BBB transport of leptin and using a lentivirus vector that selectively downregulates hypothalamic insulin receptor expression (hypo-IRAS)...
  39. Liposome-siRNA-Peptide Complexes as Therapy to Cure Prion Diseases in Mouse Model
    Mark D Zabel; Fiscal Year: 2013
    ..However, delivery of lentivirus encoding PrP short hairpin RNA for RNAi has demonstrated only modest effectiveness experimentally and limited ..
  40. Therapeutic-Expression of a Platelet-Specific Integrin
    DAVID ALLEN WILCOX; Fiscal Year: 2010
    ..have recently shown a potentially feasible solution for this problem by transferring a therapeutic gene with a lentivirus vector into hematopoietic stem cells...
  41. FIV Testing of a CNS-Relevant Antiretroviral Compound
    Rick B Meeker; Fiscal Year: 2013
    ..it potentially affords adjunctive therapeutic value specifically appropriate for central nervous system (CNS) lentivirus burdens...
  42. Testing the safety and efficacy of inhibitory RNA mediated purine analog resistan
    Christopher C Porter; Fiscal Year: 2010
    ..interfering RNA mediated purine analog resistance (iPAR) allows for efficient in vitro and in vivo selection of lentivirus transduced murine hematopoietic progenitor cells using the purine analog, 6-thioguanine (6TG)...
  43. Defining the role of the tumor-initiating cell niche in breast cancer
    Mei Zhang; Fiscal Year: 2013
    ..By applying both in vitro genetic approaches (such as short hairpin RNA (shRNA) knockdown and lentivirus transduction) as well as in vivo functional assays (limiting dilution transplantation), we should be able to ..
  44. EIAV Envelope Variation and Vaccine Efficacy
    Ronald C Montelaro; Fiscal Year: 2013
    ..previous grant period, we demonstrated for the first time that Env variation is indeed a primary determinant of lentivirus vaccine efficacy that will need to be addressed in the effort to develop broadly protective vaccines...
  45. MicroRNAs as effectors of variable human drug metabolism
    Michael H Court; Fiscal Year: 2013
    ..enables rapid identification of miRs that suppress a fluorescent- tagged miR 3'-UTR reporter from a pooled lentivirus library of all known human miRs...
  46. Optogenetic Control of Vigilance State Transition
    Luis de Lecea; Fiscal Year: 2013
    ..First, we will introduce ChR2 in Hcrt neurons using a lentivirus and we will use different photostimulation patterns to manipulate Hcrt neuronal activity during wakefulness to ..
  47. The Role of KV1.3 in Effector T cells
    Peter A Calabresi; Fiscal Year: 2012
    ..3. To better define the specific role of Kv1.3, we have generated a lentivirus construct that expresses a dominant negative (DN) form of Kv1...
  48. Calcium-Stimulated Adenylyl Cyclase Activity's Role in Stress-Facilitated Memory
    LINDSAY ANN WIECZOREK; Fiscal Year: 2010
    ..through the use of gene therapy techniques, we can assess the region-specific importance of this activity via lentivirus administration that turns on AC8 expression...
  49. Regulation of Type I IFNs in Tolerance and Autoimmunity
    Stefania Gallucci; Fiscal Year: 2013
    ..We will use two protocols: a) injection of DCs transduced with a lentivirus carrying IL-4, and b) transplantation of bone marrow progenitors transduced with the same lentivirus, and will ..
  50. The Role of SRC-1 in Breast Cancer
    Jianming Xu; Fiscal Year: 2013
    ..b>Lentivirus will be used to induce mouse mammary tumors with endogenous expression, overexpression or knockout (KO) of Twist ..
  51. DYSREGULATION OF GLUCOSE HOMEOSTASIS IN AGING
    NIR J BARZILAI; Fiscal Year: 2013
    ..The goal of Aim 3 will be to study the lifelong effects of chronic central IGF-1 overexpression by lentivirus targeted to the hypothalamus and lifelong peripheral IGF-1 receptor blockade with an antibody, alone or in ..
  52. MicroRNA and Muscle Wasting in Uremia
    XIAONAN H WANG; Fiscal Year: 2013
    ..We will use in vivo gene transfer of miR29 by lentivirus and electroporation to test our theory...
  53. Small RNA regulation of BDNF expression in schizophrenia
    Schahram Akbarian; Fiscal Year: 2012
    ..2 will further explore the regulation of BDNF expression by the RNAi pathway in cultured cortical neurons with lentivirus-based transfection assays...
  54. The Impact of Tolerance in the Newborn on Lentiviral Infection
    Joseph M McCune; Fiscal Year: 2013
    ..this hypothesis, we propose to study lentiviral infection in the context of hosts made specifically tolerant to lentivirus. Given new findings that we have made about the induction of tolerance in the human fetus, we will rely upon a ..
  55. C1, Rostroventrolateral Medulla and Integration of Cardiovascular Regulation
    JOHN PATRICK CARD; Fiscal Year: 2012
    ..Experiments in three Specific Aims are advanced to test this hypothesis. Aim 1 will use our novel technology (lentivirus mediated anterograde tracing of C1 projections) and electron microscopy to test the hypothesis that recurrent ..
  56. Src Family Kinases and Control of Epithelial Cell Paracellular Permeability
    KURT F AMSLER; Fiscal Year: 2012
    ..Individual SFK contents will be manipulated using a unique lentivirus delivery system to target constructs to the proximal tubule segment...
  57. NanoART Manufacture, Delivery and Pharmacokinetics for Optimizing Drug Adherence
    Howard E Gendelman; Fiscal Year: 2013
    ..Third, animal studies will be performed in virus-infected rhesus macaques infected with recombinant lentivirus hybrids of SIV and HIV (SHIV) for safety and efficacy investigations, respectively.
  58. Dendritic Cell Restriction and Innate Immune Sensing of HIV Infection
    Dan Littman; Fiscal Year: 2011
    ..the cells are first exposed to virus-like particles (VLPs) that deliver the Vpx protein of the related primate lentivirus, SIVmac...
  59. Impact of Presenilin 2 Mutation and Deficiency on Microglial Function
    Suman Jayadev; Fiscal Year: 2010
    ..PS2 FAD mutations will be evaluated in comparison to microglia expressing control lentiviral vectors or lentivirus expressing PS2 shRNA to induce PS2 deficiency with the goal of assessing loss or gain of function effects of PS2 ..
  60. MOLECULAR AND METABOLIC ASPECTS OF IMPLANTATION
    Kelle H Moley; Fiscal Year: 2013
    ..We will use siRNA/shRNA lentivirus to knockdown GLUT1, glucose flux studies to assess glucose utilization in these cells and then inhibit these ..
  61. Development and Maintenance of Memory CD8 T Cells
    Jianzhu Chen; Fiscal Year: 2010
    ..An efficient lentivirus- mediated gene transfer will be developed to investigate gene function in memory CD8 T cell development and ..
  62. Bioengineered factor VIII gene therapy for hemophilia A
    H Trent Spencer; Fiscal Year: 2012
    ..levels in hemophilia A mice and that human hematopoietic stem cells are readily transduced with recombinant lentivirus encoding a genetically-engineered fVIII transgene...
  63. A Novel Method of Generating Hepatitis C Virus-Like Particles using Lentivirus
    Boro Dropulic; Fiscal Year: 2009
    ..We believe that a lentivirus vector system could alleviate the production bottleneck...
  64. LYMPHOCYTE-MEDIATED IMMUNITY TO ANAPLASMA MARGINALE
    Reginald Valdez; Fiscal Year: 1999
    ..Knowles (sponsor), Traves C. McGuire (a professor who has considerable experience in lentivirus immune mechanism), William C...
  65. WEILL CORNELL PROGRAM OF EXCELLENCE IN GENE THERAPY
    Ronald Crystal; Fiscal Year: 2005
    ..The 4 proposed projects (* = clinical) are: Project 1* - Genetic treatment of ?-thalassemia by lentivirus- mediated transfer of a regulated human ?-globin gene (M...
  66. Evaluation of RNA transport elements in HIV-1 vectors
    SRINIVAS KUMAR; Fiscal Year: 2007
    ..The specific aims of this proposal are: Aim 1. Improve performance of CTE-based lentivirus packaging systems...
  67. Transduction of the Mouse Auditory System with AAV
    DIANA LURIE; Fiscal Year: 2003
    ..of vectors have been successfully used to mediate intracochlear gene delivery including liposomes, herpes virus, lentivirus, adenovirus, and adeno-associated virus (AAV)...
  68. A Novel BIV vector pseudotyped with thogoto virus gp75
    Tianci Luo; Fiscal Year: 2006
    unreadable] DESCRIPTION (provided by applicant): Lentivirus-based gene transfer systems represent a promising gene delivery technology, as they integrate into the genome of the target cell and mediate sustained expression of the ..
  69. CYTOKINE RESPONSE IN FIV AND SIV IMMUNODEFICIENCY
    Gregg Dean; Fiscal Year: 2002
    ..since 1986 and has participated in a number of original and significant studies on the identification of lentivirus target cells, effects of opportunistic infections on immunodeficiency virus pathogenesis, and the role of ..
  70. MELK as Functional Marker for Mammary Tumors
    Alexey Terskikh; Fiscal Year: 2009
    ..We will isolate mammary tumor cells from MMTV-PyMT mice and knock down MELK transcripts using lentivirus expressing MELK shRNA and the red fluorescent protein mCherry...
  71. ALVEOLAR MACROPHAGES AND DEFENSE OF THE LUNG IN AIDS
    Paul Luciw; Fiscal Year: 1993
    Human immunodeficiency virus type 1 (HIV-1), a lentivirus etiologically linked to AIDS and related disorders, infects principally CD4+ T- lymphocytes and cells in the monocyte/macrophage lineage...
  72. RRE LINKED MUCOSAL BARRIER TO LENTIVIRUS INFECTION
    Margaret Barr; Fiscal Year: 2000
    Feline immunodeficiency virus, a lentivirus of cats, provides a natural model for human immunodeficiency virus (HIV) infection and disease...
  73. ENDOMEMBRANE TRAFFICKING OF FIV ENVELOPE GLYCOPROTEINS
    D Morre; Fiscal Year: 1993
    ..characterize the endomembrane trafficking between transitional endoplasmic reticulum and cis Golgi apparatus in lentivirus coat protein processing as a strategy for target identification in antiviral drug design using cultured cells ..
  74. HIV-1 Env Microglial Tropism and Neurovirulence
    Ruth Ruprecht; Fiscal Year: 2009
    ..Based upon these data, we postulate that the ability of a primate lentivirus to cross the blood brain barrier and to replicate efficiently in MG represents a hallmark of lentiviral ..
  75. MARROW SUPPRESSION FROM A LENTIVIRUS
    JANIS ABKOWITZ; Fiscal Year: 1993
    ..FTLV is a newly described lentivirus that is structurally and biologically similar to, but antigenically distinct from, the human immunodeficiency ..
  76. Characterization of cougar lentiviruses
    Mary Poss; Fiscal Year: 2003
    In the last 2 decades, both humans and domestic cats have become new hosts for lentivirus. Infection results in progressive immunodeficiency and death in both hosts...