Summary: Genes that are introduced into an organism using GENE TRANSFER TECHNIQUES.

Top Publications

  1. pmc An optimized transgenesis system for Drosophila using germ-line-specific phiC31 integrases
    Johannes Bischof
    Frontiers in Genetics, National Center of Competence in Research, Institute of Molecular Biology, University of Zurich, Winterthurerstrasse 190, CH 8057 Zurich, Switzerland
    Proc Natl Acad Sci U S A 104:3312-7. 2007
  2. ncbi Mosaic analysis with a repressible cell marker for studies of gene function in neuronal morphogenesis
    T Lee
    Department of Biological Sciences, Stanford University, California 94305, USA
    Neuron 22:451-61. 1999
  3. pmc Single-copy insertion of transgenes in Caenorhabditis elegans
    Christian Frøkjaer-Jensen
    Department of Biology and Howard Hughes Medical Institute, University of Utah, 257 South 1400 East, Salt Lake City, Utah 84112, USA
    Nat Genet 40:1375-83. 2008
  4. 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
  5. 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
  6. 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
  7. 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
  8. ncbi A transposon-mediated gene trap approach identifies developmentally regulated genes in zebrafish
    Koichi Kawakami
    Division of Molecular and Developmental Biology, National Institute of Genetics, 1111 Yata, Mishima, Shizuoka 411 8540, Japan
    Dev Cell 7:133-44. 2004
  9. 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
  10. ncbi Co-expression of multiple transgenes in mouse CNS: a comparison of strategies
    J L Jankowsky
    Department of Pathology, Johns Hopkins School of Medicine, 720 Rutland Ave, 558 Ross Research Building, Baltimore, MD 21205, USA
    Biomol Eng 17:157-65. 2001

Detail Information

Publications346 found, 100 shown here

  1. pmc An optimized transgenesis system for Drosophila using germ-line-specific phiC31 integrases
    Johannes Bischof
    Frontiers in Genetics, National Center of Competence in Research, Institute of Molecular Biology, University of Zurich, Winterthurerstrasse 190, CH 8057 Zurich, Switzerland
    Proc Natl Acad Sci U S A 104:3312-7. 2007
    ..We generated a collection of lines with precisely mapped attP sites that allow the insertion of transgenes into many different predetermined intergenic locations throughout the fly genome...
  2. ncbi Mosaic analysis with a repressible cell marker for studies of gene function in neuronal morphogenesis
    T Lee
    Department of Biological Sciences, Stanford University, California 94305, USA
    Neuron 22:451-61. 1999
    ..As an example, we show that the short stop gene is required in mushroom body neurons for the extension and guidance of their axons...
  3. pmc Single-copy insertion of transgenes in Caenorhabditis elegans
    Christian Frøkjaer-Jensen
    Department of Biology and Howard Hughes Medical Institute, University of Utah, 257 South 1400 East, Salt Lake City, Utah 84112, USA
    Nat Genet 40:1375-83. 2008
    At present, transgenes in Caenorhabditis elegans are generated by injecting DNA into the germline. The DNA assembles into a semistable extrachromosomal array composed of many copies of injected DNA...
  4. 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...
  5. 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...
  6. 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...
  7. 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...
  8. ncbi A transposon-mediated gene trap approach identifies developmentally regulated genes in zebrafish
    Koichi Kawakami
    Division of Molecular and Developmental Biology, National Institute of Genetics, 1111 Yata, Mishima, Shizuoka 411 8540, Japan
    Dev Cell 7:133-44. 2004
    ..We propose our gene trap approach should facilitate studies of vertebrate development and organogenesis...
  9. 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
    ..required ZFN cleavage and homologous template DNA, thus establishing a platform that can target the insertion of transgenes into a predetermined genomic site...
  10. ncbi Co-expression of multiple transgenes in mouse CNS: a comparison of strategies
    J L Jankowsky
    Department of Pathology, Johns Hopkins School of Medicine, 720 Rutland Ave, 558 Ross Research Building, Baltimore, MD 21205, USA
    Biomol Eng 17:157-65. 2001
    The introduction of two transgenes into one animal is increasingly common as transgenic experiments become more sophisticated. In this study we examine two strategies for creating double transgenic founders from a single microinjection...
  11. pmc mpeg1 promoter transgenes direct macrophage-lineage expression in zebrafish
    Felix Ellett
    Cancer and Haematology Division, Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia
    Blood 117:e49-56. 2011
    ..remove this roadblock, a macrophage-specific marker was identified (mpeg1) and its promoter used in mpeg1-driven transgenes. mpeg1-driven transgenes are expressed in macrophage-lineage cells that do not express neutrophil-marking ..
  12. ncbi Systemic RNAi in C. elegans requires the putative transmembrane protein SID-1
    William M Winston
    Department of Molecular and Cellular Biology, Harvard University, 16 Divinity Avenue, Cambridge, MA 02138, USA
    Science 295:2456-9. 2002
    ..One of these loci, sid-1, encodes a conserved protein with predicted transmembrane domains. SID-1 is expressed in cells sensitive to RNAi, is localized to the cell periphery, and is required cell-autonomously for systemic RNAi...
  13. 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...
  14. pmc The Q system: a repressible binary system for transgene expression, lineage tracing, and mosaic analysis
    Christopher J Potter
    Howard Hughes Medical Institute, Stanford University, Stanford, CA 94305, USA
    Cell 141:536-48. 2010
    ..The Q system can be expanded to other uses in Drosophila and to any organism conducive to transgenesis...
  15. pmc An endogenous, systemic RNAi pathway in plants
    Patrice Dunoyer
    Institut de Biologie Moleculaire des Plantes du CNRS, UPR2357, Universite de Strasbourg, Strasbourg Cedex, France
    EMBO J 29:1699-712. 2010
    ..Collectively, these results define the existence of a bona fide, endogenous and systemic RNAi pathway in plants that may have implications in adaptation, epiallelism and trans-generational memory...
  16. 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...
  17. 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...
  18. ncbi Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
    Carmela Zincarelli
    Center for Translational Medicine, Department of Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania 19107, USA
    Mol Ther 16:1073-80. 2008
    ..AAV6 expression was observed in the heart, liver, and skeletal muscle, and the genome distribution corroborated these observations...
  19. 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...
  20. 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...
  21. ncbi Regulation of heterochromatic silencing and histone H3 lysine-9 methylation by RNAi
    Thomas A Volpe
    Cold Spring Harbor Laboratory, Watson School of Biological Sciences, Cold Spring Harbor, NY 11724, USA
    Science 297:1833-7. 2002
    ..This is accompanied by transcriptional de-repression of transgenes integrated at the centromere, loss of histone H3 lysine-9 methylation, and impairment of centromere function...
  22. ncbi RNA polymerase IV directs silencing of endogenous DNA
    A J Herr
    Sainsbury Laboratory, John Innes Centre, Norwich NR4 7UH, UK
    Science 308:118-20. 2005
    ..The existence of this distinct silencing polymerase may explain the paradoxical involvement of an RNA silencing pathway in maintenance of transcriptional silencing...
  23. ncbi PiggyBac transposon-mediated gene transfer in human cells
    Matthew H Wilson
    1Division of Genetic Medicine, Department of Medicine, Vanderbilt University, Nashville, Tennessee, USA
    Mol Ther 15:139-45. 2007
    Transposons are mobile genetic elements that can be used to integrate transgenes into host cell genomes. The piggyBac transposon system has been used for transgenesis of insects and for germline mutagenesis in mice...
  24. ncbi Imaging neuronal subsets in transgenic mice expressing multiple spectral variants of GFP
    G Feng
    Department of Anatomy and Neurobiology, Washington University School of Medicine, St Louis, Missouri 63110, USA
    Neuron 28:41-51. 2000
    ..In some lines, intense labeling of small neuronal subsets provided a Golgi-like vital stain. In double transgenic mice expressing two different XFPs, it was possible to differentially label 3 neuronal subsets in a single animal...
  25. 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
    ..strategies involve retroviral, lentiviral, adenoviral and plasmid transfection to deliver reprogramming factor transgenes. Although the latter two methods are transient and minimize the potential for insertion mutagenesis, they are ..
  26. ncbi Role of the arabidopsis DRM methyltransferases in de novo DNA methylation and gene silencing
    Xiaofeng Cao
    Department of Molecular, Cell, and Developmental Biology, University of California, Los Angeles 90095, USA
    Curr Biol 12:1138-44. 2002
    ..Thus, drm mutants prevent the establishment but not the maintenance of gene silencing at FWA and SUP, suggesting that the DRMs encode the major de novo methylation enzymes affecting these genes...
  27. 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...
  28. ncbi A shift from reversible to irreversible X inactivation is triggered during ES cell differentiation
    A Wutz
    Whitehead Institute for Biomedical Research, Cambridge, Massachusetts 02142, USA
    Mol Cell 5:695-705. 2000
    ..Upon differentiation, autosomal transgenes did not effect counting, but transgenic Xist RNA induced late replication and histone H4 hypoacetylation...
  29. pmc Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale
    Martin Lock
    Gene Therapy Program, Department of Pathology and Laboratory Medicine, Division of Transfusion Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA
    Hum Gene Ther 21:1259-71. 2010
    ..In addition, the iodixanol gradient purification process described effectively separates infectious particles from empty capsids, a desirable property for reducing toxicity and unwanted immune responses during preclinical studies...
  30. pmc Comparative analysis of transposable element vector systems in human cells
    Ivana Grabundzija
    Max Delbruck Center for Molecular Medicine, Berlin, Germany
    Mol Ther 18:1200-9. 2010
    ..SB, Tol2, and PB constitute complementary research tools for gene transfer in mammalian cells with important implications for fundamental and translational research...
  31. ncbi Control of memory formation through regulated expression of a CaMKII transgene
    M Mayford
    Center for Neurobiology and Behavior, College of Physicians and Surgeons of Columbia University, and Howard Hughes Medical Institute, 722 West 168 Street, New York, NY 10032, USA
    Science 274:1678-83. 1996
    ..Thus, the CaMKII signaling pathway is critical for both explicit and implicit memory storage, in a manner that is independent of its potential role in development...
  32. ncbi Paramutation in Drosophila linked to emergence of a piRNA-producing locus
    Augustin de Vanssay
    Laboratoire Biologie du Développement, UMR7622, CNRS Universite Pierre et Marie Curie, 9 quai Saint Bernard, 75005 Paris, France
    Nature 490:112-5. 2012
    ..We now show that clusters of P-element-derived transgenes that induce strong TSE can convert other homologous transgene clusters incapable of TSE into strong silencers, ..
  33. ncbi High-throughput assessment of transgene copy number in sugarcane using real-time quantitative PCR
    Rosanne E Casu
    CSIRO Plant Industry, Queensland Bioscience Precinct, St Lucia, QLD, Australia
    Plant Cell Rep 31:167-77. 2012
    ..This method requires much less input DNA, can be performed much earlier in the production of transgenic sugarcane plants and allows much more efficient assessment of numerous potentially transgenic lines than Southern blotting...
  34. pmc Distinct requirements for somatic and germline expression of a generally expressed Caernorhabditis elegans gene
    W G Kelly
    Carnegie Institution of Washington, Department of Embryology, Baltimore, Maryland 21210, USA
    Genetics 146:227-38. 1997
    ..The resulting transgenes show robust expression in both germline and soma...
  35. pmc Suberoylanilide hydroxamic acid, a histone deacetylase inhibitor, ameliorates motor deficits in a mouse model of Huntington's disease
    Emma Hockly
    Medical and Molecular Genetics, Guy s, King s and St Thomas School of Medicine, King s College London, Eighth Floor Guy s Tower, Guy s Hospital, London SE1 9RT, United Kingdom
    Proc Natl Acad Sci U S A 100:2041-6. 2003
    ..SAHA dramatically improved the motor impairment in R6/2 mice, clearly validating the pursuit of this class of compounds as HD therapeutics...
  36. pmc Two plant viral suppressors of silencing require the ethylene-inducible host transcription factor RAV2 to block RNA silencing
    Matthew W Endres
    Department of Biological Sciences, University of South Carolina, Columbia, South Carolina, USA
    PLoS Pathog 6:e1000729. 2010
  37. ncbi Viral 2A peptides allow expression of multiple proteins from a single ORF in transgenic zebrafish embryos
    Elayne Provost
    Department of Surgery, Johns Hopkins University School of Medicine, Baltimore, Maryland 21205, USA
    Genesis 45:625-9. 2007
    ..The successful application of this technology in zebrafish will be valuable for visually marking transgenic embryos and transgene-expressing cells, or in any situation where reliable expression of multiple transgenes is desired.
  38. ncbi Gene targeting by homologous recombination in Drosophila
    Y S Rong
    Department of Biology, University of Utah, Salt Lake City, UT 84112, USA
    Science 288:2013-8. 2000
    ..A method is described that enables analogous manipulations of the Drosophila genome. This technique may also be applicable to other organisms for which gene-targeting procedures do not yet exist...
  39. ncbi Defective neuromuscular synaptogenesis in agrin-deficient mutant mice
    M Gautam
    Department of Anatomy and Neurobiology, Washington University Medical School St Louis, Missouri 63110, USA
    Cell 85:525-35. 1996
  40. ncbi A mouse model for spinal muscular atrophy
    H M Hsieh-Li
    Institute of Molecular Biology, Academia Sinica, Taipei, Taiwan
    Nat Genet 24:66-70. 2000
    ..Our results demonstrate that SMN2 can partially compensate for lack of SMN1. The variable phenotypes of Smn-/-SMN2 mice reflect those seen in SMA patients, providing a mouse model for this disease...
  41. ncbi Conditional control of gene expression in the mouse
    M Lewandoski
    Section of Genetics of Vertebrate Development, Laboratory of Cancer and Developmental Biology, National Cancer Institute Frederick Cancer Research and Development Center, Frederick, Maryland 21702 1201, USA
    Nat Rev Genet 2:743-55. 2001
    ..Here, I review these systems and discuss certain studies that exemplify the advantages and limitations of each system...
  42. ncbi Intraneuronal Abeta, non-amyloid aggregates and neurodegeneration in a Drosophila model of Alzheimer's disease
    D C Crowther
    Department of Medicine, University of Cambridge, Cambridge Institute for Medical Research, Wellcome Trust MRC Building, Hills Road, Cambridge CB2 2XY, UK
    Neuroscience 132:123-35. 2005
    ..Moreover it provides a platform to dissect the pathways of neurodegeneration in Alzheimer's disease and to develop novel therapeutic interventions...
  43. pmc Transgene expression is associated with copy number and cytomegalovirus promoter methylation in transgenic pigs
    Qingran Kong
    College of Life Science, Northeast Agricultural University of China, Harbin, People s Republic of China
    PLoS ONE 4:e6679. 2009
    ..Our results demonstrate that transgene expression is associated with copy number and CMV promoter methylation in transgenic pigs...
  44. ncbi I-SceI meganuclease mediates highly efficient transgenesis in fish
    Violette Thermes
    INRA Junior Group, UPR2197, Institut de Neurobiologie A Fessard, CNRS, Avenue de la Terrasse, 91 198 Gif sur Yvette, France
    Mech Dev 118:91-8. 2002
    ..use of fish as model systems is still limited by the mosaic distribution of cells transiently expressing transgenes leading to a low frequency of transgenic fish. Here we present a strategy that overcomes this problem...
  45. ncbi Direct and long-range action of a wingless morphogen gradient
    M Zecca
    Zoologisches Institut der Universitat Zurich, Switzerland
    Cell 87:833-44. 1996
    ..We interpret these findings as evidence that Wg can act directly and at long range as a gradient morphogen during normal development...
  46. ncbi Temporally regulated and tissue-specific gene manipulations in the adult and embryonic heart using a tamoxifen-inducible Cre protein
    D S Sohal
    Department of Pediatrics, University of Cincinnati, Children s Hospital Medical Center, Division of Molecular Cardiovascular Biology, Cincinnati, Ohio, USA
    Circ Res 89:20-5. 2001
  47. pmc Site-specific integration and expression of an anti-malarial gene in transgenic Anopheles gambiae significantly reduces Plasmodium infections
    Janet M Meredith
    Centre for Applied Entomology and Parasitology, Keele University, Keele, United Kingdom
    PLoS ONE 6:e14587. 2011
    ..In the future, this technology will allow effective comparisons and informed choices to be made, potentially leading to complete transmission blockade...
  48. ncbi Myc-induced T cell leukemia in transgenic zebrafish
    David M Langenau
    Department of Pediatric Oncology, Dana Farber Cancer Institute, Harvard Medical School, Boston, MA 02115, USA
    Science 299:887-90. 2003
    ..This transgenic model provides a platform for drug screens and for genetic screens aimed at identifying mutations that suppress or enhance c-myc- induced carcinogenesis...
  49. pmc Functional genomics in Trypanosoma brucei: a collection of vectors for the expression of tagged proteins from endogenous and ectopic gene loci
    Steven Kelly
    Sir William Dunn School of Pathology, University of Oxford, South Parks Road, Oxford OX1 3RE, UK
    Mol Biochem Parasitol 154:103-9. 2007
  50. ncbi Transgenic strategies for combinatorial expression of fluorescent proteins in the nervous system
    Jean Livet
    Department of Molecular and Cellular Biology and Center for Brain Science, Harvard University, Cambridge, Massachusetts 02138, USA
    Nature 450:56-62. 2007
    ..In Brainbow transgenes, Cre/lox recombination is used to create a stochastic choice of expression between three or more fluorescent ..
  51. ncbi The complete family of genes encoding G proteins of Caenorhabditis elegans
    G Jansen
    Division of Molecular Biology, The Netherlands Cancer Institute, Centre for Biomedical Genetics, Amsterdam
    Nat Genet 21:414-9. 1999
  52. ncbi Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
    Brian D Brown
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Via Olgettina, 58, 20132 Milan, Italy
    Nat Biotechnol 25:1457-67. 2007
    ..Notably, the vectors did not detectably perturb endogenous miRNA expression or regulation of natural targets. The properties of miRNA-regulated vectors should allow for safer and more effective therapeutic applications...
  53. pmc pEPito: a significantly improved non-viral episomal expression vector for mammalian cells
    Rudolf Haase
    Max von Pettenkofer Institute, University of Munich, Munich, Germany
    BMC Biotechnol 10:20. 2010
  54. ncbi Myotonic dystrophy in transgenic mice expressing an expanded CUG repeat
    A Mankodi
    Department of Neurology, School of Medicine and Dentistry, University of Rochester, Box 673, 601 Elmwood Avenue, Rochester, NY 14642, USA
    Science 289:1769-73. 2000
    ..Thus, transcripts with expanded CUG repeats are sufficient to generate a DM phenotype. This result supports a role for RNA gain of function in disease pathogenesis...
  55. pmc High-level transgene expression by homologous recombination-mediated gene transfer
    Mélanie Grandjean
    Laboratory of Molecular Biotechnology, Center for Biotechnology UNIL EPFL, University of Lausanne, Lausanne, Switzerland
    Nucleic Acids Res 39:e104. 2011
    ..We conclude that MAR elements may promote homologous recombination, and that cells and vectors can be engineered to take advantage of this property to mediate highly efficient gene transfer and expression...
  56. pmc High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
    Hilda Petrs-Silva
    Department of Ophthalmology, University of Florida, Gainesville, Florida 32610 0284, USA
    Mol Ther 17:463-71. 2009
    ..These enhanced AAV vectors have a great potential for future therapeutic applications for retinal degenerations and ocular neovascular diseases...
  57. pmc Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine
    J H McIntosh
    Department of Haematology, UCL Cancer Institute, University College London, Oxford, UK
    Gene Ther 19:78-85. 2012
    ..These observations could significantly improve the prospects of using rAAV vectors for chronic disorders by allowing for repeated vector administration and avoiding the development of antibodies to the transgene product...
  58. pmc Controlling transgene expression in subcutaneous implants using a skin lotion containing the apple metabolite phloretin
    Marc Gitzinger
    Department of Biosystems Science and Engineering, Eidgenossische Technische Hochschule Zurich, Mattenstrasse 26, CH 4058 Basel, Switzerland
    Proc Natl Acad Sci U S A 106:10638-43. 2009
    Adjustable control of therapeutic transgenes in engineered cell implants after transdermal and topical delivery of nontoxic trigger molecules would increase convenience, patient compliance, and elimination of hepatic first-pass effect in ..
  59. ncbi Generation of a prostate epithelial cell-specific Cre transgenic mouse model for tissue-specific gene ablation
    X Wu
    Department of Pathology, Keck School of Medicine, University of Southern California, 2011 Zonal Avenue, Los Angeles, CA 90089, USA
    Mech Dev 101:61-9. 2001
    ..The PB-Cre4 mice will be a useful resource for genetic-based studies on prostate development and prostatic disease...
  60. pmc Exploiting position effects and the gypsy retrovirus insulator to engineer precisely expressed transgenes
    Michele Markstein
    Department of Genetics and Howard Hughes Medical Institute, Harvard Medical School, Boston, Massachusetts 02115, USA
    Nat Genet 40:476-83. 2008
    A major obstacle to creating precisely expressed transgenes lies in the epigenetic effects of the host chromatin that surrounds them...
  61. ncbi The arterial pole of the mouse heart forms from Fgf10-expressing cells in pharyngeal mesoderm
    R G Kelly
    Departement de Biologie Moleculaire, Institut Pasteur, Paris, France
    Dev Cell 1:435-40. 2001
    ..25 and 10.5. These data suggest that arterial pole myocardium originates outside the classical heart field...
  62. ncbi The production of recombinant pharmaceutical proteins in plants
    Julian K C Ma
    Department of Infectious Diseases, St George s Hospital Medical School, Cranmer Terrace, London SW17 0RE, UK
    Nat Rev Genet 4:794-805. 2003
    ..The first plant-derived recombinant pharmaceutical proteins are now approaching commercial approval, and many more are expected to follow...
  63. ncbi Estimating the copy number of transgenes in transformed rice by real-time quantitative PCR
    Litao Yang
    School of Life Science and Biotechnology, Shanghai Jiao Tong University, 800 Dongchuan Road, Shanghai, 200240, People s Republic of China
    Plant Cell Rep 23:759-63. 2005
    ..Furthermore, our copy number estimations of both the GUS reporter gene and the HPT selective marker gene showed that rearrangements of the T-DNA occurred more frequently than is generally believed in transgenic rice...
  64. 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
  65. pmc BAC TransgeneOmics: a high-throughput method for exploration of protein function in mammals
    Ina Poser
    Max Planck Institute for Molecular Cell Biology and Genetics, Pfotenhauerstrasse 108, D 01307 Dresden, Germany
    Nat Methods 5:409-15. 2008
    ..The large size of the BAC transgenes ensures the presence of most, if not all, regulatory elements and results in expression that closely matches ..
  66. 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
    The residual presence of integrated transgenes following the derivation of induced pluripotent stem (iPS) cells is highly undesirable...
  67. pmc Gene therapy for red-green colour blindness in adult primates
    Katherine Mancuso
    Department of Ophthalmology, Box 356485, University of Washington, 1959 North East Pacific Street, Seattle, Washington 98195, USA
    Nature 461:784-7. 2009
    ..Thus, trichromacy can arise from a single addition of a third cone class and it does not require an early developmental process. This provides a positive outlook for the potential of gene therapy to cure adult vision disorders...
  68. pmc Site-specific recombination for the modification of transgenic strains of the Mediterranean fruit fly Ceratitis capitata
    Marc F Schetelig
    Department of Developmental Biology, Georg August University Gottingen, GZMB, Ernst Caspari Haus, Johann Friedrich Blumenbach Institute of Zoology and Anthropology, 37077 Gottingen, Germany
    Proc Natl Acad Sci U S A 106:18171-6. 2009
    ..Here we describe an efficient method for the modification of previously inserted transgenes by the use of the site-specific integration system from phage phiC31 in a tephritid pest species, the ..
  69. ncbi A gene expression atlas of the central nervous system based on bacterial artificial chromosomes
    Shiaoching Gong
    GENSAT Project, Howard Hughes Medical Institute, The Rockefeller University, 1230 York Avenue, Box 260, New York 10021, USA
    Nature 425:917-25. 2003
    ..The atlas, library of BAC vectors and BAC transgenic mice generated in this screen provide a rich resource that allows a broad array of investigations not previously available to the neuroscience community...
  70. pmc Complete rescue of obesity, diabetes, and infertility in db/db mice by neuron-specific LEPR-B transgenes
    Carl De Luca
    Department of Medicine, Division of Preventative Medicine, Columbia University, New York, New York, USA
    J Clin Invest 115:3484-93. 2005
    ..We have also generated mice that are compound hemizygotes for the transgenes SYN-LEPR-B and neuron-specific enolase-LEPR B (NSE-LEPR-B)...
  71. pmc Creation of low-copy integrated transgenic lines in Caenorhabditis elegans
    V Praitis
    Department of Molecular Genetics and Cell Biology, University of Chicago, Chicago, Illinois 60637, USA
    Genetics 157:1217-26. 2001
    ..This technique is a reliable means of expressing transgenes in C. elegans, but its use has limitations...
  72. pmc Constitutive expression of transgenes encoding derivatives of the synthetic antimicrobial peptide BP100: impact on rice host plant fitness
    Anna Nadal
    Institute of Food and Agricultural Technology, University of Girona, Campus Montilivi, Spain
    BMC Plant Biol 12:159. 2012
    ..Specific challenges were the small length, peptide degradation by plant proteases and toxicity to the host plant. Here we approached the expression of the BP100 peptide series in plants using BP100 as a proof-of-concept...
  73. pmc A method for producing transgenic cells using a multi-integrase system on a human artificial chromosome vector
    Shigeyuki Yamaguchi
    Department of Biomedical Science, Institute of Regenerative Medicine and Biofunction, Graduate School of Medical Sciences, Tottori University, Yonago, Japan
    PLoS ONE 6:e17267. 2011
    ..The ability to insert transgenes at a precise location in the genome, using site-specific recombinases such as Cre, FLP, and ΦC31, has major ..
  74. pmc Human artificial chromosomes for gene delivery and the development of animal models
    Yasuhiro Kazuki
    Department of Biomedical Science, Institute of Regenerative Medicine and Biofunction, Graduate School of Medical Science, Tottori University, Yonago, Japan
    Mol Ther 19:1591-601. 2011
    ..This review describes the lessons learned and prospects identified mainly from studies in the construction of HACs and HAC-mediated gene expression systems in cultured cells, as well as in animals...
  75. ncbi Genome-wide prediction of matrix attachment regions that increase gene expression in mammalian cells
    Pierre Alain Girod
    Institute of Biotechnology, University of Lausanne, and Center for Biotechnology of the University of Lausanne and École Polytechnique Fédérale de Lausanne, CH 1015 Lausanne, Switzerland
    Nat Methods 4:747-53. 2007
    ..Thus we computationally identified potent epigenetic regulators, opening new strategies toward high and stable transgene expression for research, therapeutic production or gene-based therapies...
  76. ncbi A light-switchable gene promoter system
    Sae Shimizu-Sato
    Department of Plant and Microbial Biology, University of California, Berkeley, CA 94720, USA
    Nat Biotechnol 20:1041-4. 2002
    ..Thus, this system has the potential to provide rapid, noninvasive, switchable control of the expression of a desired gene to a preselected level in any suitable cell by simple exposure to a light signal...
  77. pmc Fitness of anopheline mosquitoes expressing transgenes that inhibit Plasmodium development
    Luciano A Moreira
    Department of Genetics, School of Medicine, Case Western Reserve University, Cleveland, Ohio 44106 4955, USA
    Genetics 166:1337-41. 2004
    ..Mosquitoes carrying either of these transgenes were impaired for Plasmodium berghei transmission...
  78. ncbi Spatiotemporal rescue of memory dysfunction in Drosophila
    Sean E McGuire
    Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, TX 77030, USA
    Science 302:1765-8. 2003
    ..The TARGET system offers general utility in simultaneously addressing issues of when and where gene products are required...
  79. pmc Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle
    Chunping Qiao
    Division of Molecular Pharmaceutics, University of North Carolina Eshelman School of Pharmacy, Chapel Hill, NC 27599, USA
    Hum Gene Ther 21:1343-8. 2010
    ..Our results suggest that tyrosine-mutant AAV6 vectors may represent powerful tools for testing muscle gene therapy in animal models and potentially in humans...
  80. ncbi Building a zoo of mice for genetic analyses: a comprehensive protocol for the rapid generation of BAC transgenic mice
    Torbjörn Johansson
    Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland
    Genesis 48:264-80. 2010
    ..With the provided "toolbox" of already available transgene constructs, the generation of the BAC transgenes is made easy and rapid...
  81. pmc Expression of the native cholera toxin B subunit gene and assembly as functional oligomers in transgenic tobacco chloroplasts
    H Daniell
    Department of Molecular Biology and Microbiology and Center for Discovery of Drugs and Diagnostics, University of Central Florida, 12722 Research Parkway, Orlando, FL 32826 3227, USA
    J Mol Biol 311:1001-9. 2001
  82. pmc Virus-free induction of pluripotency and subsequent excision of reprogramming factors
    Keisuke Kaji
    MRC Centre for Regenerative Medicine, Institute for Stem Cell Research, University of Edinburgh, Edinburgh EH9 3JQ, UK
    Nature 458:771-5. 2009
  83. ncbi Recombineering BAC transgenes for protein tagging
    Giovanni Ciotta
    Genomics, BioInnovationsZentrum, Technische Universitaet Dresden, Am Tatzberg 47, 01307 Dresden, Germany
    Methods 53:113-9. 2011
    ..BAC (bacterial artificial chromosome) transgenes present a way to express a chosen protein at physiological levels with all regulatory elements in their native ..
  84. ncbi Pig transgenesis by Sleeping Beauty DNA transposition
    Jannik E Jakobsen
    Department of Human Genetics, University of Aarhus, Wilh Meyers Allé 1240, 8000, Aarhus C, Denmark
    Transgenic Res 20:533-45. 2011
  85. pmc Efficient integration of transgenes into a defined locus in human embryonic stem cells
    Kenji Sakurai
    Institute for Frontier Medical Sciences, Kyoto University, Sakyo ku, Kyoto 606 8507, Japan
    Nucleic Acids Res 38:e96. 2010
    ..We show that reporter gene expression was responsive to doxycycline and that the resulting transgenic human ES cells retain their self-renewal capacity and pluripotency...
  86. ncbi Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo
    Zhi Ying Chen
    Department of Pediatrics and Department of Genetics, Stanford University School of Medicine, Stanford, California 94305, USA
    Mol Ther 8:495-500. 2003
    ..Our data suggest that minicircles are capable of expressing high and persistent levels of therapeutic products in vivo and have a great potential to serve as episomal vectors for the treatment of a wide variety of diseases...
  87. ncbi Lentiviral vectors with two independent internal promoters transfer high-level expression of multiple transgenes to human hematopoietic stem-progenitor cells
    Xiaobing Yu
    Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Department of Oncology, Baltimore, Maryland 21231, USA
    Mol Ther 7:827-38. 2003
    ..We obtained inconsistent results in our attempts to coexpress two transgenes linked by an internal ribosomal entry site (IRES) element in a single bicistronic LV transcript...
  88. 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...
  89. pmc The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
    Jiangao Zhu
    Department of Medicine, Duke University Medical Center, Durham, North Carolina 27710, USA
    J Clin Invest 119:2388-98. 2009
  90. pmc The RING finger/B-box factor TAM-1 and a retinoblastoma-like protein LIN-35 modulate context-dependent gene silencing in Caenorhabditis elegans
    J Hsieh
    Carnegie Institution of Washington, Department of Embryology, Baltimore, Maryland 21210, USA
    Genes Dev 13:2958-70. 1999
    ..We have used tandem array transgenes as a model substrate in a screen for Caenorhabditis elegans mutants that affect context-dependent gene silencing ..
  91. ncbi Transcriptional regulation of nanog by OCT4 and SOX2
    David J Rodda
    Stem Cell and Developmental Biology and Gene Regulation Laboratory, Genome Institute of Singapore, Singapore 138672
    J Biol Chem 280:24731-7. 2005
    ..These studies extend the understanding of the pluripotent genetic regulatory network within which the Sox2-Oct4 complex are at the top of the regulatory hierarchy...
  92. ncbi Transient and stable transgene expression in human embryonic stem cells
    Chee Gee Liew
    Centre for Stem Cell Biology, Department of Biomedical Science, Western Bank, University of Sheffield, Sheffield S10 2TN, United Kingdom
    Stem Cells 25:1521-8. 2007
    ..Our data also demonstrate that pUbiC, pR26, pCAGG, and pCAG are more superior to the pCMV for generation of stable transfectants in hES cells. Disclosure of potential conflicts of interest is found at the end of this article...
  93. pmc The vasa regulatory region mediates germline expression and maternal transmission of proteins in the malaria mosquito Anopheles gambiae: a versatile tool for genetic control strategies
    Philippos A Papathanos
    Imperial College London, Division of Cell and Molecular Biology, Imperial College Road, London, UK
    BMC Mol Biol 10:65. 2009
    ..Germline specific promoters are an essential component of potential vector control strategies which function by genetic drive, however suitable promoters are not currently available for the main human malaria vector Anopheles gambiae...
  94. pmc Notch and Kras reprogram pancreatic acinar cells to ductal intraepithelial neoplasia
    Jean Paul De La O
    Department of Human Genetics, University of Utah, 15 North 2030 East, Room 2100, Salt Lake City, UT 84112, USA
    Proc Natl Acad Sci U S A 105:18907-12. 2008
    ..At the cellular level, Notch/Kras coactivation promotes rapid reprogramming of acinar cells to a duct-like phenotype, providing an explanation for how a characteristically ductal tumor can arise from nonductal acinar cells...
  95. pmc Translating Sleeping Beauty transposition into cellular therapies: victories and challenges
    Zsuzsanna Izsvák
    Max Delbrück Center for Molecular Medicine MDC, Berlin, Germany
    Bioessays 32:756-67. 2010
    Recent results confirm that long-term expression of therapeutic transgenes can be achieved by using a transposon-based system in primary stem cells and in vivo...
  96. pmc Activation of the NF-kappaB pathway by adeno-associated virus (AAV) vectors and its implications in immune response and gene therapy
    Giridhara R Jayandharan
    Division of Cellular and Molecular Therapy, Department of Pediatrics, Department of Pharmacology and Therapeutics, University of Florida College of Medicine, Gainesville, FL 32611, USA
    Proc Natl Acad Sci U S A 108:3743-8. 2011
  97. ncbi Dual roles for glucokinase in glucose homeostasis as determined by liver and pancreatic beta cell-specific gene knock-outs using Cre recombinase
    C Postic
    Department of Molecular Physiology and Biophysics, Vanderbilt University School of Medicine, Nashville, Tennessee 37232, USA
    J Biol Chem 274:305-15. 1999
    ..beta cell- and hepatocyte-specific expression of Cre was achieved using transgenes that contain either insulin or albumin promoter/enhancer sequences...
  98. ncbi High-level expression of a suite of thermostable cell wall-degrading enzymes from the chloroplast genome
    Kerstin Petersen
    Max Planck Institut fur Molekulare Pflanzenphysiologie, Am Muhlenberg 1, 14476 Potsdam Golm, Germany
    Plant Mol Biol 76:311-21. 2011
    ..Although further optimization is needed, our data indicate that transgenic plastids offer great potential for the production of enzyme cocktails for the bioconversion of cellulosic biomass...
  99. ncbi Stable integration and conditional expression of electroporated transgenes in chicken embryos
    Yuki Sato
    Graduate School of Biological Sciences, Nara Institute of Science and Technology, 8916 5, Takayama, Ikoma, Nara, 630 0192, Japan
    Dev Biol 305:616-24. 2007
    ..However, the conventional electroporation technique fails to retain the expression of transgenes for more than several days because transgenes are not integrated into the genome...
  100. 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
    Here we describe the sustained expression of transgenes introduced into human embryonic stem (ES) cells using self-inactivating lentiviral vectors...
  101. ncbi Only half the transcriptomic differences between resistant genetically modified and conventional rice are associated with the transgene
    Maria Montero
    Institut de Tecnologia Agroalimentària INTEA, Universitat de Girona, Campus Montilivi, Girona, Spain
    Plant Biotechnol J 9:693-702. 2011
    ..Unintended effects might, as such, putatively result in widening the self-resistance characteristics because of the transgene in GM plants...

Research Grants74

  1. Glucocorticoids, Stress and Blood Pressure Regulation
    Deborah A Scheuer; Fiscal Year: 2013
    ..The synthetic dopamine-ss-hydroxylase promoter PRSx8 will be used to drive expression of the transgenes selectively in catecholaminergic neurons...
  2. Genomic actions of progesterone receptors in human myometrial cells
    Sam Mesiano; Fiscal Year: 2010
    ..modified immortalized myometrial cell model, hTERT-HMA/B cells, containing independent inducible PR-A and PR-B transgenes. This is a novel and powerful approach to determine the specific genomic actions of progesterone via PR-A and PR-..
  3. Genetic correction of human beta-thalassemic induced pluripotent stem cells
    EIRINI PAPAPETROU; Fiscal Year: 2012
    ..I worked on the engineering of lineage- and developmental stage- specific expression of lentivirally-encoded transgenes in the hematopoietic system by exploiting microRNA-mediated gene regulation and on the generation and genetic ..
    Eric F Johnson; Fiscal Year: 2013
    ..The CYP4A11 and CYP4F2 transgenes will be expressed in Cyp4a10 null mice to gauge the effect of expression of each transgene on the susceptibility ..
  5. Rescuing Niemann-Pick C Disease: Pathways of Liver and Brain Degeneration
    Matthew P Scott; Fiscal Year: 2013
    ..Our transgenes are engineered with Tet-technology to allow cell-specific and temporal regulation of tagged Npc1 production...
  6. Genetic correction of human beta-thalassemic induced pluripotent stem cells
    EIRINI PAPAPETROU; Fiscal Year: 2013
    ..I worked on the engineering of lineage- and developmental stage-specific expression of lentivirally-encoded transgenes in the hematopoietic system by exploiting microRNA-mediated gene regulation and on the generation and genetic ..
  7. Analysis and Therapy of Age-Dependent Proteostasis Failure in Neurodegeneration
    ..elegans models of Alzheimer's, initiated or seeded by neuron-specific tau and Ass transgenes. Protein complexes from nematodes with age-dependent or induced aggregates will be pulled down with antibody to ..
  8. Regulators of Cancer-Specific DNA Hypermethylation.
    Jean Pierre J Issa; Fiscal Year: 2013
    ..For this testing we will use a series of cellular models developed in our laboratory where engineered transgenes can be inserted in specific genomic loci, and later on moved in and out of repressive contexts due to the ..
    Eric J Nestler; Fiscal Year: 2013
    ..for the construction of several new lines of mutant mice, including those with inducible tissue-specific transgenes and knockouts...
  10. Toward suicidal automation of porphyric Leishmania for photodynamic vaccination
    Kwang Poo Chang; Fiscal Year: 2010
    ..The relevant transgenes will be placed under the regulation of known 3'-UTR sequences for stage-specific expression as the mutants ..
  11. AAV vectors for retinal delivery in a mouse model of age-related retinopathy
    KATHARINE LIANG; Fiscal Year: 2013
    ..AAV is a nonpathogenic, non-integrating virus that can be used to deliver therapeutic transgenes to healthy or diseased tissues to prevent or treat disease, respectively...
  12. Induced Pluripotent Stem Cells from Swine: application to genetic modification
    ROBERT MICHAEL ROBERTS; Fiscal Year: 2013
    ..additional lines of LIF-dependent ICM-derived porcine (p) iPSC (pICM-iPSC) by upregulating human POU5F1 and KLF4 transgenes assembled on "tet-on" bicistronic lentiviral vectors, which offer the advantage of effective transgene silencing ..
  13. Viral Gene Expression to Therapeutic Levels in Models of Heart Failure
    Joseph E Rabinowitz; Fiscal Year: 2012
    ..AAV9 as well as the best transcapsidation mixtures we will assess the therapeutic function of 2ARKct and S100A1 transgenes, after systemic injection, in a dose dependent manner...
  14. Sox10 Alleles for Functional Analysis of Glial Lineages
    E Michelle Southard-Smith; Fiscal Year: 2013
    ..Regulatory regions from the Sox10 gene have previously been used to drive expression of single fluorophore transgenes for tracking the migration of glial progenitors and the distribution of mature glial cell types...
  15. DNA Pro-Drug Technology in Localized Wound Healing and Tissue Regeneration
    Arthur A Decarlo; Fiscal Year: 2011
    ..outcomes include histomorphometric assessments of inflammation, molecular evidence for in situ expression of the transgenes, and a pathology analysis...
  16. Transgenic Study of ALS-Linked CCS Mutations
    Han Xiang Deng; Fiscal Year: 2011
    ..challenges and opportunities, we propose to develop and characterize transgenic mouse models using human CCS transgenes: wild-type CCS (wtCCS), CCSR71W and CCSG222R...
  17. Phase 1 Study of Umbilical Cord Blood-Derived T Cells in Malignant B Cells
    Elizabeth J Shpall; Fiscal Year: 2013
    ..impact their therapeutic potential Aim #3, undertake secondary objectives to determine immune response(s) to the transgenes;trafficking of CAR+ T cells;development of oligoclonal sub-population(s) of infused T cells;emergence of ..
  18. Oxidative damage and cone cell death in RP
    Peter A Campochiaro; Fiscal Year: 2013
    ..after subretinal or intravitreous injections in mice or pigs and we initiated studies with therapeutic transgenes in mouse and pig models of RP...
  19. Regulated Expression of Hypothalamic CPT1 Using Gutless Adenovirus
    Silvana Obici; Fiscal Year: 2010
    ..since to date no suitable gene expression system has been developed that allows controlled expression of transgenes at moderate levels...
  20. Molecular Genetic Tools for Parasitic Helminths
    Edward J Pearce; Fiscal Year: 2013
    ..New methods for chromosomal integration of transgenes will be developed and insulator sequences will be identified as measures against epigenetic transgene silencing ..
  21. Transgenic Mouse Models of FUS/TLS-Mediated Amyotrophic Lateral Sclerosis
    Zuoshang Xu; Fiscal Year: 2010
    ..In the second approach, we will generate mice in which expression of FUS/TLS transgenes is activated only after crossing with mice that express Cre recombinase in specific tissues...
  22. Systematic functional analysis of non-coding sequences in transgenic zebrafish
    Shannon Fisher; Fiscal Year: 2010
    ..use of the phage &C31 site specific recombinase in zebrafish, to facilitate re-engineering of existing transgenes in situ...
  23. Defining the Role of MafA in Islet Beta Cells
    Roland W Stein; Fiscal Year: 2013
    ..This hypothesis will be tested by analyzing the functional characteristics of transgenes expressing either a MafB/A chimera or MafB on islet [unreadable] cell function in MafA(panc mice, which lack ..
  24. Hepatic expression of small hairpin RNA using gutless adenoviral vectors
    Nuria Morral; Fiscal Year: 2012
    ..vectors are optimal tools for gene delivery to the liver because they result in long-term expression of transgenes and negligible toxicity when administered at high doses...
  25. Behavioral and Molecular Effects of Antenatal SSRI Exposure
    Lawrence S Quang; Fiscal Year: 2013
    ..possible by the identification of the Pet-1 cis regulatory region that directs highly reproducible expression of transgenes in developing and adult brain 5-HT neurons, Dr...
  26. Using Microfluidic Affinity Analysis to Probe Transcriptional Regulation
    POLLY MORRELL FORDYCE; Fiscal Year: 2013 regulatory sequence can lead to transcriptional dysfunction and disease and improving rational design of transgenes for gene therapy...
  27. Role of microRNA in HIV associated neurological disorder (HAND).
    Bassel E Sawaya; Fiscal Year: 2013
    ..defenders of genome integrity in response to foreign or invasive nucleic acids such as viruses, transposons, and transgenes. Both categories of RNAs act in somatic lineages in a broad range of eukaryotic species...
  28. Modeling the KIF5B-RET Fusion Gene of Lung Cancer in Vivo
    Jie Wu; Fiscal Year: 2013
    ..from different lines of existing Cre-RMCE-capable transgenic mice to replace the original floxed tetO- SHP2 transgenes with tetO-KIF5B-RET...
  29. Role of miR-8 and Ena in Synaptic Structure and Function
    CARLOS MANUEL LOYA; Fiscal Year: 2010
    ..and presynaptic ultrastructural apparatus at the electron microscopy level;and 3) test a series of Ena mutant transgenes to determine which conserved sequence elements are critical for the localization and function of Ena at the NMJ...
  30. MR Spectroscopy to Evaluate Liver Repopulation by Transplanted Hepatocytes
    Chandan Guha; Fiscal Year: 2009
    ..of individual liver lobes by the transplanted hepatocytes and enable monitoring the expression of target transgenes in the diseased host liver...
  31. Molecular control of neurogenesis in the adult subventricular zone
    Masato Nakafuku; Fiscal Year: 2013
    ..We will also perform conditional GOF experiments using mice carrying tet-O promoter-driven Gsx1 and Gsx2 transgenes. Virus-mediated overexpression and knockdown of Gsx1 and Gsx2 will be performed in parallel...
  32. DNA Demethylation and Transcriptional Gene Silencing
    Jian Kang Zhu; Fiscal Year: 2013
    ..genome stability, developmental specific gene expression, genomic imprinting, and transcriptional silencing of transgenes and other foreign DNA. Cancers and many other diseases are associated with epigenetic alterations...
  33. Develop an Oral HIV Vaccine Using Papilloma Virus-Like Particles as a Vector
    Liang Qiao; Fiscal Year: 2012
    ..We will also determine if these vaccines can protect mice with human CD4 and CCR5 transgenes against HIV-1 challenge...
  34. Characteristics and mechanisms of pain in sickle cell disease
    DONALD SIMONE; Fiscal Year: 2013
    ..Sickle hBERK mice have a mixed genetic background and carry a single copy of linked transgenes for human 1 and 2S globins...
  35. VEGF receptor-1-mediated protection in dilated cardiomyopathy
    Fabio A Recchia; Fiscal Year: 2013
    ..Aim 3 is to test the cardioprotective effects of VEGF-B transgenes inducible by endogenous stimuli specifically occurring in the failing heart...
  36. Development of Novel AIDS Gene Therapy Strategies in the Macaque SHIV Model
    Hans Peter Kiem; Fiscal Year: 2012
    ..The anti-HIV vectors we propose to test contain MGMT transgenes to allow in vivo selection post-transplantation...
  37. Transgenic Tethered Peptides
    Michael N Nitabach; Fiscal Year: 2013
    ..based on the UAS-GAL4 binary expression system that separates cell- and circuit- specific promoter GAL4 driver transgenes from UAS transgenes containing cDNAs that encode effectors such as t-peptides, it will be of direct utility to ..
  38. Role of macrophages in MSC-mediated recovery of hematopoiesis after irradiation
    Manoj M Pillai; Fiscal Year: 2010
    ..This transgenic mouse can be used to express transgenes that can either augment or inhibit macrophages in a reversible manner, thereby providing a means to directly ..
    Paul Kaufman; Fiscal Year: 2009
    ..used in combination with common glaucoma therapies or resulting from over-expression of viral vector delivered transgenes encoding for cytoskeleton modulating proteins (caldesmon and C3), (3) create a new primate model of glaucoma by ..
  40. Immune Cell Carriers for the Delivery of Oncolytic Viruses to Metastatic Tumors
    Richard G Vile; Fiscal Year: 2013
    ..virus loaded lymphocytes by enhancing the potency of the virus payload using oncolytic VSV expressing additional transgenes to increase T cell recruitment, direct cytotoxicity and/or immune activation...
    CLAUDIA T KAPPEN; Fiscal Year: 2010
    ..This idea is supported by transgenic paradigms. In mice, transgenes for the pancreatic transcription factor Isl-1 induce phenotypes that resemble human diabetic embryopathy, ..
    James A Birchler; Fiscal Year: 2012
    ..this novel process by targeting proteins thought to be involved, either singly or in combinations, to reporter transgenes on the X and autosomes in males and females of various genotypes...
  43. Investigating the role of ribeye in retinal ribbon function
    DAVID PAUL ZENISEK; Fiscal Year: 2013
    ..the role of ribeye in synaptic transmission, we will investigate the effects of over expression of ribeye transgenes on synaptic release from rod bipolar cells on to AII amacrine cells in retinal slice recordings...
  44. How do you build a parasite?
    John Murray; Fiscal Year: 2009
    ..efficiency of transient transfection permits rapid assessment of recombinant plasmid function (even for lethal transgenes)...
  45. In Vivo Gene Therapy for Treatment of Osteoarthritis
    Jeffrey B Mason; Fiscal Year: 2012
    ..Vectors containing Wnt10b and Dkk-1 transgenes will be injected into surgical and control joints and the efficacy of gene therapy-induced changes will be ..
  46. Satiety signaling in Caenorhabditis elegans
    Leon Avery; Fiscal Year: 2013
    ..sites and times of action of signal and receptor genes in the insulin, TGF-2, and cGMP pathways by expression of transgenes from neuron-specific promoters in combination with laser microsurgery (hypotheses 2, 4, and 5)...
  47. Genetic Pathways Directing Ventral Folding Morphogenesis in Mammalian Embryos
    ELIZABETH H LACY; Fiscal Year: 2013
    ..Three specific aims will investigate this hypothesis. Aim 1 uses lineage-specific Cre transgenes and a conditional allele of Bmpr1a to determine if AVE-expressed Bmp2 signals to definitive endoderm, ectoderm ..
  48. Biological Mechanism of FSGS-1
    Martin R Pollak; Fiscal Year: 2011
    ..These will include transgenes with an inactivated ABS1 and transgenes altering phosphorylation sites. 4...
  49. Role of PDZK1 in lipid metabolism and atherosclerosis
    Olivier N Kocher; Fiscal Year: 2013
    ..correlate the results obtained in vitro using a transgenic mouse model, where altered (mutated/truncated) PDZK1 transgenes will be expressed in the liver of PDZK1 KO mice...
    CAROLYN GIBSON; Fiscal Year: 2012
    ..dimensions and orientation in the Amelx null, MMP20 null, and Amelx null mice with normal and mutated amelogenin transgenes for fundamental information about the role of the amelogenin C-terminus in vivo;(iii) To determine the role of ..
  51. Human tissue-engineered blood vessels using induced pluripotent stem cells
    Yibing Qyang; Fiscal Year: 2013
    ..As the potential reactivation of reprogramming transgenes in these iPSCs could ultimately affect their safety as therapy and utility in disease modeling, we will generate ..
  52. Cell Type-Specific Roles of TLR Signaling In Immune Responses
    Anthony L DeFranco; Fiscal Year: 2012
    ..This conditional allele of myd88 will be used together with transgenes that express Cre either selectively in dendritic cells (CD11c-Cre), selectively in macrophages and neutrophils (..
    Anthony A James; Fiscal Year: 2013
    ..stephensi carrying one or more transgenes expressing anti-parasite scFvs relative to control laboratory colonies using life-table parameters...
  54. Novel treatment for polycystic kidney disease
    Vicente E Torres; Fiscal Year: 2013
    ..Conditional kidney specific transgenes will be generated to confirm positive results only...
  55. Mutant human a-synuclein toxicity in mice
    Deborah E Cabin; Fiscal Year: 2012
    ..2005). Thus transgenes driven by the human a- synuclein promoter will be studied on both wild type and a-synuclein null backgrounds...
  56. Transgenic tools for Gal4 regulated gene expression in zebrafish
    Michael Parsons; Fiscal Year: 2012
    ..An additional goal is to optimize the Gal4/UAS system by exploring the basis of transcriptional silencing of zebrafish transgenes and devising approaches to overcome it.
  57. CB1 and DA Receptor Deletion by RNAi in Cocaine Self-Administration
    CAROLINE EVELYN BASS; Fiscal Year: 2011
    ..her skills in the development of adenoassociated virus (AAV) vectors for the delivery of RNA interference (RNAi) transgenes to develop her own project focused on the knock down of D1, D2 and CB1 receptors in strategically defined brain ..
  58. COPAS BIOSORT Worm Sorter
    WOLFGANG B LIEDTKE; Fiscal Year: 2010
    ..advantage is optic transparency of the organism and availability of numerous gene-specific promoters that direct transgenes such as fluorescent proteins to specific cells, including neurons...
  59. Role of Ectodermal Signals in Facial Prominence Outgrowth and Development
    Trevor J Williams; Fiscal Year: 2013
    ..A number of Cre recombinase transgenes can target gene expression in the developing facial ectoderm, but most are limited by the extent and/or timing ..
  60. The Jak/Stat Pathway and Mitochondrial Function
    ANDREW CHARLES LARNER; Fiscal Year: 2013
    ..mitochondria function appear to oppose the effects of mitoStat3, we will examine if mitochondrial targeted Stat3 transgenes show enhanced protection in a Stat1-/- background...
  61. Genetic Dissection of Context-Dependent and Opposing Roles of Androgen Receptor
    Diane M Robins; Fiscal Year: 2013
    ..In Aim II, pathologic activity of variant ARs will be tested in mice with prostate- targeted transgenes, in mice predisposed to cancer by deletion of one PTEN allele, and in mice with varying androgen axis strength...
  62. Neuronal Role of Lipid Flippases
    KONRAD ERNST ZINSMAIER; Fiscal Year: 2011
    ..federal funding to test the synaptic role of dATP8B, we suggest in Aim 1 to generate antibodies and tagged transgenes to resolve the tissue-specific expression pattern and synaptic localization of dATP8B and its putative co-factor ..
    Leonard Shultz; Fiscal Year: 1999
    ..listed below focus on the use of genes disrupted by homologous recombination and the use of human growth factor transgenes to maximize the potential of NOD/LtSz-scid/scid mice as hosts for human adult peripheral blood leukocytes (PBL), ..
    EDWARD ROSLONIEC; Fiscal Year: 2001
    ..These transgenes were subsequently shown to confer susceptibility to collagen induced arthritis in normally resistant B10.M...
  65. Studies of Collagen Gene Regulation in Two Murine Models
    Stephen Clark; Fiscal Year: 2005
    ..These elements will be defined by studying the expression of Collal CAT reporter transgenes bearing various segments of the 5' promoter region as well as specific deletions of the first intron...
    Thaddeus Golos; Fiscal Year: 2003
    ..Specific Aim 1 is to deliver transgenes to rhesus monkey preimplantation embryos with Epstein-Barr Virus (EBV)-based and retrovirus-based vectors...
    Raif Geha; Fiscal Year: 2007
    ..assess the role of TRAF proteins in CD40 mediated isotype switching, we have examined the ability of mutant CD40 transgenes that lack one or both TRAF binding sites (ATRAF6, ATRAF2/3 and ATRAFs respectively) to reconstitute isotype ..
    Ursula Storb; Fiscal Year: 2000
    ..plans are described in this proposal: During the current grant period it was found that gamma2b heavy chain transgenes inhibit the development of B cells, because gamma2b causes strong feedback inhibition of H gene rearrangement, ..
  69. Nucleic acid delivery of growth factors and heparan sulfate proteoglycans for enh
    April L Ellis; Fiscal Year: 2010
    ..Plasmid DNA expression constructs of the proteoglycan and the VEGF165 transgenes are characterized and ready for this proposed project...
  70. Differential regulation of multiple transgenes for treatment of eye disease
    RICHARD J contact SAMULSKI; Fiscal Year: 2010
    ..While these systems have been proven effective, the requirements prohibit differential regulation of multiple transgenes. Additionally, the requirement of co-delivering a trans-activator adds significantly to the payload required of ..
  71. Photoreceptor Degeneration Assay-Transgenic Xenopus(RMI)
    Nicholas Marsh Armstrong; Fiscal Year: 2005
    ..We have developed lines of Xenopus laevis in which transgenes both cause rod photoreceptors to degenerate in a manner that resemble human retinitis pigmentosa as well as ..
    Douglas Fearon; Fiscal Year: 2006
    ..For Aims 2-5, mice will be created in which transgenes can be inducibly expressed in a lineage-specific manner...
  73. NIH Director's Pioneer Award
    Vicki Chandler; Fiscal Year: 2009
    ..In New Research Direction, I briefly describe the new approaches I am proposing, explain how this builds on my past work, and discuss why the NDPA process provides a unique opportunity to pursue this work. ..
  74. In vitro culture and transformation of Wolbachia from mosquitoes
    Ann Fallon; Fiscal Year: 2007
    ..The next challenge to implementation of transgenic mosquitoes is development of tools to drive the spread of transgenes from small numbers of laboratory- manipulated mosquitoes into field populations...