genetic transduction


Summary: The transfer of bacterial DNA by phages from an infected bacterium to another bacterium. This also refers to the transfer of genes into eukaryotic cells by viruses. This naturally occurring process is routinely employed as a GENE TRANSFER TECHNIQUE.

Top Publications

  1. 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
  2. pmc AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method
    Ronald L Klein
    Department of Pharmacology, Toxicology, and Neuroscience, Louisiana State University Health Sciences Center, Shreveport, Louisiana, USA
    Mol Ther 16:89-96. 2008
  3. ncbi A hybrid vector for ligand-directed tumor targeting and molecular imaging
    Amin Hajitou
    Department of Genitourinary Medical Oncology, The University of Texas M D Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA
    Cell 125:385-98. 2006
  4. ncbi Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
    Alessandro Aiuti
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Milan, Italy
    Science 296:2410-3. 2002
  5. ncbi A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice
    S Charrier
    Genethon UMR CNRS 8115, Evry, France
    Gene Ther 12:597-606. 2005
  6. pmc Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates
    Patricia A Lawlor
    Department of Molecular Medicine and Pathology, The University of Auckland, Auckland, New Zealand
    Mol Ther 17:1692-702. 2009
  7. ncbi Major role of local immune responses in antibody formation to factor IX in AAV gene transfer
    L Wang
    Department of Pediatrics, University of Pennsylvania Medical Center, Philadelphia, PA, USA
    Gene Ther 12:1453-64. 2005
  8. ncbi Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells
    Greg M Podsakoff
    Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA 90027, USA
    Mol Ther 12:77-86. 2005
  9. ncbi A single immunization with a minute dose of a lentiviral vector-based vaccine is highly effective at eliciting protective humoral immunity against West Nile virus
    Maria Candela Iglesias
    Groupe de Virologie Moléculaire et Vectorologie, Institut Pasteur, 28 rue du Dr Roux 75724 Paris Cedex 15, France
    J Gene Med 8:265-74. 2006
  10. pmc Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain
    Cassia N Cearley
    W F Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA
    Mol Ther 16:1710-8. 2008

Detail Information

Publications313 found, 100 shown here

  1. 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...
  2. pmc AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method
    Ronald L Klein
    Department of Pharmacology, Toxicology, and Neuroscience, Louisiana State University Health Sciences Center, Shreveport, Louisiana, USA
    Mol Ther 16:89-96. 2008
    ..The purification method can therefore impact the transduction pattern as well as the results when comparing serotype strengths...
  3. ncbi A hybrid vector for ligand-directed tumor targeting and molecular imaging
    Amin Hajitou
    Department of Genitourinary Medical Oncology, The University of Texas M D Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX 77030, USA
    Cell 125:385-98. 2006
    ..This new class of targeted hybrid viral particles will enable a wide range of applications in biology and medicine...
  4. ncbi Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
    Alessandro Aiuti
    San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Milan, Italy
    Science 296:2410-3. 2002
    ..Both patients are currently at home and clinically well, with normal growth and development. These results indicate the safety and efficacy of HSC gene therapy combined with nonmyeloablative conditioning for the treatment of SCID...
  5. ncbi A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice
    S Charrier
    Genethon UMR CNRS 8115, Evry, France
    Gene Ther 12:597-606. 2005
    ..These data support further development of such lentiviral vectors for the gene therapy of WAS...
  6. pmc Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates
    Patricia A Lawlor
    Department of Molecular Medicine and Pathology, The University of Auckland, Auckland, New Zealand
    Mol Ther 17:1692-702. 2009
  7. ncbi Major role of local immune responses in antibody formation to factor IX in AAV gene transfer
    L Wang
    Department of Pediatrics, University of Pennsylvania Medical Center, Philadelphia, PA, USA
    Gene Ther 12:1453-64. 2005
    ..IX. In summary, the data document substantial influence of target tissue, local antigen presentation, and antigen levels on lymphocyte responses to F.IX...
  8. ncbi Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells
    Greg M Podsakoff
    Childrens Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA 90027, USA
    Mol Ther 12:77-86. 2005
    ..These findings indicate that there was a selective survival advantage for PBMC containing the huM10 gene during the time of increased HIV-1 load...
  9. ncbi A single immunization with a minute dose of a lentiviral vector-based vaccine is highly effective at eliciting protective humoral immunity against West Nile virus
    Maria Candela Iglesias
    Groupe de Virologie Moléculaire et Vectorologie, Institut Pasteur, 28 rue du Dr Roux 75724 Paris Cedex 15, France
    J Gene Med 8:265-74. 2006
    ..WNV is a mosquito-borne flavivirus that emerged in North America and causes encephalitis in humans, birds and horses. Neutralizing anti-WNV antibodies have been shown to be crucial for protection against WNV encephalitis...
  10. pmc Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain
    Cassia N Cearley
    W F Goodman Center for Comparative Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA
    Mol Ther 16:1710-8. 2008
    ..These unique patterns of cell transduction expand the potential repertoire for targeting AAV vectors to selected subsets of brain cells...
  11. ncbi Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo
    D M McCarty
    Division of Drug Delivery and Disposition, School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599 7352, USA
    Gene Ther 10:2112-8. 2003
    ..The scAAV is unaffected by this barrier, and provides an extremely efficient vector for gene transfer into many types of cells in vivo...
  12. pmc Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system
    B L Davidson
    Program in Gene Therapy, Department of Internal Medicine, University of Iowa College of Medicine, Iowa City, IA 52242, USA
    Proc Natl Acad Sci U S A 97:3428-32. 2000
    ..These differences could be exploited to improve gene therapy for CNS disorders...
  13. pmc Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization
    Michael A Lochrie
    Avigen, Inc, Alameda, California 94502 6541, USA
    J Virol 80:821-34. 2006
    ..These mutations define areas on the surface of the AAV-2 capsid that are important determinants of transduction and antibody neutralization...
  14. ncbi Long-term survival and concomitant gene expression of ribozyme-transduced CD4+ T-lymphocytes in HIV-infected patients
    Janet L MacPherson
    Johnson and Johnson Research Pty Limited, Locked Bag 4555, Strawberry Hills, Sydney, NSW Australia 2012
    J Gene Med 7:552-64. 2005
    ..We report here the results of a phase I gene transfer clinical trial using Rz2...
  15. ncbi Deregulated expression of HOXB4 enhances the primitive growth activity of human hematopoietic cells
    Christian Buske
    Terry Fox Laboratory, British Columbia Cancer Agency, and the Department of Medical Genetics, University of British Columbia, Vancouver, British Columbia, Canada
    Blood 100:862-8. 2002
    ..These findings reveal HOXB4 as a novel, positive regulator of the primitive growth activity of human hematopoietic progenitor cells and underline the relevance of early developmental factors for stem cell fate decisions...
  16. pmc Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous
    Deniz Dalkara
    Department of Chemical Engineering, The University of California at Berkeley, Berkeley, California 94720 3190, USA
    Mol Ther 17:2096-102. 2009
    ..These findings may help avoid limitations, risks, and damage associated with subretinal injections currently necessary for clinical gene therapy...
  17. pmc Engineered lentivector targeting of dendritic cells for in vivo immunization
    Lili Yang
    Division of Biology, California Institute of Technology, 1200 E California Blvd, Pasadena, California 91125, USA
    Nat Biotechnol 26:326-34. 2008
    ..Thus, lentiviral vectors targeting dendritic cells provide a simple method of producing effective immunity and may provide an alternative route for immunization with protein antigens...
  18. ncbi High-level sustained transgene expression in human embryonic stem cells using lentiviral vectors
    Yue Ma
    National Primate Research Center, School of Medicine, University of Wisconsin, Madison, Wisconsin, USA
    Stem Cells 21:111-7. 2003
    ..These results also have important implications for the possible future use of gene-modified human ES cells in transplantation and tissue regeneration applications...
  19. pmc Cancer regression in patients after transfer of genetically engineered lymphocytes
    Richard A Morgan
    Surgery Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, 10 Center Drive, Bethesda, MD 20892, USA
    Science 314:126-9. 2006
    ..This study suggests the therapeutic potential of genetically engineered cells for the biologic therapy of cancer...
  20. pmc Mechanism of reduction in titers from lentivirus vectors carrying large inserts in the 3'LTR
    Fabrizia Urbinati
    Division of Experimental Hematology and Cancer Biology, Cincinnati Children s Hospital Medical Center, University of Cincinnati, Cincinnati, Ohio 45229 3039, USA
    Mol Ther 17:1527-36. 2009
    ..These studies have important implications in design of integrating vectors...
  21. ncbi Time course of transgene expression after intrastriatal pseudotyped rAAV2/1, rAAV2/2, rAAV2/5, and rAAV2/8 transduction in the rat
    Sharon Reimsnider
    Department of Neuroscience, Powell Gene Therapy Center, McKnight Brain Institute, University of Florida College of Medicine, Gainesville, Florida 32610, USA
    Mol Ther 15:1504-11. 2007
    ..The inflammatory response to these rAAV vectors was present up to 4 weeks after transduction but was not apparent 9 months post injection. Thus, rAAV-mediated transgene expression begins earlier than previously thought...
  22. ncbi Improved foamy virus vectors with minimal viral sequences
    Grant Trobridge
    Department of Medicine, Division of Hematology, University of Washington, Seattle, Washington 98195, USA
    Mol Ther 6:321-8. 2002
    ..A deltaphi vector containing a 9.2-kb transgene cassette was produced at unconcentrated titers of over 10(5) transducing units/ml, demonstrating the utility of these deleted vectors for large therapeutic genes...
  23. pmc DNA shuffling of adeno-associated virus yields functionally diverse viral progeny
    James T Koerber
    Department of Chemical Engineering, University of California at Berkeley, Berkeley, California 94720 1462, USA
    Mol Ther 16:1703-9. 2008
    ..Thus, directed evolution can potentially yield unlimited numbers of new AAV variants with novel gene-delivery properties, and subsequent analysis of these variants can further extend basic knowledge of AAV biology...
  24. pmc Identification of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding
    Shaun R Opie
    Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, Florida 32610 0266, USA
    J Virol 77:6995-7006. 2003
    ..Another mutant, H358A, was defective for capsid assembly. Finally, an R459A mutant produced significantly lower levels of full capsids, suggesting a packaging defect...
  25. ncbi Polybrene increases retrovirus gene transfer efficiency by enhancing receptor-independent virus adsorption on target cell membranes
    Howard E Davis
    Center for Engineering in Medicine Surgical Services, Massachusetts General Hospital, Harvard Medical School and the Shriners Hospital for Children, Boston, MA 02114, USA
    Biophys Chem 97:159-72. 2002
    ..The implication of these findings with respect to the development of targeted retrovirus-mediated gene therapy protocols is discussed...
  26. ncbi In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance
    Brian D Brown
    San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy
    Blood 109:2797-805. 2007
  27. pmc Designer gene delivery vectors: molecular engineering and evolution of adeno-associated viral vectors for enhanced gene transfer
    Inchan Kwon
    Department of Chemical Engineering and Helen Wills Neuroscience Institute, University of California, 201 Gilman Hall, Berkeley, California 94720 1462, USA
    Pharm Res 25:489-99. 2008
    ..Molecular engineering and directed evolution of AAV vectors therefore offer promise for generating 'designer' gene delivery vectors with enhanced properties...
  28. pmc Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity
    Joseph E Rabinowitz
    Gene Therapy Center Laboratoire de Thérapie Génique, CHU Hotel Dieu, 44035 Nantes Cedex 01, France
    J Virol 76:791-801. 2002
    ..The development of these helper plasmids should facilitate direct comparisons of serotypes, as well as begin the standardization of production for further clinical development...
  29. pmc Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
    Amit C Nathwani
    Department of Haemotology, University College London, UK, and Department of Surgery, St Jude Children s Research Hospital, Memphis, TN, USA
    Blood 109:1414-21. 2007
    ..These results should make vector administration to patients, especially those with a severe bleeding diathesis, significantly easier and safer...
  30. pmc Efficient entry inhibition of human and nonhuman primate immunodeficiency virus by cell surface-expressed gp41-derived peptides
    R C Zahn
    Division of Viral Pathogenesis, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA 02115, USA
    Gene Ther 15:1210-22. 2008
  31. ncbi Lentivirally transduced dendritic cells as a tool for cancer immunotherapy
    Karine Breckpot
    Department of Physiology and Immunology, Medical School of the Vrije Universiteit Brussel VUB, Laarbeeklaan 103 E, 1090 Brussels, Belgium
    J Gene Med 5:654-67. 2003
    ..Dendritic cells (DC) are the professional antigen-presenting cells of the immune system, fully equipped to prime naive T cells and thus essential components for cancer immunotherapy...
  32. pmc Molecular evolution of adeno-associated virus for enhanced glial gene delivery
    James T Koerber
    Department of Chemical Engineering, Helen Wills Neuroscience Institute, The University of California, Berkeley, California 94720 1462, USA
    Mol Ther 17:2088-95. 2009
  33. ncbi HIV-1 Tat-mediated protein transduction of Cu,Zn-superoxide dismutase into pancreatic beta cells in vitro and in vivo
    Won Sik Eum
    Department of Physiology, College of Medicine, Hallym University, Chunchon 200 702, South Korea
    Free Radic Biol Med 37:339-49. 2004
    ..These results suggest that the transduction of Tat-SOD offers a new strategy for protecting pancreatic beta cells from destruction by relieving oxidative stress in ROS-implicated diabetes...
  34. ncbi Rev-independent expression of synthetic gag-pol genes of human immunodeficiency virus type 1 and simian immunodeficiency virus: implications for the safety of lentiviral vectors
    R Wagner
    Institut für medizinische Mikrobiologie and Hygiene, Universitat Regensburg, D 93053 Regensburg, Germany
    Hum Gene Ther 11:2403-13. 2000
    ..By eliminating regions of homology and sequences involved in packaging, synthetic gag-pol genes should improve the safety profile of lentiviral vectors...
  35. ncbi Functional integration of electrically active cardiac derivatives from genetically engineered human embryonic stem cells with quiescent recipient ventricular cardiomyocytes: insights into the development of cell-based pacemakers
    Tian Xue
    Department of Medicine, Johns Hopkins University, Baltimore, MD 21205, USA
    Circulation 111:11-20. 2005
    ..Although CMs can be derived from hESCs ex vivo, it remains uncertain whether a functional syncytium can be formed between donor and recipient cells after engraftment...
  36. ncbi Efficient gene delivery to human and rodent islets with double-stranded (ds) AAV-based vectors
    K K Rehman
    Department of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, Pittsburgh, PA 15261, USA
    Gene Ther 12:1313-23. 2005
    ..Finally, we demonstrated that dsAAV serotypes 2, 6, and 8 infect human islets efficiently. Taken together, these results suggest that dsAAV based vectors are highly appropriate for gene transfer to islets to facilitate transplantation...
  37. ncbi Human T-lymphocyte cytotoxicity and proliferation directed by a single chimeric TCRzeta /CD28 receptor
    John Maher
    Department of Human Genetics Medicine, Gene Transfer and Somatic Cell Engineering Laboratory, and Immunology Program, Memorial Sloan Kettering Cancer Center, New York, NY 10021, USA
    Nat Biotechnol 20:70-5. 2002
    ..These findings have important implications for adoptive immunotherapy of cancer, especially in the context of tumor cells that fail to express major histocompatibility complex antigens and co-stimulatory molecules...
  38. ncbi Certification assays for HIV-1-based vectors: frequent passage of gag sequences without evidence of replication-competent viruses
    Lakshmi Sastry
    Department of Microbiology and Immunology, Indianapolis, Indiana 46202, USA
    Mol Ther 8:830-9. 2003
    ..These findings have important implications for the design and safety of HIV-1-based vectors intended for clinical applications...
  39. 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...
  40. ncbi Modulation of human dendritic-cell function following transduction with viral vectors: implications for gene therapy
    Peng H Tan
    Department of Immunology, Faculty of Medicine, Imperial College London, Hammersmith Hospital, Du Cane Road, London, W12 ONN, United Kingdom
    Blood 105:3824-32. 2005
    ..Inhibition of IDO restores the ability of mDCs to stimulate an MLR, indicating that IDO is responsible for the modulation of mDC function. These data have important implications for the use of viral vectors in the transduction of DCs...
  41. pmc Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles
    Wuping Li
    Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599 7352, USA
    Mol Ther 16:1252-60. 2008
    ..Application of this technology to alternative cell/tissue types using AAV or other viral capsid sequences is likely to yield a new class of biological nanoparticles as vectors for human gene transfer...
  42. pmc Impact of the central polypurine tract on the kinetics of human immunodeficiency virus type 1 vector transduction
    Bénédicte Van Maele
    Rega Institute for Medical Research, Leuven, Flanders, Belgium
    J Virol 77:4685-94. 2003
  43. pmc RevM10-expressing T cells derived in vivo from transduced human hematopoietic stem-progenitor cells inhibit human immunodeficiency virus replication
    M L Bonyhadi
    Progenesys Program, SyStemix, Inc, Palo Alto, California 94304, USA
    J Virol 71:4707-16. 1997
    ..Moreover, we demonstrate that RevM10 can inhibit HIV replication in T cells derived from transduced HPSC after expansion in vitro. This is the first demonstration of anti-HIV efficacy in T cells derived from transduced human HPSC...
  44. pmc Wild-type levels of nuclear localization and human immunodeficiency virus type 1 replication in the absence of the central DNA flap
    Ana Limon
    Department of Cancer Immunology and AIDS, Dana Farber Cancer Institute, and Department of Pathology, Harvard Medical School, Boston, Massachusetts 02115, USA
    J Virol 76:12078-86. 2002
    ..Our results indicate that the central DNA flap does not play a major role in either preintegration complex nuclear import or HIV-1 replication in a variety of cell types...
  45. ncbi Two key challenges for effective adenovirus-mediated liver gene therapy: innate immune responses and hepatocyte-specific transduction
    Delphyne Descamps
    Institut Pasteur, Unité de Défense Innée et Inflammation, 75724 Paris Cedex 15, France
    Curr Gene Ther 9:115-27. 2009
    ..The full potential of Ad vectors will only be reached when their immunogenicity is abolished and hepatocyte-specific transduction achieved...
  46. pmc Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro
    Maria P Limberis
    Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA
    Mol Ther 17:294-301. 2009
    ..2 exhibits improved transduction efficiency compared to previously reported AAVs in mouse airways and in culture models of human airway epithelium and that this vector requires further development for preclinical and clinical testing...
  47. pmc Genetic fate of recombinant adeno-associated virus vector genomes in muscle
    Bruce C Schnepp
    Columbus Children s Research Institute, Columbus Children s Hospital, and Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, Ohio 43205, USA
    J Virol 77:3495-504. 2003
    ..5% of vector DNA was not integrated. Additional analyses using a novel DNA exonuclease showed that the majority of the rAAV vector DNA in muscle persisted over time as transcriptionally active monomeric and concatameric episomes...
  48. ncbi Dystrophinopathy carrier determination and detection of protein deficiencies in muscular dystrophy using lentiviral MyoD-forced myogenesis
    Sandra T Cooper
    Neurogenetics Research Unit and Institute for Neuromuscular Research, The Children s Hospital at Westmead, Locked Bag 4001, Sydney, NSW 2145, Australia
    Neuromuscul Disord 17:276-84. 2007
  49. ncbi Targeted transgene expression in rat brain using lentiviral vectors
    Johan Jakobsson
    Wallenberg Neuroscience Center, Department of Physiological Science, Lund University, Lund, Sweden
    J Neurosci Res 73:876-85. 2003
  50. ncbi Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow
    Dao Pan
    Department of Pediatrics and Institute of Human Genetics, University of Minnesota, Minneapolis, Minnesota 55455, USA
    Mol Ther 6:19-29. 2002
    ..The observation of bone marrow transduction after intravenous vector administration suggests the possibility of an in vivo approach to stem cell gene therapy...
  51. pmc Functional role of inward rectifier current in heart probed by Kir2.1 overexpression and dominant-negative suppression
    Junichiro Miake
    Institute of Molecular Cardiobiology, Department of Medicine, The Johns Hopkins University School of Medicine, Baltimore, Maryland, USA
    J Clin Invest 111:1529-36. 2003
    ..Additionally, the long-QT phenotype seen in Andersen syndrome is a direct consequence of dominant-negative suppression of Kir2 channel function...
  52. ncbi A novel dual-targeted lentiviral vector leads to specific transduction of prostate cancer bone metastases in vivo after systemic administration
    Nonia Pariente
    Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles, California 90095, USA
    Mol Ther 15:1973-81. 2007
    ..The LV presented here is a powerful tool for obtaining stable and site-specific gene expression and can be easily modified for its use in other diseases...
  53. pmc Targeted cell entry of lentiviral vectors
    Sabrina Funke
    1Division of Medical Biotechnology, Paul Ehrlich Institut, Langen, Germany
    Mol Ther 16:1427-36. 2008
    ..Because MV enters cells through direct fusion at the cell membrane, this novel targeting system should be widely applicable...
  54. ncbi Vectors selected from adeno-associated viral display peptide libraries for leukemia cell-targeted cytotoxic gene therapy
    Stefan Michelfelder
    Department of Hematology and Oncology, University of Freiburg Medical Center, Freiburg, Germany
    Exp Hematol 35:1766-76. 2007
    ..For acute myeloid leukemia (AML), gene therapy may be used to treat patients refractory to conventional chemotherapy. However, availability of vectors sufficiently and specifically transducing this cell type is very limited...
  55. ncbi Engineering targeted viral vectors for gene therapy
    Reinhard Waehler
    Division of Human Gene Therapy, 502 Biomedical Research Building II, 901 19th Street, South Birmingham, Alabama 35294 2172, USA
    Nat Rev Genet 8:573-87. 2007
    ..Although important challenges remain for in vivo applications, the first clinical trials with targeted vectors have already begun to take place...
  56. ncbi Specific transgene expression in human and mouse CD4+ cells using lentiviral vectors with regulatory sequences from the CD4 gene
    Gilles Marodon
    Centre National de la Recherche Scientifique UMR 7087, Biologie et Thérapeutique des Pathologies Immunitaires, Centre d Etude et de Recherche en Virologie et en Immunologie, hôpital La Pitié Salpêtrière, Paris, France
    Blood 101:3416-23. 2003
    ..Ultimately, these vectors may prove useful for in situ injections for in vivo gene therapy of HIV infection or genetic immunodeficiencies...
  57. ncbi Primary polyclonal human T lymphocytes targeted to carcino-embryonic antigens and neural cell adhesion molecule tumor antigens by CD3zeta-based chimeric immune receptors
    David E Gilham
    CRC Department of Medical Oncology, Paterson Institute for Cancer Research, University of Manchester, UK
    J Immunother 25:139-51. 2002
    ..Viability was improved by a costimulatory signal indicating that such signals may be vital in the maintenance of long-term functional activity of receptor modified T lymphocytes...
  58. ncbi Beyond oncolytic virotherapy: replication-competent retrovirus vectors for selective and stable transduction of tumors
    Charlotte Dalba
    EPIXIS, Paris, France
    Curr Gene Ther 5:655-67. 2005
    ..In addition, the ability of these vectors to achieve stable transgene expression in infected tumor cells may allow therapeutic applications that move beyond oncolysis per se...
  59. ncbi Neonatal intraperitoneal or intravenous injections of recombinant adeno-associated virus type 8 transduce dorsal root ganglia and lower motor neurons
    Kevin D Foust
    Department of Pediatrics, Evelyn F and William L McKnight Brian Institute, University of Florida College of Medicine, Gainesville, FL 32610, USA
    Hum Gene Ther 19:61-70. 2008
    ..Our data suggest that systemic injection of rAAV8 is not an effective delivery route to target lower motor neurons, but could be useful for targeting sensory pathways in chronic pain...
  60. ncbi HOXB4-induced expansion of adult hematopoietic stem cells ex vivo
    Jennifer Antonchuk
    Terry Fox Laboratory, British Columbia Cancer Agency, 601 West 10th Avenue, V5Z 1L3, Vancouver, British Columbia, Canada
    Cell 109:39-45. 2002
    ..Importantly, these HSCs retained full lympho-myeloid repopulating potential and enhanced in vivo regenerative potential, demonstrating the feasibility of achieving significant ex vivo expansion of HSCs without functional impairment...
  61. ncbi Hot spots of retroviral integration in human CD34+ hematopoietic cells
    Claudia Cattoglio
    Italian Institute of Technology, Unit of Molecular Neuroscience, Istituto Scientifico H San Raffaele, Milan, Italy
    Blood 110:1770-8. 2007
    ..The lower propensity of LV vectors for integrating in potentially dangerous regions of the human genome may be a factor determining a better safety profile for gene therapy applications...
  62. ncbi Enhanced purification of cell-permeant Cre and germline transmission after transduction into mouse embryonic stem cells
    Michael Peitz
    Stem Cell Engineering Group, Institute of Reconstructive Neurobiology, Life and Brain Center and Hertie Foundation, University of Bonn, Bonn, Germany
    Genesis 45:508-17. 2007
    ..The protocol described here allows rapid and highly efficient conditional mutagenesis of cultured cells...
  63. ncbi Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors
    A Schambach
    Department of Experimental Hematology, Hannover Medical School, Carl Neuberg Strasse 1, D 30625 Hannover, Germany
    Gene Ther 13:1524-33. 2006
    ..This study introduces a new generation of efficient gammaretroviral SIN vectors as a platform for further optimizations of retroviral vector performance...
  64. ncbi Lentivirus-mediated gene transfer in primary T cells is enhanced by a central DNA flap
    V Dardalhon
    , UMR 5535/IFR 22, France
    Gene Ther 8:190-8. 2001
    ..Thus, this system allows stable expression of transgenes in T lymphocytes following a short ex vivo transduction protocol...
  65. pmc Visualization of targeted transduction by engineered lentiviral vectors
    K I Joo
    Mork Family Department of Chemical Engineering and Materials Science, University of Southern California, Los Angeles, CA 90089, USA
    Gene Ther 15:1384-96. 2008
    ..This imaging study sheds some light on the infection mechanism of the engineered lentivirus and can be beneficial to the design of more efficient gene delivery vectors...
  66. ncbi Reversal of neurochemical changes and pain-related behavior in a model of neuropathic pain using modified lentiviral vectors expressing GDNF
    Sophie Pezet
    The London Pain Consortium, Neurorestoration, The Wolfson Centre for Age Related Diseases, King s College London, London SE1 1UL, UK
    Mol Ther 13:1101-9. 2006
  67. ncbi Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
    Zhong Wang
    Dept of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, 200 Lothrop Street, Pittsburgh, Pennsylvania 15261, USA
    Nat Biotechnol 23:321-8. 2005
    ..Gene expression persisted in muscle and heart, but diminished in tissues undergoing rapid cell division, such as neonatal liver. This technology should prove useful for muscle-directed systemic gene therapy...
  68. ncbi Engraftment of retroviral EGFP-transduced bone marrow in mice prevents rejection of EGFP-transgenic skin grafts
    Goran Andersson
    BioTransplant Incorporated, Charlestown Navy Yard, Boston, Massachusetts 02129, USA
    Mol Ther 8:385-91. 2003
  69. ncbi Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy
    T Vandendriessche
    Center for Transgene Technology and Gene Therapy, Flanders Interuniversity Institute for Biotechnology, University of Leuven, Leuven, Belgium
    J Thromb Haemost 5:16-24. 2007
    ..Adeno-associated viral (AAV) and lentiviral vectors are promising vectors for gene therapy for hemophilia because they are devoid of viral genes and have the potential for long-term gene expression...
  70. ncbi Efficient gene transfer into human primary blood lymphocytes by surface-engineered lentiviral vectors that display a T cell-activating polypeptide
    Marielle Maurice
    Laboratoire Vectorologie Rétrovirale et Thérapie Génique, Unité de Virologie Humaine, U412 INSERM, IFR 74 and ENS de Lyon, 46 allee d Italie, 69364 Lyon Cedex 07, France
    Blood 99:2342-50. 2002
    ..Moreover these results provide a proof of concept for an approach that may have utility in various gene transfer applications, including in vivo gene delivery...
  71. ncbi An adenovirus vector with a chimeric fiber derived from canine adenovirus type 2 displays novel tropism
    Joel N Glasgow
    Division of Human Gene Therapy, Department of Medicine, University of Alabama at Birmingham Birmingham, AL 35294 2172, USA
    Virology 324:103-16. 2004
    ..Further, we confirmed the importance of cellular integrins to Ad5Luc1-CK transduction. Herein, we present the rationale, design, purification, and characterization of a novel tropism modified, infectivity-enhanced Ad vector...
  72. ncbi Cellular manipulation of human embryonic stem cells by TAT-PDX1 protein transduction
    Young Do Kwon
    Institute of Reproductive Medicine and Population, Medical Research Center, Korea
    Mol Ther 12:28-32. 2005
    ..These results demonstrate that protein transduction could be used in the cellular manipulation of hESCs and would provide a significant breakthrough for basic and therapeutic research in hESCs...
  73. ncbi Gene delivery to hematopoietic stem cells using lentiviral vectors
    Hiroyuki Miyoshi
    Subteam for Manipulation of Cell Fate, BioResource Center, RIKEN Tsukuba Institute, Tsukuba, Ibaraki, Japan
    Methods Mol Biol 246:429-38. 2004
    ..The procedures include the isolation of human CD34+ cells, preparation of lentiviral vectors, transduction of human CD34+ cells, and in vivo analysis of transduced HSCs using NOD/SCID mice...
  74. pmc A versatile targeting system with lentiviral vectors bearing the biotin-adaptor peptide
    Kouki Morizono
    Department of Medicine, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA
    J Gene Med 11:655-63. 2009
    ..We have now developed a more stable conjugation strategy utilizing the interaction between avidin and biotin...
  75. ncbi Rate limiting steps of AAV transduction and implications for human gene therapy
    S Sanlioglu
    Department of Internal Medicine, Division of Pulmonary and Critical Care, Center for Gene Therapy, University of Iowa, College of Medicine, Iowa City, IA 52242, USA
    Curr Gene Ther 1:137-47. 2001
    ..This review will focus on the identification of molecular components important for recombinant AAV (rAAV) transduction while highlighting the techniques used to discover them and potential clinical application of research findings...
  76. ncbi Immune response to fetal calf serum by two adenosine deaminase-deficient patients after T cell gene therapy
    Laura Tuschong
    Clinical Gene Therapy Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892 1820, USA
    Hum Gene Ther 13:1605-10. 2002
    ..These findings suggest that the unique immune response mounted by patient 2 may have influenced the outcome of the gene transfer treatments in this patient...
  77. ncbi Lentiviral vector-mediated transduction of neural progenitor cells before implantation into injured spinal cord and brain to detect their migration, deliver neurotrophic factors and repair tissue
    Bas Blits
    The Miami Project to Cure Paralysis, University of Miami Miller School of Medicine, Miami, FL, USA
    Restor Neurol Neurosci 23:313-24. 2005
    ..Furthermore, when the expression cassette is incorporated into the host genome ex vivo, this technique also may be used as a method to trace cells following implantation into tissues of interest...
  78. ncbi Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy
    Ming Jie Li
    Division of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, CA 91010, USA
    Mol Ther 12:900-9. 2005
    ..Our data demonstrate the validity and efficacy of a combinatorial RNA-based gene therapy for the treatment of HIV-1 infection...
  79. pmc Antisense-mediated inhibition of human immunodeficiency virus (HIV) replication by use of an HIV type 1-based vector results in severely attenuated mutants incapable of developing resistance
    Xiaobin Lu
    VIRxSYS Corporation, Gaithersburg, MD 20877, USA
    J Virol 78:7079-88. 2004
    ..These clones were not found to be escape mutants, as their replicative ability was severely attenuated, and they did not replicate in the presence of vector...
  80. ncbi Expression of exogenous human telomerase in cultures of endometrial stromal cells does not alter their hormone responsiveness
    Claire S Barbier
    Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, 27599, USA
    Biol Reprod 73:106-14. 2005
  81. ncbi Pois(s)on--it's a question of dose
    B Fehse
    Bone Marrow Transplantation, University Hospital Eppendorf, Hamburg, Germany
    Gene Ther 11:879-81. 2004
  82. ncbi Predictable and efficient retroviral gene transfer into murine bone marrow repopulating cells using a defined vector dose
    Zhixiong Li
    Experimental Cell Therapy, Department of Hematology and Oncology, Hannover Medical School, Carl Neuberg Strasse 1, 30625 Hannover, Germany
    Exp Hematol 31:1206-14. 2003
    ..We developed and evaluated a reliable transduction protocol that is more related to clinical methods...
  83. pmc HIV sequence variation associated with env antisense adoptive T-cell therapy in the hNSG mouse model
    Rithun Mukherjee
    Department of Microbiology, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania, USA
    Mol Ther 18:803-11. 2010
    ..Unexpectedly, this enrichment was only detected after the challenge with HIV-1(BaL), where the viral genome would form an imperfect duplex with envAS, and not HIV-1(NL4-3), where a perfectly matched duplex would form...
  84. ncbi Adeno-associated viral gene transfer of transforming growth factor-beta1 to human mesenchymal stem cells improves cartilage repair
    M R Pagnotto
    Department of Orthopaedic Surgery, University of Pittsburgh, Pittsburgh, PA 15232, USA
    Gene Ther 14:804-13. 2007
    ..0047). These results show that AAV is a suitable vector for gene delivery to improve the cartilage repair potential of human mesenchymal stem cells...
  85. pmc Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors
    C L Halbert
    Fred Hutchinson Cancer Research Center, 1100 Fairview Ave. North, Seattle, WA 98109, USA
    J Virol 75:6615-24. 2001
    ..These results, combined with our previous results showing lower immunogenicity of AAV6 than of AAV2 vectors, indicate that AAV6 vectors may provide significant advantages over AAV2 for gene therapy of lung diseases like cystic fibrosis...
  86. 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
  87. ncbi Retroviral vector silencing during iPS cell induction: an epigenetic beacon that signals distinct pluripotent states
    Akitsu Hotta
    Developmental and Stem Cell Biology Program, SickKids Hospital, Toronto, Ontario, Canada
    J Cell Biochem 105:940-8. 2008
  88. pmc Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes
    Katsuyuki Mitomo
    DNAVEC Corporation, Tsukuba, Japan
    Mol Ther 18:1173-82. 2010
    ..Our data suggest that this lentiviral vector may provide a step change in airway transduction efficiency relevant to a clinical programme of gene therapy for CF...
  89. ncbi Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8
    M Natkunarajah
    Division of Molecular Therapy, UCL Institute of Ophthalmology, University College London, London, UK
    Gene Ther 15:463-7. 2008
    ..This improved transduction efficiency might facilitate the development of improved gene therapy protocols for inherited retinal degenerations, particularly those caused by defects in photoreceptor-specific genes...
  90. ncbi Differentiation of transplanted bone marrow cells in the adult mouse brain
    K Nakano
    Department of Biochemistry and Molecular Biology, Nippon Medical School, 1-1-5 Sendagi, Bunkyo-ku, Tokyo 113-8602, Japan
    Transplantation 71:1735-40. 2001
    ..Autologous bone marrow cells may be useful as carriers for ex vivo gene therapy for lysosomal disorders with neurological symptoms...
  91. pmc A high throughput method for genome-wide analysis of retroviral integration
    Julie Mantovani
    Genethon CNRS Université d Evry Val d Essonne, UMR 8115, Evry, France
    Nucleic Acids Res 34:e134. 2006
    ..As a validation of this method, we have characterized 1349 different lentiviral vector insertion sites in transduced HeLa cells, from only 487 sequencing reactions, with a background of <2% false positive tags...
  92. pmc Adeno-associated virus capsids displaying immunoglobulin-binding domains permit antibody-mediated vector retargeting to specific cell surface receptors
    Martin U Ried
    Laboratorium für Molekulare Biologie, Genzentrum, Germany
    J Virol 76:4559-66. 2002
    ..These results demonstrate for the first time that an immunoglobulin binding domain can be inserted into the AAV2 capsid and coupled to various antibodies, which mediate the retargeting of rAAV vectors to specific cell surface receptors...
  93. pmc Hematopoietic stem cell expansion and distinct myeloid developmental abnormalities in a murine model of the AML1-ETO translocation
    Cristina G de Guzman
    Department of Human Genetics, University of Alabama at Birmingham, 1824 6th Avenue South, Birmingham, AL 35294, USA
    Mol Cell Biol 22:5506-17. 2002
    ..This suggests that the principal contribution of AML1-ETO to acute myeloid leukemia is the inhibition of multiple developmental pathways...
  94. ncbi Modified adenoviral vectors ablated for coxsackievirus-adenovirus receptor, alphav integrin, and heparan sulfate binding reduce in vivo tissue transduction and toxicity
    Naoya Koizumi
    National Institute of Biomedical Innovation, Osaka 567 0085, Japan
    Hum Gene Ther 17:264-79. 2006
    ..We therefore succeeded in further improving the mutant Ad vector, abolishing both viral natural tropism and toxicity. This new Ad vector appears to be a fundamental vector for targeted gene delivery...
  95. ncbi Liver-targeted gene therapy by SV40-based vectors using the hydrodynamic injection method
    Uri Arad
    Department of Hematology, Hebrew University Hadassah Medical School and Hadassah Hospital, Jerusalem 91120, Israel
    Hum Gene Ther 16:361-71. 2005
    ..Vectors packaged in vitro, using recombinant capsid proteins and plasmid DNA, were also effective in liver transduction. These results suggest that SV40 vectors may be useful for liver gene therapy...
  96. ncbi Mesenchymal stem cells effectively deliver an oncolytic adenovirus to intracranial glioma
    Adam M Sonabend
    Brain Tumor Center, University of Chicago, Chicago, Illinois, USA
    Stem Cells 26:831-41. 2008
    ..Taken together, these results indicate that hMSC migrate and deliver CRAd to distant glioma cells. This delivery strategy should be explored further, as it could improve the outcome of oncolytic virotherapy for glioma...
  97. pmc In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning
    Steven P Zielske
    Molecular Virology Program, Case Western Reserve University, Cleveland, Ohio 44106, USA
    J Clin Invest 112:1561-70. 2003
    ..To our knowledge, these results provide the first evidence of potent in vivo selection of MGMT(P140K) lentivirus-transduced human SRCs following BG/BCNU treatment...
  98. pmc Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors
    Clare E Thomas
    Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA
    J Virol 78:3110-22. 2004
    ..Our data suggest that the rate of uncoating of vector genomes determines the ability of complementary plus and minus single-stranded genomes to anneal together and convert to stable, biologically active double-stranded molecular forms...
  99. pmc Heterologous human immunodeficiency virus type 1 lentiviral vectors packaging a simian immunodeficiency virus-derived genome display a specific postentry transduction defect in dendritic cells
    Caroline Goujon
    INSERM U412, Ecole Normale Supérieure de Lyon Etablissement Français du Sang, Lyon, France
    J Virol 77:9295-304. 2003
  100. pmc Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors
    Mariacarmela Allocca
    Telethon Institute of Genetics and Medicine, Via P Castellino 111, 80131 Napoli, Italy
    J Virol 81:11372-80. 2007
  101. ncbi Generation of tumor-specific, HLA class I-restricted human Th1 and Tc1 cells by cell engineering with tumor peptide-specific T-cell receptor genes
    Takemasa Tsuji
    Division of Immunoregulation, Institute for Genetic Medicine, Hokkaido University, Sapporo 060 0815, Japan
    Blood 106:470-6. 2005
    ..Thus, TCR gene-modified HLA-class I-restricted Th1 and Tc1 cells are a powerful strategy for the application to adoptive immunotherapy of human cancer...

Research Grants67

  1. Chimeric adenoviruses for an improved HIV vaccine
    Qiana L Matthews; Fiscal Year: 2013
    ..One particularly promising approach is based upon genetic transduction via replication-incompetent adenoviral vectors (Ad)...
  2. A novel protein transduction method to generate induced pluripotent stem cells wi
    JAMES FREDERICK ELIASON; Fiscal Year: 2010
    ..focused on delivery of four genes (Oct4, Sox2, and either Klf4 plus c-Myc, or NANOG plus Lin 28) using genetic transduction methods that potentially can create mutations and/or tumors...
  3. TCR Transduction for EBV Specific Immunotherapy
    Rimas Orentas; Fiscal Year: 2004
    ..The specific aims of this project seek to determine which TCRs are the best candidates for genetic transduction by comparing the CTL activity of the original cell to the lytic activity newly conferred upon the ..
  4. Novel dendritic cell therapy for human cancers
    Selvarangan Ponnazhagan; Fiscal Year: 2005
    ..b>Genetic transduction of DCs represents another method of tumor antigen presentation with potential advantages over pulsing...
  5. Regulation of Cell survival Following T Cell Recognition
    Alfred Bothwell; Fiscal Year: 2007
    ..of human umbilical vein endothelial cells (HUVEC) from human cytotoxic T lymphocytes (huCTL) by genetic transduction of the anti-apoptotic caspase-resistant Bcl-2 gene...
    Douglas Smith; Fiscal Year: 1999
    ..that continuous single-pass medium exchange will enable enhanced T- cell production, function, and genetic transduction using flow- through technology in the AastromReplicell TM Cell Production System will be tested...
    Stephanos Kyrkanides; Fiscal Year: 2004
    ..abstract_text> ..
  8. Recombinant FIV vectors for the delivery of siRNA therapy to joints
    Stephanos Kyrkanides; Fiscal Year: 2007
    ..unreadable] [unreadable] [unreadable]..
  9. Does peripheral localized chronic inflammation predispose to neurodegeneration?
    Stephanos Kyrkanides; Fiscal Year: 2007
    ..unreadable] [unreadable] [unreadable]..
  10. Joint degeneration: Somatic mosaic analysis in a transgenic mouse
    Stephanos Kyrkanides; Fiscal Year: 2007
    ..unreadable] [unreadable] [unreadable]..
  11. Gene therapy for treatment of craniofacial dysplasia
    Stephanos Kyrkanides; Fiscal Year: 2004
    ..Completion of this study will aid in the development of novel therapies for the management of genetic disorders. ..
  12. Adenoviral vectors to target cells
    Patricia Yotnda; Fiscal Year: 2006
    ..At the conclusion of this study we will have in place an approach that could be implemented to allow truly targeted delivery of therapeutic vectors to disseminated malignancies. ..
  13. Hematopoietic Potential of Muscle-Derived Cells
    Hans Peter Kiem; Fiscal Year: 2004
    ..abstract_text> ..
  14. Center for Fetal Gene Transfer for HLB Diseases
    Alice Tarantal; Fiscal Year: 2005
    ..abstract_text> ..
  15. Delivery of Therapeutic Genes to Osteoarthritic Joints
    Christopher Evans; Fiscal Year: 2002
  16. Lanthanides as Novel Anti-Arthritic Agents
    Christopher Evans; Fiscal Year: 2004
    ..A successful outcome to these studies will have identified the basis for a potential new, very inexpensive anti-rheumatic agent whose mode of action differs from that of existing drugs. ..
    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. ..
  18. 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]..
  19. Mesenchymal Stem Cells for Fetal Gene Delivery
    Alice Tarantal; Fiscal Year: 2006
    ..abstract_text> ..
  20. MGMT-Mediated in vivo Selection in a Large Animal Model
    Hans Peter Kiem; Fiscal Year: 2006
    ..These experiments will provide a platform for studies using gene-modified allogeneic stem cells to improve nonmyeloablative transplant strategies. ..
  21. Novel Nanostructured Calcium Phosphate for Gene Delivery to Bone
    Christopher Evans; Fiscal Year: 2005
    ..2. To evaluate the ability of nCaP-vector constructs to enhance osteogenic responses in vivo. ..
  22. AAOS/NIH Mulit-Year Research Symposia for 2005-2009
    Christopher Evans; Fiscal Year: 2005
  23. Gene therapy for Fanconi anemia
    Hans Peter Kiem; Fiscal Year: 2009
    ..Our ultimate goal is to improve or cure the hematopoietic symptoms in FA patients by lentivirus-mediated HSC gene transfer, and develop methodologies that would be applicable to other genetic disorders and hematological diseases. ..
  24. Center for Pediatric Stem/Progenitor Cell Translational Research
    Alice Tarantal; Fiscal Year: 2008
    ..If imaging techniques can be developed that allows one to monitor the cells over time, this would provide a very powerful tool to determine the fate of the cells after injection into humans. ..
  25. Cord Blood Expansion and Transplantation in a Nonhuman Primate Model
    Hans Peter Kiem; Fiscal Year: 2009
  26. Lineage decision in blood stem and progenitor cells
    Koichi Akashi; Fiscal Year: 2007
    ..Thus, we hope these studies will help understand mechanistic effects of these transcription factors on lineage decision in normal and malignant hematopoietic development. ..
  27. Homer proteins, glutamate and alcoholism
    KAREN SZUMLINSKI; Fiscal Year: 2007
    ..unreadable] [unreadable] [unreadable]..
  28. BK Virus as a Co-Factor in Prostate Cancer
    Michael Imperiale; Fiscal Year: 2009
    ..If a role for BKV in prostate cancer exists, there is the possibility of developing therapeutics or vaccines that are specific for the virus, thereby reducing the morbidity, mortality, and even the incidence of this cancer. ..
  29. Recombinant antibodies as an anti-microbial shield
    Alexander Pereboev; Fiscal Year: 2004
    ..To test the concept of a gene-therapy approach of the recAb anti-microbial defense in animal model. ..
  30. Stem Cell Adaptability in Parkinson' Disease
    DAVID YUREK; Fiscal Year: 2009
    ..The variable of host age on ESC-derived dopamine neuron adaptability will be studied. ..
  31. Synaptic Targeting of NMDA Receptors
    Michael Ehlers; Fiscal Year: 2009
    ..Moreover, because NMDA receptors participate in the pathogenesis of a wide range of neurologic disorders, psychiatric disease, and states of addiction, these studies hold promise for the development of novel therapeutic strategies. ..
    David Emery; Fiscal Year: 2008
    ..abstract_text> ..
  33. High Throughput Chemical Genomics to Identify Novel Inhibitors of CHOP
    Randal Kaufman; Fiscal Year: 2008
    ..unreadable] [unreadable] [unreadable]..
  34. Unfolded proteins in the Endoplasmic Reticulum to Disease
    Randal Kaufman; Fiscal Year: 2007
    ..unreadable] [unreadable] [unreadable]..
  35. Activating PTPN11 and c-kit Mutations in Myeloproliferative Disorder
    Rebecca Chan; Fiscal Year: 2008
  36. Angiogenesis and Cardiac Growth as Heart Failure Therapy
    Y Woo; Fiscal Year: 2008
    ..The candidate's institution, sponsor, and chairman provide a uniquely supportive, high-caliber, educationally stimulating environment highly conducive to the achievement of these goals. ..
  37. High Gulcose Promotes Myocyte Apoptosis by PKC Pathways
    Ashwani Malhotra; Fiscal Year: 2008
    ..The proposed investigations are novel and fundamental to the development of gene based therapy to inhibit myocyte apoptosis in the diabetic heart. ..
    Arthur Bank; Fiscal Year: 2004
    ..abstract_text> ..
  39. Gene Therapy, Neural Grafts & Parkinson's Disease
    DAVID YUREK; Fiscal Year: 2005
    ..These studies will also determine if combinations of viral vectors expressing different neurotrophic factors can be used to improve the therapeutic effects of dopamine grafts. ..
  40. Forkhead Transcription Factors in Stem Cell Development
    Saghi Ghaffari; Fiscal Year: 2005
    ..abstract_text> ..
    W Stamer; Fiscal Year: 2004
    ..abstract_text> ..
  42. Cytokine-modified stem cells in kidney transplantation
    Sifeng Chen; Fiscal Year: 2006
    ..abstract_text> ..
  43. Gene transfer to the kidney using lentiviral vectors
    Frank Park; Fiscal Year: 2004
    ..abstract_text> ..
  44. In Vivo BM Stem Cell Gene Transfer for MPS type I
    Dao Pan; Fiscal Year: 2006
    ..Taken together, these data would not only open a door to a novel approach for treatment of human diseases, but also provide a new tool to study adult stem cell plasticity and the nature of hematopoiesis. ..
  45. Recombinant Adenovirus Vaccines Against B. anthracis
    Michael Imperiale; Fiscal Year: 2006
    ..anthracis vaccine, and will establish a platform for similar vaccines against other infectious agents that might be used by bioterrorists. ..
  46. Novel Gene Therapy using Helper-Dependent Adenovirus
    Kazuhiro Oka; Fiscal Year: 2006
    ..abstract_text> ..
  47. Gene Therapy for Correction of PiZ Mutation
    Sihong Song; Fiscal Year: 2006
    ..Liver pathology will be examined. We anticipate achieving the therapeutic correction in PiZ transgenic mouse model. The success of this project may lead to a novel gene therapy for clinical treatment of human AAT deficiency. ..
  48. Evaluation of Hypoxia in Grafted Dopamine Neurons
    Caryl Sortwell; Fiscal Year: 2005
    ..abstract_text> ..
    Arthur Bank; Fiscal Year: 2002
    Michael Imperiale; Fiscal Year: 2001
    ..Third, the reason for expression of the 52/55 kDa protein during the early phase of the infection will be examined. Together, these studies address the how and why of regulation of expression of L1 mRNAs during infection. ..
  51. AIF-1 Expression In VSMC Growth And Arteriopathy
    Michael Autieri; Fiscal Year: 2009
    ..Characterization of AIF-1 function will clarify our understanding of inflammation-mediated signal transduction leading to VSMC pathobiology and vascular- immune cell cross talk. ..
    W Daniel Stamer; Fiscal Year: 2010
    ..Results obtained from these investigations will provide a basic understanding of the role of cadherin proteins in aqueous outflow resistance and uncover novel therapeutic targets for glaucoma therapy. ..
  53. Developmental Checkpoints of Murine Mast Cells
    Koichi Akashi; Fiscal Year: 2009
    ..Results obtained from this project will be critical to study mast cell biology, which eventually will help develop new therapeutic strategies for a variety of allergic and autoimmune disorders. ..
  54. Nitric Oxide Regulation of Adrenal Steroidogenesis
    William Campbell; Fiscal Year: 2004
    ..4) We will determine the role of endothelial NO in regulating steroidogenesis and adrenal blood flow in the in situ perfused adrenal gland and in vivo in anesthetized rats. ..
    Jeffrey Schechner; Fiscal Year: 2004
    ..abstract_text> ..
  56. Ex Vivo Expansion of Cord Blood Progenitor Cells
    Colleen Delaney; Fiscal Year: 2008
    ..abstract_text> ..
    Ratna Ray; Fiscal Year: 2006
    ..To investigate the mechanism by which it induces apoptosis To test the biological effect of MBP-1 disruption in KO mice To determine whether adenovirus-mediated MBP-1 gene transfer can regress tumor growth in vivo. ..
  58. Adenoviral GFP targeting of metastatic human tumors using multiple delivery route
    Hiroyuki Kishimoto; Fiscal Year: 2008
    ..Having obtained these results, we can move forward in the Phase II grant application with experiments to bring OBP-401 to the clinic for use in cancer surgery in human patients. [unreadable] [unreadable] [unreadable]..
  59. Unfolded Protein Response in Glucose Homeostasis
    Randal Kaufman; Fiscal Year: 2006
    ..These in vivo models have the potential for providing novel biologic insights into human disease and may also facilitate testing of new therapies. [unreadable] [unreadable] [unreadable]..
    Howard Kaufman; Fiscal Year: 2004
    ..This may have important implications for the future design of tumor vaccines in melanoma and other settings. ..
  61. Metalloproteinase regulation of ErbB signaling in skin
    Stefan Stoll; Fiscal Year: 2006
    ..Specific Aim 2 determines whether and in which contexts ADAM10 is involved in the shedding of ErbB ligands in human KCs. ..
  62. Gene Therapy of Autosomal Recessive RP
    Simon Petersen Jones; Fiscal Year: 2006
    ..abstract_text> ..
  63. Mechanism of photoreceptor cell degeneration in animal models of retinal diseases
    Muna Naash; Fiscal Year: 2008
    ..cone OSs morphogenesis and maintenance. Experiments put forth in this application will provide biochemical and physiological evidence for the role of Rds and its associated proteins during genesis of rod and cone OSs. ..
    ..abstract_text> ..
  65. Efficacy and Safety of Foamy Retroviral Vectors for AIDS Gene Therapy
    Grant Trobridge; Fiscal Year: 2007
    ..unreadable] [unreadable] [unreadable]..
  66. The ER Stress Response and ATF6 Activation in the Heart
    ..The ERSR has gone virtually unstudied in the heart; accordingly, the proposed studies are novel and will enhance our understanding of the ERSR and the roles played by ATF6 alpha and beta in the stressed myocardium. ..