genetic vectors


Summary: DNA molecules capable of autonomous replication within a host cell and into which other DNA sequences can be inserted and thus amplified. Many are derived from PLASMIDS; BACTERIOPHAGES; or VIRUSES. They are used for transporting foreign genes into recipient cells. Genetic vectors possess a functional replicator site and contain GENETIC MARKERS to facilitate their selective recognition.

Top Publications

  1. ncbi Enzymatic assembly of DNA molecules up to several hundred kilobases
    Daniel G Gibson
    The J Craig Venter Institute, Synthetic Biology Group, Rockville, Maryland, USA
    Nat Methods 6:343-5. 2009
  2. pmc A gateway cloning vector set for high-throughput functional analysis of genes in planta
    Mark D Curtis
    Institute of Plant Biology and Zurich Basel Plant Science Centre, University of Zurich, Zollikerstrasse 107, Ch 8008 Zurich, Switzerland
    Plant Physiol 133:462-9. 2003
  3. ncbi Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector
    Andrea L Szymczak
    Nat Biotechnol 22:589-94. 2004
  4. pmc Human induced pluripotent stem cells free of vector and transgene sequences
    Junying Yu
    Morgridge Institute for Research, Madison, WI 53707 7365, USA
    Science 324:797-801. 2009
  5. pmc Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    Amit C Nathwani
    Department of Haematology, University College London Cancer Institute, London, United Kingdom
    N Engl J Med 365:2357-65. 2011
  6. pmc A simplified system for generating recombinant adenoviruses
    T C He
    The Howard Hughes Medical Institute, 424 North Bond Street, Baltimore, MD 21231, USA
    Proc Natl Acad Sci U S A 95:2509-14. 1998
  7. pmc Safety and efficacy of gene transfer for Leber's congenital amaurosis
    Albert M Maguire
    Scheie Eye Institute, University of Pennsylvania, USA
    N Engl J Med 358:2240-8. 2008
  8. ncbi A variant of yellow fluorescent protein with fast and efficient maturation for cell-biological applications
    Takeharu Nagai
    Laboratory for Cell Function and Dynamics, Advanced Technology Development Center, Brain Science Institute, RIKEN, 2 1 Hirosawa, Wako City, Saitama, 351 0198, Japan
    Nat Biotechnol 20:87-90. 2002
  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 Gateway-compatible vectors for plant functional genomics and proteomics
    Keith W Earley
    Biology Department, Washington University, 1 Brookings Drive, St Louis, MO 63130, USA
    Plant J 45:616-29. 2006

Research Grants

Detail Information

Publications292 found, 100 shown here

  1. ncbi Enzymatic assembly of DNA molecules up to several hundred kilobases
    Daniel G Gibson
    The J Craig Venter Institute, Synthetic Biology Group, Rockville, Maryland, USA
    Nat Methods 6:343-5. 2009
    ..This assembly method can be used to seamlessly construct synthetic and natural genes, genetic pathways and entire genomes, and could be a useful molecular engineering tool...
  2. pmc A gateway cloning vector set for high-throughput functional analysis of genes in planta
    Mark D Curtis
    Institute of Plant Biology and Zurich Basel Plant Science Centre, University of Zurich, Zollikerstrasse 107, Ch 8008 Zurich, Switzerland
    Plant Physiol 133:462-9. 2003
    ..All vectors were derived from pCambia T-DNA cloning vectors, with the exception of a chemically inducible vector, for Agrobacterium sp.-mediated transformation of a wide range of plant species...
  3. ncbi Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector
    Andrea L Szymczak
    Nat Biotechnol 22:589-94. 2004
  4. pmc Human induced pluripotent stem cells free of vector and transgene sequences
    Junying Yu
    Morgridge Institute for Research, Madison, WI 53707 7365, USA
    Science 324:797-801. 2009
    ..These results demonstrate that reprogramming human somatic cells does not require genomic integration or the continued presence of exogenous reprogramming factors and removes one obstacle to the clinical application of human iPS cells...
  5. pmc Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
    Amit C Nathwani
    Department of Haematology, University College London Cancer Institute, London, United Kingdom
    N Engl J Med 365:2357-65. 2011
    ..Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder...
  6. pmc A simplified system for generating recombinant adenoviruses
    T C He
    The Howard Hughes Medical Institute, 424 North Bond Street, Baltimore, MD 21231, USA
    Proc Natl Acad Sci U S A 95:2509-14. 1998
    ..Homogeneous viruses can be obtained from this procedure without plaque purification. This system should expedite the process of generating and testing recombinant adenoviruses for a variety of purposes...
  7. pmc Safety and efficacy of gene transfer for Leber's congenital amaurosis
    Albert M Maguire
    Scheie Eye Institute, University of Pennsylvania, USA
    N Engl J Med 358:2240-8. 2008
    ..Although the follow-up was very short and normal vision was not achieved, this study provides the basis for further gene therapy studies in patients with LCA...
  8. ncbi A variant of yellow fluorescent protein with fast and efficient maturation for cell-biological applications
    Takeharu Nagai
    Laboratory for Cell Function and Dynamics, Advanced Technology Development Center, Brain Science Institute, RIKEN, 2 1 Hirosawa, Wako City, Saitama, 351 0198, Japan
    Nat Biotechnol 20:87-90. 2002
    ..With the improved speed and efficiency of maturation and the increased resistance to environment, Venus will enable fluorescent labelings that were not possible before...
  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 Gateway-compatible vectors for plant functional genomics and proteomics
    Keith W Earley
    Biology Department, Washington University, 1 Brookings Drive, St Louis, MO 63130, USA
    Plant J 45:616-29. 2006
    ..Antibodies detecting the FLAG, HA, cMyc and AcV5 tags show relatively little cross-reaction with endogenous proteins in a variety of monocotyledonous and dicotyledonous plants, suggesting broad utility for the tags and vectors...
  11. ncbi P[acman]: a BAC transgenic platform for targeted insertion of large DNA fragments in D. melanogaster
    Koen J T Venken
    Program in Developmental Biology, Baylor College of Medicine, Houston, TX 77030, USA
    Science 314:1747-51. 2006
    ..This transgenesis platform should greatly facilitate structure/function analyses of most Drosophila genes...
  12. 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
    ..While the actual biosafety of the vector will ultimately be proven in vivo, the improved design presented here should facilitate testing of lentivirus vectors...
  13. pmc A one pot, one step, precision cloning method with high throughput capability
    Carola Engler
    Icon Genetics GmbH, Biozentrum Halle, Halle, Germany
    PLoS ONE 3:e3647. 2008
  14. 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...
  15. ncbi LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
    S Hacein-Bey-Abina
    INSERM Unit 429, Cedex 15, France
    Science 302:415-9. 2003
    ..Thus, retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter...
  16. pmc Tight regulation, modulation, and high-level expression by vectors containing the arabinose PBAD promoter
    L M Guzman
    Department of Microbiology and Molecular Genetics, Harvard Medical School, Boston, Massachusetts 02115, USA
    J Bacteriol 177:4121-30. 1995
    ..We have exploited the tight regulation of the PBAD promoter to study the phenotypes of null mutations of essential genes and explored the use of pBAD vectors as an expression system...
  17. ncbi Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy
    Nathalie Cartier
    INSERM UMR745, University Paris Descartes, 75279 Paris, France
    Science 326:818-23. 2009
    ..Thus, lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD...
  18. ncbi Filamentous fusion phage: novel expression vectors that display cloned antigens on the virion surface
    G P Smith
    Science 228:1315-7. 1985
    ..Fusion phage may provide a simple way of cloning a gene when an antibody against the product of that gene is available...
  19. ncbi Generation of mouse induced pluripotent stem cells without viral vectors
    Keisuke Okita
    Center for iPS Cell Research and Application CiRA, Institute for Integrated Cell Material Sciences, Kyoto University, Kyoto 606 8507, Japan
    Science 322:949-53. 2008
    ..The production of virus-free iPS cells, albeit from embryonic fibroblasts, addresses a critical safety concern for potential use of iPS cells in regenerative medicine...
  20. ncbi Inducible gene targeting in mice
    R Kuhn
    Institute for Genetics, University of Cologne, Germany
    Science 269:1427-9. 1995
    ..Deletion was complete in liver and nearly complete in lymphocytes within a few days, whereas partial deletion was obtained in other tissues. This method can be used for the inducible inactivation of any other gene in vivo...
  21. ncbi Efficient transposition of the piggyBac (PB) transposon in mammalian cells and mice
    Sheng Ding
    Institute of Developmental Biology and Molecular Medicine, School of Life Sciences, Fudan University, 220 Handan Road, Shanghai 200433, China
    Cell 122:473-83. 2005
    ..These data provide a first and critical step toward a highly efficient transposon system for a variety of genetic manipulations including transgenesis and insertional mutagenesis in mice and other vertebrates...
  22. pmc Induced pluripotent stem cells generated without viral integration
    Matthias Stadtfeld
    Massachusetts General Hospital Cancer Center and Center for Regenerative Medicine, 185 Cambridge Street, Boston, MA 02114, USA
    Science 322:945-9. 2008
    ..Adenoviral reprogramming may provide an improved method for generating and studying patient-specific stem cells and for comparing embryonic stem cells and iPS cells...
  23. ncbi Virus-induced gene silencing in tomato
    Yule Liu
    Department of Molecular, Cellular and Developmental Biology, OML 451, Yale University, PO Box 208104, New Haven, CT 06520 8104, USA
    Plant J 31:777-86. 2002
    ..Using this vector, we have silenced RbcS and an endogenous gene homologous to the tomato EST cLED3L14. In the future, this modified vector system will facilitate large-scale functional analysis of tomato ESTs...
  24. 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...
  25. pmc Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
    Kevin D Foust
    Center for Gene Therapy, The Research Institute at Nationwide Children s Hospital, 700 Children s Drive, Columbus, Ohio 43205, USA
    Nat Biotechnol 27:59-65. 2009
    ..It may also be useful for rapid postnatal genetic manipulations in basic neuroscience studies...
  26. ncbi Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    Catherine S Manno
    The Children s Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania, 19104, USA
    Nat Med 12:342-7. 2006
    ..We conclude that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels of F.IX, but that future studies in humans may require immunomodulation to achieve long-term expression...
  27. ncbi GATEWAY vectors for Agrobacterium-mediated plant transformation
    Mansour Karimi
    Dept of Plant Systems Biology, Flanders Interuniversity Institute for Biotechnology VIB, Ghent University, K L Ledeganckstraat 35, B 9000, Gent, Belgium
    Trends Plant Sci 7:193-5. 2002
    ..Because these vectors are generally large, cloning can be time-consuming and laborious. Recently, the GATEWAY conversion technology has provided a fast and reliable alternative to the cloning of sequences into large acceptor plasmids...
  28. pmc DNA cloning using in vitro site-specific recombination
    J L Hartley
    Life Technologies, Inc, Rockville, Maryland 20850, USA
    Genome Res 10:1788-95. 2000
    ..In this paper, we outline the concepts of this approach and provide several examples that highlight some of its potential...
  29. pmc Golden gate shuffling: a one-pot DNA shuffling method based on type IIs restriction enzymes
    Carola Engler
    Icon Genetics GmbH, Biozentrum Halle, Halle, Germany
    PLoS ONE 4:e5553. 2009
  30. pmc Induction of immunity to human immunodeficiency virus type-1 by vaccination
    M Juliana McElrath
    Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N, D3 100, Seattle, WA 98109, USA
    Immunity 33:542-54. 2010
  31. ncbi A system for stable expression of short interfering RNAs in mammalian cells
    Thijn R Brummelkamp
    Division of Molecular Carcinogenesis, Division of Tumor Biology, The Netherlands Cancer Institute, Plesmanlaan 121, 1066 CX Amsterdam, Netherlands
    Science 296:550-3. 2002
    ..Therefore, the pSUPER vector constitutes a new and powerful system to analyze gene function in a variety of mammalian cell types...
  32. pmc Phase Ia clinical evaluation of the safety and immunogenicity of the Plasmodium falciparum blood-stage antigen AMA1 in ChAd63 and MVA vaccine vectors
    Susanne H Sheehy
    Centre for Clinical Vaccinology and Tropical Medicine, Churchill Hospital, Oxford, United Kingdom
    PLoS ONE 7:e31208. 2012
    ..falciparum. The development of a vaccine that could induce both cell-mediated and humoral immune responses would enable important proof-of-concept efficacy studies to be undertaken to address this question...
  33. ncbi A time- and cost-efficient system for high-level protein production in mammalian cells
    A Radu Aricescu
    Cancer Research UK Receptor Structure Research Group, Division of Structural Biology, Wellcome Trust Centre for Human Genetics, University of Oxford, Oxford, England
    Acta Crystallogr D Biol Crystallogr 62:1243-50. 2006
    ..The system is suitable for use in conventional laboratories or can be implemented in a medium- or high-throughput pipeline...
  34. ncbi Lentiviral vectors: basic to translational
    Toshie Sakuma
    Department of Molecular Medicine, Mayo Clinic College of Medicine, 200 First Street SW, Rochester, MN 55905, USA
    Biochem J 443:603-18. 2012
  35. ncbi Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
    Federico Mingozzi
    Center for Cellular and Molecular Therapeutics, The Children s Hospital of Philadelphia, 3501 Civic Center Boulevard, 5th Floor CTRB, Philadelphia, Pennsylvania 19104, USA
    Nat Rev Genet 12:341-55. 2011
    ..Recent exciting results have raised hopes for the treatment of many other diseases. As we discuss here, the prospects and challenges for AAV gene therapy are to a large extent dependent on the target tissue and the specific disease...
  36. 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
  37. pmc A nonviral minicircle vector for deriving human iPS cells
    Fangjun Jia
    Departments of Medicine and Radiology, Stanford University School of Medicine, Stanford, California, USA
    Nat Methods 7:197-9. 2010
    ..Here we use a single minicircle vector to generate transgene-free iPSCs from adult human adipose stem cells...
  38. pmc Sequential expression of pluripotency markers during direct reprogramming of mouse somatic cells
    Tobias Brambrink
    Whitehead Institute for Biomedical Research, Massachusetts Institute of Technology, 9 Cambridge Center, Cambridge, MA 02142, USA
    Cell Stem Cell 2:151-9. 2008
    ..Importantly, the virally transduced cDNAs needed to be expressed for at least 12 days in order to generate iPS cells. Our results are a step toward understanding some of the molecular events governing epigenetic reprogramming...
  39. pmc Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years
    Samuel G Jacobson
    Scheie Eye Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, USA
    Arch Ophthalmol 130:9-24. 2012
    ..To determine the safety and efficacy of subretinal gene therapy in the RPE65 form of Leber congenital amaurosis using recombinant adeno-associated virus 2 (rAAV2) carrying the RPE65 gene...
  40. pmc Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
    Guang Ping Gao
    Institute for Human Gene Therapy and Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA
    Proc Natl Acad Sci U S A 99:11854-9. 2002
  41. pmc Potent CD8+ T-cell immunogenicity in humans of a novel heterosubtypic influenza A vaccine, MVA-NP+M1
    Tamara K Berthoud
    The Jenner Institute, Oxford University, Oxford, United Kingdom
    Clin Infect Dis 52:1-7. 2011
    ..A vaccine providing protective immunity to the highly conserved internal antigens could provide longer-lasting protection against multiple influenza subtypes...
  42. pmc Generation of transgene-free induced pluripotent mouse stem cells by the piggyBac transposon
    Kosuke Yusa
    Wellcome Trust Sanger Institute, Wellcome Trust Genome Campus, Hinxton, Cambridge, UK
    Nat Methods 6:363-9. 2009
    ..iPSCs fulfilled all criteria of pluripotency, such as pluripotency gene expression, teratoma formation and contribution to chimeras. piggyBac transposon-based reprogramming may be used to generate therapeutically applicable iPSCs...
  43. pmc A phage integrase directs efficient site-specific integration in human cells
    A C Groth
    Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305 5120, USA
    Proc Natl Acad Sci U S A 97:5995-6000. 2000
    ..These observations form the basis for site-specific integration strategies potentially useful in a broad range of genetic engineering applications...
  44. ncbi Hexon-chimaeric adenovirus serotype 5 vectors circumvent pre-existing anti-vector immunity
    Diane M Roberts
    Division of Viral Pathogenesis, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts 02215, USA
    Nature 441:239-43. 2006
    ..Such chimaeric viral vectors may have important practical implications for vaccination and gene therapy...
  45. ncbi Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
    Corinna Burger
    Department of Molecular Genetics and Microbiology, University of Florida, Gainesville 32610, USA
    Mol Ther 10:302-17. 2004
    ..Retrograde transport of rAAV1 and rAAV5 was also observed in particular CNS areas. These results suggest that vectors based on distinct AAV serotypes can be chosen for specific applications in the nervous system...
  46. ncbi Viral vectors as vaccine platforms: deployment in sight
    Christine S Rollier
    Oxford Vaccine Group, Department of Paediatrics, Center for Clinical Vaccine and Tropical Medicine, University of Oxford, Churchill Hospital, Churchill Drive, Oxford OX3 7LJ, United Kingdom
    Curr Opin Immunol 23:377-82. 2011
  47. pmc A versatile viral system for expression and depletion of proteins in mammalian cells
    Eric Campeau
    Program in Gene Function and Expression, University of Massachusetts Medical School, Worcester, Massachusetts, United States of America
    PLoS ONE 4:e6529. 2009
    ..As new technologies emerge, the vectors in this collection can be easily modified and adapted without the need for extensive recloning...
  48. pmc Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial
    William W Hauswirth
    Department of Ophthalmology, University of Florida, Gainesville, FL 32610, USA
    Hum Gene Ther 19:979-90. 2008
    ..99). Comparisons are drawn between the present work and two other studies of ocular gene therapy for RPE65-LCA that were carried out contemporaneously and reported...
  49. pmc A suite of Gateway cloning vectors for high-throughput genetic analysis in Saccharomyces cerevisiae
    Simon Alberti
    Whitehead Institute for Biomedical Research, Cambridge, MA, USA
    Yeast 24:913-9. 2007
    ..We have made this yeast Gateway vector kit available to the research community via the non-profit Addgene Plasmid Repository (
  50. pmc A versatile ligation-independent cloning method suitable for high-throughput expression screening applications
    Nick S Berrow
    The Oxford Protein Production Facility, Henry Wellcome Building for Genomic Medicine, University of Oxford, Oxford, UK
    Nucleic Acids Res 35:e45. 2007
    ..g. Sf9 cells). These high-throughput screening activities have prompted the development and validation of automated protocols for transfection of mammalian cells and Ni-NTA protein purification...
  51. pmc MicroRNA sponges: competitive inhibitors of small RNAs in mammalian cells
    Margaret S Ebert
    Center for Cancer Research, E17 529B Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA
    Nat Methods 4:721-6. 2007
    ..RNA polymerase II promoter (Pol II)-driven sponges contain a fluorescence reporter gene for identification and sorting of sponge-treated cells. We envision the use of stably expressed sponges in animal models of disease and development...
  52. ncbi Retrovirus-mediated gene transfer and expression cloning: powerful tools in functional genomics
    Toshio Kitamura
    Divisions of Cellular Therapy and Hematopoietic Factors, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan
    Exp Hematol 31:1007-14. 2003
    ..In this review, we describe retrovirus-mediated strategies used for investigation of gene functions and function-based screening strategies...
  53. 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
  54. ncbi Design and development of polymers for gene delivery
    Daniel W Pack
    Department of Chemical and Biomolecular Engineering, University of Illinois, Box C 3, 600 South Mathews Avenue, Urbana, IL 61801, USA
    Nat Rev Drug Discov 4:581-93. 2005
    ..With the growing understanding of polymer gene-delivery mechanisms and continued efforts of creative polymer chemists, it is likely that polymer-based gene-delivery systems will become an important tool for human gene therapy...
  55. pmc Profound early control of highly pathogenic SIV by an effector memory T-cell vaccine
    Scott G Hansen
    Vaccine and Gene Therapy Institute, Department of Molecular Microbiology, Oregon Health and Science University, Beaverton, Oregon 97006, USA
    Nature 473:523-7. 2011
    ..Thus, persistent vectors such as CMV and their associated T(EM) responses might significantly contribute to an efficacious HIV/AIDS vaccine...
  56. pmc Avoiding cytotoxicity of transposases by dose-controlled mRNA delivery
    Melanie Galla
    Department of Experimental Hematology, Institute of Transplantation Immunology, Institute of Cell and Molecular Pathology, Hannover Medical School, Hannover, Germany
    Nucleic Acids Res 39:7147-60. 2011
    ..Transposase-mediated gene transfer can thus be tuned to maintain high efficiency in the absence of overt cell damage...
  57. ncbi Progress and problems with the use of viral vectors for gene therapy
    Clare E Thomas
    Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA
    Nat Rev Genet 4:346-58. 2003
    ..With the development of a leukaemia-like syndrome in two of the small number of patients that have been cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go...
  58. ncbi Many paths to many clones: a comparative look at high-throughput cloning methods
    Gerald Marsischky
    Institute of Proteomics, Harvard Medical School, Department of Biological Chemistry and Molecular Pharmacology, Boston, Massachusetts 02115, USA
    Genome Res 14:2020-8. 2004
    ..Alternative approaches to creating clones involving mixing of cloning methods, including gap-repair cloning, are also explored...
  59. pmc Comparative genomic integration profiling of Sleeping Beauty transposons mobilized with high efficacy from integrase-defective lentiviral vectors in primary human cells
    Brian Moldt
    Department of Human Genetics, University of Aarhus, Aarhus, Denmark
    Mol Ther 19:1499-510. 2011
    ..Based on these properties, HIV-1/SB vectors may become valuable tools for genetic engineering and therapeutic gene transfer...
  60. pmc Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses
    Li Zhong
    Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32610, USA
    Proc Natl Acad Sci U S A 105:7827-32. 2008
    ..These studies have led to the development of AAV vectors that are capable of high-efficiency transduction at lower doses, which has important implications in their use in human gene therapy...
  61. ncbi Effect of gene therapy on visual function in Leber's congenital amaurosis
    James W B Bainbridge
    Institute of Ophthalmology, University College London, London, United Kingdom
    N Engl J Med 358:2231-9. 2008
    ..These findings provide support for further clinical studies of this experimental approach in other patients with mutant RPE65. ( number, NCT00643747 [].)...
  62. pmc Efficient generation of transgene-free human induced pluripotent stem cells (iPSCs) by temperature-sensitive Sendai virus vectors
    Hiroshi Ban
    DNAVEC Corporation, Tsukuba, Ibaraki 300 2611, Japan
    Proc Natl Acad Sci U S A 108:14234-9. 2011
    ..We suggest that generation of transgene-free iPSCs from cord blood cells should be an important step in providing allogeneic iPSC-derived therapy in the future...
  63. ncbi Induction of broadly neutralizing H1N1 influenza antibodies by vaccination
    Chih Jen Wei
    Vaccine Research Center, National Institute of Allergy and Infectious Diseases NIAID, National Institutes of Health NIH, Bethesda, MD 20892 3005, USA
    Science 329:1060-4. 2010
    ..Cross-neutralization of H1N1 subtypes elicited by this approach provides a basis for the development of a universal influenza vaccine for humans...
  64. pmc Transposon vectors containing non-antibiotic resistance selection markers for cloning and stable chromosomal insertion of foreign genes in gram-negative bacteria
    M Herrero
    GBF National Research Centre for Biotechnology, Braunschweig, Federal Republic of Germany
    J Bacteriol 172:6557-67. 1990
    ..g., in open systems and natural environments), and that do not carry antibiotic resistance markers characteristic of most available cloning vectors (as is currently required of live bacterial vaccines)...
  65. pmc piggyBac transposition reprograms fibroblasts to induced pluripotent stem cells
    Knut Woltjen
    Samuel Lunenfeld Research Institute, Mount Sinai Hospital, Toronto, Ontario M5G 1X5, Canada
    Nature 458:766-70. 2009
    ..We anticipate that the unique properties of this virus-independent simplification of iPS cell production will accelerate this field further towards full exploration of the reprogramming process and future cell-based therapies...
  66. pmc Innate immune sensing of modified vaccinia virus Ankara (MVA) is mediated by TLR2-TLR6, MDA-5 and the NALP3 inflammasome
    Julie Delaloye
    Department of Medicine, Infectious Diseases Service, Centre Hospitalier Universitaire Vaudois and University of Lausanne, Lausanne, Switzerland
    PLoS Pathog 5:e1000480. 2009
    ..Delineation of the host response induced by MVA is critical for improving our understanding of poxvirus antiviral escape mechanisms and for designing new MVA vaccine vectors with improved immunogenicity...
  67. ncbi Production and characterization of adeno-associated viral vectors
    Joshua C Grieger
    Curriculum in Genetics and Molecular Biology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, USA
    Nat Protoc 1:1412-28. 2006
    ..This review provides an up-to-date, detailed description of essential methods such as production, purification and titering and their application to characterize current AAV vectors for preclinical and clinical use...
  68. pmc A high-efficiency system for the generation and study of human induced pluripotent stem cells
    Nimet Maherali
    Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute, Massachusetts General Hospital Center for Regenerative Medicine, Massachusetts General Hospital Cancer Center, Boston, MA 02114, USA
    Cell Stem Cell 3:340-5. 2008
    ..The ability to reprogram cells at high efficiency provides a unique platform to dissect the underlying molecular and biochemical processes that accompany nuclear reprogramming...
  69. ncbi Expression of heterologous proteins in Pichia pastoris: a useful experimental tool in protein engineering and production
    Rachel Daly
    ARC Special Research Centre for Green Chemistry, Monash University, Building 23, Wellington Road, Clayton, Victoria 3800, Australia
    J Mol Recognit 18:119-38. 2005
    ..Procedures to prevent glycosylation through manipulation of cell culture conditions or via enzymatic and site-directed mutagenesis methods are also discussed...
  70. pmc Refinement of tools for targeted gene expression in Drosophila
    Barret D Pfeiffer
    Janelia Farm Research Campus, Howard Hughes Medical Institute, Ashburn, VA 20147, USA
    Genetics 186:735-55. 2010
    ..The increased strength and reliability of these optimized reagents overcome many of the previous limitations of these methods and will facilitate genetic manipulations of greater complexity and sophistication...
  71. pmc Overall survival analysis of a phase II randomized controlled trial of a Poxviral-based PSA-targeted immunotherapy in metastatic castration-resistant prostate cancer
    Philip W Kantoff
    Medical Director, BN ImmunoTherapeutics, 2425 Garcia Ave, Mountain View, CA 94043, USA
    J Clin Oncol 28:1099-105. 2010
    ..PROSTVAC-VF treatment was evaluated for safety and for prolongation of progression-free survival (PFS) and overall survival (OS) in a randomized, controlled, and blinded phase II study...
  72. pmc Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
    Francesca Simonelli
    Department of Ophthalmology, Second University of Naples, Naples, Italy
    Mol Ther 18:643-50. 2010
    ..The safety of the intervention and the stability of the improvement in visual and retinal function in these subjects support the use of AAV-mediated gene augmentation therapy for treatment of inherited retinal diseases...
  73. pmc Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates
    Lluis Samaranch
    Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA
    Hum Gene Ther 23:382-9. 2012
    ..Last, we have demonstrated that delivery of AAV9 into the CSF does not shield against AAV antibodies. This has obvious implications when developing and/or implementing any clinical trial studies...
  74. 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...
  75. pmc Phase Ia clinical evaluation of the Plasmodium falciparum blood-stage antigen MSP1 in ChAd63 and MVA vaccine vectors
    Susanne H Sheehy
    Centre for Clinical Vaccinology and Tropical Medicine, The Jenner Institute, University of Oxford, Churchill Hospital, Oxford, UK
    Mol Ther 19:2269-76. 2011
    ..Further studies are required to assess whether this strategy can achieve protective efficacy against blood-stage malaria infection...
  76. pmc Disruption of overlapping transcripts in the ROSA beta geo 26 gene trap strain leads to widespread expression of beta-galactosidase in mouse embryos and hematopoietic cells
    B P Zambrowicz
    Division of Basic Sciences, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA
    Proc Natl Acad Sci U S A 94:3789-94. 1997
    ..This third transcript potentially encodes a novel protein of at least 505 amino acids that is conserved in humans and in Caenorhabditis elegans...
  77. pmc Cre reporter strains produced by targeted insertion of EYFP and ECFP into the ROSA26 locus
    S Srinivas
    Department of Genetics and Development, Columbia University, New York, USA
    BMC Dev Biol 1:4. 2001
  78. pmc New insights on adenovirus as vaccine vectors
    Marcio O Lasaro
    The Wistar Institute Vaccine Center, Philadelphia, Pennsylvania 19104, USA
    Mol Ther 17:1333-9. 2009
    ..In summary, although Ad vectors have seen their share of setbacks in recent years, they remain viable tools for prevention or treatment of a multitude of diseases...
  79. ncbi RF cloning: a restriction-free method for inserting target genes into plasmids
    Fusinita van den Ent
    MRC Laboratory of Molecular Biology, Hills Road, Cambridge CB2 2QH, UK
    J Biochem Biophys Methods 67:67-74. 2006
    ..The absence of any alterations to the protein as well as the simplicity of both the primer design and the procedure itself makes it suitable for high-throughput expression and ideal for structural genomics...
  80. pmc Analysis of mutation in human cells by using an Epstein-Barr virus shuttle system
    R B DuBridge
    Mol Cell Biol 7:379-87. 1987
    ..A total of 33 of 34 lacI nonsense mutations and 26 of 27 missense mutations involve G X C to A X T transitions. These data provide support for the mutational theory of cancer...
  81. ncbi DNA sequence quality trimming and vector removal
    H H Chou
    Department of Zoology and Genetics, Department of Computer Science, Iowa State University, Ames, IA 50011, USA
    Bioinformatics 17:1093-104. 2001
    ..This step is necessary before other genomic data processing stages can be carried out, such as fragment assembly or EST clustering. A specialized tool is therefore needed to solve this apparent dilemma...
  82. pmc Conditional-replication, integration, excision, and retrieval plasmid-host systems for gene structure-function studies of bacteria
    A Haldimann
    Department of Biological Sciences, Purdue University, West Lafayette, Indiana 47907, USA
    J Bacteriol 183:6384-93. 2001
    ..These helper plasmids facilitate integration, excision ("curing"), or retrieval of the CRIM plasmids...
  83. pmc Reprogramming of murine and human somatic cells using a single polycistronic vector
    Bryce W Carey
    Whitehead Institute for Biomedical Research and Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02142, USA
    Proc Natl Acad Sci U S A 106:157-62. 2009
    ..In addition we have generated human induced pluripotent stem (hiPS) cell lines from human keratinocytes, demonstrating that a single polycistronic virus can reprogram human somatic cells...
  84. pmc Adenovirus-5-vectored P. falciparum vaccine expressing CSP and AMA1. Part B: safety, immunogenicity and protective efficacy of the CSP component
    Cindy Tamminga
    U S Military Malaria Vaccine Program, Naval Medical Research Center, Silver Spring, Maryland, United States of America
    PLoS ONE 6:e25868. 2011
    ..As adenovirus vaccine vectors induce both these responses in humans, a Phase 1/2a clinical trial was conducted to evaluate the efficacy of an adenovirus serotype 5-vectored malaria vaccine against sporozoite challenge...
  85. ncbi Prime-boost vectored malaria vaccines: progress and prospects
    Adrian V S Hill
    The Jenner Institute, University of Oxford, Oxford, UK
    Hum Vaccin 6:78-83. 2010
    ..These viral vectors now provide a major option for inclusion in a high efficacy multi-stage malaria vaccine that should achieve deployable levels of efficacy in endemic settings...
  86. ncbi A Tn7-based broad-range bacterial cloning and expression system
    Kyoung Hee Choi
    Department of Microbiology, Immunology and Pathology, Colorado State University, Fort Collins, Colorado 80523, USA
    Nat Methods 2:443-8. 2005
    ..pestis. This system will thus have widespread biomedical and environmental applications, especially in environments where plasmids and antibiotic selection are not feasible, namely in plant and animal models or biofilms...
  87. ncbi Baculovirus as a gene delivery vector: recent understandings of molecular alterations in transduced cells and latest applications
    Chi Yuan Chen
    Department of Chemical Engineering, National Tsing Hua University, Hsinchu 30013, Taiwan
    Biotechnol Adv 29:618-31. 2011
  88. 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
  89. ncbi MVA and NYVAC as vaccines against emergent infectious diseases and cancer
    Carmen E Gomez
    Department of Molecular and Cellular Biology, Centro Nacional de Biotecnologia, CSIC, Ciudad Universitaria Cantoblanco, Madrid, Spain
    Curr Gene Ther 11:189-217. 2011
  90. ncbi Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors
    Sylvie Boutin
    Laboratoire d Immunologie, Genethon R and D, Evry Cedex, France
    Hum Gene Ther 21:704-12. 2010
    ..Better characterization of the preexisting humoral responses to the AAV capsid and cross-reactivity will allow development of new strategies to circumvent AAV acquired immune responses...
  91. ncbi Optogenetic interrogation of neural circuits: technology for probing mammalian brain structures
    Feng Zhang
    Department of Bioengineering, Stanford University, Stanford, California, USA
    Nat Protoc 5:439-56. 2010
    ..Together, these methods may help in providing circuit-level insight into the dynamics underlying complex mammalian behaviors in health and disease...
  92. ncbi Development of series of gateway binary vectors, pGWBs, for realizing efficient construction of fusion genes for plant transformation
    Tsuyoshi Nakagawa
    Department of Molecular and Functional Genomics, Center for Integrated Research in Science, Shimane University, 1060 Nishikawatsu, Matsue, Japan
    J Biosci Bioeng 104:34-41. 2007
    ..Moreover, tissue-specific and inducible gene expression using a promoter was also monitored with pGWBs. It is expected that, the pGWB system will serve as a powerful tool for plasmid construction in plant research...
  93. 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...
  94. pmc Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year
    Artur V Cideciyan
    Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
    Hum Gene Ther 20:999-1004. 2009
    ..The safety and efficacy of human retinal gene transfer with rAAV2-RPE65 vector extends to at least 1 year posttreatment...
  95. ncbi Systemic Agrobacterium tumefaciens-mediated transfection of viral replicons for efficient transient expression in plants
    Sylvestre Marillonnet
    Icon Genetics, Biozentrum Halle, Weinbergweg 22, D 06120 Halle Saale, Germany
    Nat Biotechnol 23:718-23. 2005
    ..tumefaciens, the high expression yield obtained with viral vectors, and the post-translational capabilities of a plant), does not require genetic modification of plants and is faster than other existing methods...
  96. pmc The role of heterologous chloroplast sequence elements in transgene integration and expression
    Tracey Ruhlman
    Department of Molecular Biology and Microbiology, College of Medicine, University of Central Florida, Orlando, Florida 32816 2364, USA
    Plant Physiol 152:2088-104. 2010
  97. pmc Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection
    Boris Kantor
    Gene Therapy Center, University of North Carolina School of Medicine, Chapel Hill, NC 27514, USA
    Proc Natl Acad Sci U S A 106:18786-91. 2009
    ..Finally, we propose a mechanism describing the role of episomal HIV-1 forms in the viral life cycle in a SCFA-rich gut environment...
  98. ncbi Oncolytic (replication-competent) adenoviruses as anticancer agents
    Karoly Toth
    Department of Molecular Microbiology and Immunology, Saint Louis University School of Medicine, St Louis, Missouri, USA
    Expert Opin Biol Ther 10:353-68. 2010
    ..One option is genetically-engineered oncolytic adenovirus (Ad) 'vectors'. These kill cancer cells via the viral replication cycle, and amplify the anti-tumor effect by producing progeny virions able to infect neighboring tumor cells...
  99. ncbi Functional expression of secreted proteins from a bicistronic retroviral cassette based on foot-and-mouth disease virus 2A can be position dependent
    Dominic G Rothwell
    Cancer Research UK Department of Medical Oncology, School of Cancer and Imaging Sciences, University of Manchester, Manchester Academic Health Science Centre, Christie NHS Trust, Manchester M20 4BX, United Kingdom
    Hum Gene Ther 21:1631-7. 2010
    ..These results highlight that to achieve functional expression of secreted proteins consideration must be taken of the transgenic protein's posttranslational modification and trafficking when using 2A-based bicistronic cassettes...
  100. ncbi Gene therapy for immunodeficiency due to adenosine deaminase deficiency
    Alessandro Aiuti
    San Raffaele Telethon Institute for Gene Therapy, Milan, Italy
    N Engl J Med 360:447-58. 2009
    ..We investigated the long-term outcome of gene therapy for severe combined immunodeficiency (SCID) due to the lack of adenosine deaminase (ADA), a fatal disorder of purine metabolism and immunodeficiency...
  101. pmc Frequent endonuclease cleavage at off-target locations in vivo
    Lisa M Petek
    Department of Pediatrics, Division of Genetic Medicine, University of Washington, Seattle, Washington, USA
    Mol Ther 18:983-6. 2010

Research Grants79

  1. Small Arteries as a Therapeutic Target in AD
    Nienwen Chow; Fiscal Year: 2004
    ..Potential products to be ultimately developed are new drugs or genetic vectors that would affect SRF/myocardin function in small arteries in AD with a goal to improve CBF regulations by ..
    MEAD MCCABE; Fiscal Year: 1993
    ..b>Genetic Vectors has developed a chemical procedure for incorporation of detectable labels into nucleic acids...
  3. CNS Cellular Senescence/Targeting Endothelial SIPS
    Nienwen Chow; Fiscal Year: 2004
    ..The potential products ultimately to be developed are therapeutic drugs and genetic vectors to selectively correct dysfunctions of senescent brain endothelium in neurodegenerative disorders associated ..
    David Jacoby; Fiscal Year: 1999
    The development of genetic vectors that can deliver therapeutic DNA sequences to the central nervous system (CNS) in situ promises to be a powerful means of intervention in neurologic diseases, many of which have not current form of ..
  5. Target Assessment in Alzheimer's Neurovasculature/BBB
    Jan Sallstrom; Fiscal Year: 2004
    ..The potential products include new diagnostics DNA chips and brain imaging agents, therapeutic genetic vectors and new drugs to treat neurovascular AD disorder.
  6. Cellular physical properties in relation to cancer
    William Craelius; Fiscal Year: 2002 standard cytometers that will be useful for researchers screening potential anti- angiogenic drugs or genetic vectors, and for clinicians to biopsy specimens...
  7. Gene delivery to striated muscle by systemic AAV vectors
    Dwight D Koeberl; Fiscal Year: 2010
    ..Efficacious muscle-targeted gene therapy in GSD-II will have implications for gene therapy in other muscular dystrophies and myopathies. ..
    Katherine High; Fiscal Year: 2001
    ..These studies will involve a collaboration between the P.I.'s lab and the laboratory of Dr. Hildegund Ertl, an immunologist with experience in characterizing immune responses in the setting of viral vectors. ..
  9. Gene Therapy for hemophilia using muscle-expressed FVIIa
    Katherine A High; Fiscal Year: 2011
    ..The proposed work will allow us to increase the therapeutic applicability of our approach and enhance its safety, prior to a clinical application. ..
  10. Mechanisms for immune tolerance in Pompe Disease
    Dwight D Koeberl; Fiscal Year: 2010
    ..These comparisons will guide preclinical experiments to further immunomodulatory gene therapy in Pompe disease and other lysosomal storage disorders. ..
  11. Nanoparticle & AAV Approaches to Rapid Onset, Stable Retinal Gene Therapy
    Jijing Pang; Fiscal Year: 2008
    ..Our ultimate goal is to establish a retinal gene delivery system that is fast, of long duration and can accommodate large therapeutic cDNAs. [unreadable] [unreadable] [unreadable]..
    David Scott; Fiscal Year: 2001
    ..The results of these studies will provide information on regulation of neoplastic growth as well as mechanism for inducing apoptosis in a model for deletional tolerance. ..
  13. Development of an VEE Replicon Vaccine Against Smallpox
    Kurt Kamrud; Fiscal Year: 2005
    David Scott; Fiscal Year: 2003
    ..abstract_text> ..
  15. Mechanism of B-cell Delivered Tolerance in Diabetes
    David Scott; Fiscal Year: 2009
    ..Our goal is to modulate pathogenic responses to GAD, for example, as a prelude to applying this protocol as a complementary ..
  16. Directed Evolution of Adeno-Associated Virus Vectors for Seizure Gene Therapy
    Thomas McCown; Fiscal Year: 2008
    ..If successful, this novel approach could greatly advance the application of gene therapy to the treatment of neurological disorders. [unreadable] [unreadable] [unreadable] [unreadable]..
    RICHARD MORAN; Fiscal Year: 2008
    ..These studies are a logical extension of past studies supported by this grant, but also represent new directions to therapeutics suggested by recent advances in cancer cell biology. ..
  18. Evaluation of High-Capacity Adenovectors in the Eye
    Joseph Bruder; Fiscal Year: 2006
    ..The long-term goal of this proposal is the clinical testing and commercialization of PEDF expressing HC vector for the treatment of AMD and other ocular neovascular diseases. [unreadable] [unreadable]..
  19. Vaccination with Regulatory T Cell Depletion
    Michael Morse; Fiscal Year: 2007
    ..Future studies will assess whether the greater magnitude and durability of the CEA-specific immune responses will translate into a long-term clinical benefit. [unreadable] [unreadable] [unreadable]..
  20. Engineering transgenic silkworms to produce spider silk fibers
    Donald Jarvis; Fiscal Year: 2008
    ..These projects will exploit current knowledge of specific peptide motifs contributing tensile strength or elasticity to spider silks. [unreadable] [unreadable] [unreadable]..
  21. Gene Therapy for Usher Syndrome (USH1C)
    Jean Bennett; Fiscal Year: 2008
    ..unreadable] [unreadable] [unreadable]..
    Kenneth Cornetta; Fiscal Year: 2007
    ..abstract_text> ..
  23. Molecular Basis of Flavivirus Neurovirulence
    William Wold; Fiscal Year: 2007
    ..abstract_text> ..
    RICHARD MORAN; Fiscal Year: 2008
    ..abstract_text> ..
  25. Immunoglobulin Allotypes in Hepatitis C Virus Infection
    JANARDAN PANDEY; Fiscal Year: 2008
    ..Results of this investigation will advance our understanding of the role of host genetic factors in clearance and persistence of hepatitis C virus infection. ..
  26. A Biological Basis for Repair of the ACL
    Christopher Evans; Fiscal Year: 2009
    ..MRI, histology and immunohistochemistry will also be used to evaluate the healed ligament. These studies will advance our knowledge of the biology of the injured ACL and suggest novel, biologically-based approaches to healing. ..
    GEORGE ZUBENKO; Fiscal Year: 2009
    ..abstract_text> ..
    Wei Li; Fiscal Year: 2010
    ..These efforts will advance our understanding of the disease etiology and prognosis, and improve the diagnosis and therapy. ..
    Katherine P Ponder; Fiscal Year: 2010
    ..These studies may demonstrate that the development of disease in the aorta and the cervical spine in MPS is due to a common mechanism, and may identify a therapy for these difficult-to-treat sites. ..
  30. Genetic Regulatory Network Controlling Vertebrate Eye Formation
    Michael E Zuber; Fiscal Year: 2010
    ..Identification of the genes and genetic pathways in the developing eye will provide a much-needed understanding of the genetic mechanisms underlying eye formation and provide a window into understanding and treating human blindness. ..
  31. A novel transgenic silkworm system for recombinant glycoprotein production
    Donald L Jarvis; Fiscal Year: 2010
    ..Finally, these results will be of great interest to bioengineers working to overcome the evolutionary limitations of lower eukaryotic systems for recombinant glycoprotein production. ..
  32. Molecular Genetics of Cadmium Toxicity
    Daniel W Nebert; Fiscal Year: 2010
    J Thomas Cunningham; Fiscal Year: 2010
    ..Benefits: The results will provide new information regarding the control of vasopressin secretion and how these systems contribute to pathophysiology. ..
    Donald L Jarvis; Fiscal Year: 2010
  35. Tumor Sensitization to Purine Analogs by E. coli PNP
    Eric J Sorscher; Fiscal Year: 2010
  36. Hamster Model for Oncolytic Adenovirus Vectors
    WILLIAM SM WOLD; Fiscal Year: 2010
    ..Although not proposed specifically in this application, these studies should also provide novel information on adenovirus pathogenesis. ..
  37. PAHs: Balance of Detoxication vs Metabolic Activation
    Daniel Nebert; Fiscal Year: 2009
    ..dissemination of ingested BaP and will be informative in clinical studies in which we would determine which haplotypes of these three human genes might be associated with resistance vs. sensitivity to PAH-induced toxicity and cancer. ..
  38. Transfer of IL-1Ra cDNA to Osteoarthritic Knee
    Christopher Evans; Fiscal Year: 2008
    ..abstract_text> ..
  39. (SCOR) Mechanistic Studies of CF Pathogenesis and CL- S*
    Eric Sorscher; Fiscal Year: 2006
    ..In summary, we propose an aggressive, multi-pronged attack aimed at developing a comprehensive and mechanistic understanding of CI- secretion in CF pathophysiology. ..
  40. Gene Therapy of Autosomal Recessive RP
    Simon Petersen Jones; Fiscal Year: 2006
    ..abstract_text> ..
    KATHERINE PONDER; Fiscal Year: 2005
    ..Success in this project might lead to a safe, effective, and permanent therapy for Hemophilia B. ..
    Daniel Nebert; Fiscal Year: 2005
    ..abstract_text> ..
  43. Animal Model for Adenovirus Oncolytic Vectors
    William Wold; Fiscal Year: 2004
    ..We will inject subcutaneous tumors with vectors and evaluate the efficacy and safety of these vectors by gross pathology, immunohistochemistry, and histopathology. ..
  44. Animal Model for a Hereditary Macular Degeneration
    Jean Bennett; Fiscal Year: 2004
    ..These studies aim to reveal specific biological pathways relevant to the pathology in this animal model. Any such pathways could potentially be manipulated in the future to slow/prevent the disease process. ..
  45. Modulation of CD4+ T cells in autoimmune uveitis
    Wei Li; Fiscal Year: 2003
    ..These studies will not only yield new insights for the underlying causes and pathogenesis of autoimmune uveitis, but may also lead to a more selective immunotherapy for the disease. ..
    GEORGE ZUBENKO; Fiscal Year: 2003
    GEORGE ZUBENKO; Fiscal Year: 2002
    ..A timetable is proposed for rapid sharing of all biological materials, blinded clinical data, genotypes and linkage analyses with the scientific community through the NIMH-sponsored Center for Genetic Studies. ..
    Barry Byrne; Fiscal Year: 2002
    ..These studies will yield important new information in establishing a clinically relevant treatment for these fatal diseases and add new understanding to the basic pathophysiology of GSD. ..
    David Waxman; Fiscal Year: 2002
    Daniel Nebert; Fiscal Year: 2002
    ..S. population. ..
    Michael Morse; Fiscal Year: 2002
    ..This clinical trial will form the background for further trials designed to demonstrate both immunology and clinical benefits of dexosome-based immunotherapy. ..
    Stephen Russell; Fiscal Year: 2002
    ..abstract_text> ..
    Donald Kohn; Fiscal Year: 2001
    ..Successful tolerance induction in murine and canine models of MPS I will be an Important step toward application of gene therapy in human patients. ..
    J Cunningham; Fiscal Year: 2005
    ..Additionally, these data may contribute to understanding of health problems associated with an aging population and with cardiovascular disease such as congestive heart failure. ..
  55. Syrian Hamster as a Permissive Model for Testing Anti-Adenovirus Drugs
    William Wold; Fiscal Year: 2007
    ..Success in this project could lead to the eventual approval of drugs to treat adenovirus infections in immunosuppressed patients. [unreadable] [unreadable] [unreadable]..
    JON REUTER; Fiscal Year: 2006
    ..abstract_text> ..
  57. BD Biosciences FACS Canto Flow Cytometer
    LAWRENCE LAMB; Fiscal Year: 2006
    ..The laboratory will require an instrument that will serve the laboratory in the development and execution of advanced cell therapy trials. [unreadable] [unreadable]..
    William Wold; Fiscal Year: 2001
  59. Postsynthetic Modifications of Bacterial Membrane Lipids
    John Cronan; Fiscal Year: 2006
    ..These processes will be studied by a combination of biochemical, genetic, and molecular biological approaches including tests of pathogenesis in a mouse model system ..
  60. Viral Mediated Gene Therapy for Retinal Diseases
    John Flannery; Fiscal Year: 2005
    ..In summary, this application supports key, initial "proof-of-principle" experiments to create retina-specific viral vectors and systems to transfer neurotrophic factors for gene therapy of retinal degeneration. ..
  61. Vaccine Induced Mucosal Protection Against TB Infection
    DANIEL HOFT; Fiscal Year: 2005
    ..Prime/boosting strategies found to prevent BCG infection after aerosol challenges will be studied for the ability to prevent infection with virulent M. tuberculosis by aerosol challenge. ..
    Donald Kohn; Fiscal Year: 2005
    ..This "intracellular immunization" approach may provide a therapeutic measure against HIV-1 that adds to pre-existing therapies, such as anti-retroviral drugs. ..
  63. Novel Neuronal Transport Mechanisms of Lyosomal Enzymes
    Mark Sands; Fiscal Year: 2004
    ..abstract_text> ..
    DANIEL HOFT; Fiscal Year: 2008
    ..Chemokine and/or integrin receptor expression will be studied by flow cytometry and RT-Real-Time PCR, and the in vivo effects of defective chemokine and integrin binding and/or signaling will be evaluated. [unreadable] [unreadable]..
    William Wold; Fiscal Year: 2002
  66. HSV Amplicon Vectors for Cerebral Ischemia
    Ennio Chiocca; Fiscal Year: 2004
    ..abstract_text> ..
  67. AAV-mediated gene correction in retina
    Jean Bennett; Fiscal Year: 2007
    ..unreadable] [unreadable]..
    Thomas J McCown; Fiscal Year: 2011
    ..The present proposed studies will define the most efficacious adeno-associated virus vector which will provide a viable gene therapy treatment for intractable epilepsy. ..
    Paul Robbins; Fiscal Year: 2002
    ..The experiments described in the proposal are expected to provide important insights into the mechanism of how RB and cyclin D1 function to regulate transcription directly, cell differentiation, and tumorigenesis. ..
  70. Effect of HIV-1 Vpr on Basic Cellular Functions (II)
    Richard Zhao; Fiscal Year: 2007
    ..abstract_text> ..
    Ranjit Ray; Fiscal Year: 2009
    ..Our proposed complimentary experimental approaches in this vaccinated cohort will help in developing successful vaccine strategies for the intervention of the first crucial step of the HCV life cycle. ..
  72. 2008 Viral Vectors for Gene Therapy, The Science of Gordon Research Conference
    Paul Robbins; Fiscal Year: 2008
    ..End of Abstract) [unreadable] [unreadable] [unreadable]..
  73. Inhibition of NF-KB to Facilitate Islet Transplantation
    Paul Robbins; Fiscal Year: 2003
    ..The successful completion of the proposed studies should determine if NF-kB inhibition in islets by peptide and gene mediated transduction is able to improve islet viability prior to and post-transplantation. ..
  74. Study of Leptin Receptor Deficiency in Fat
    Allan Zhao; Fiscal Year: 2007
    ..We believe that these studies will provide a possible molecular linkage between adipocyte-selective leptin receptor deficiency and type 2 diabetes. ..
  75. S. typhimiurium Vaccine Against Bacterial Enteropathogens
    Roy Curtiss; Fiscal Year: 2007
    ..abstract_text> ..
  76. Mesenchymal Stem Cells for Fetal Gene Delivery
    Alice Tarantal; Fiscal Year: 2006
    ..abstract_text> ..
  77. Hantavirus Vaccines Based On Nonreplicating Adenoviruses
    David Johnson; Fiscal Year: 2005
    ..This will be a prelude to challenge experiments in which vaccinated hamsters will be challenged with Andes virus that causes a lethal pulmonary disease similar to that in humans. ..