Genomes and Genes
Summary: DNA molecules capable of autonomous replication within a host cell and into which other DNA sequences can be inserted and thus amplified. Many are derived from PLASMIDS; BACTERIOPHAGES; or VIRUSES. They are used for transporting foreign genes into recipient cells. Genetic vectors possess a functional replicator site and contain GENETIC MARKERS to facilitate their selective recognition.
Publications292 found, 100 shown here
- Enzymatic assembly of DNA molecules up to several hundred kilobasesDaniel G Gibson
The J Craig Venter Institute, Synthetic Biology Group, Rockville, Maryland, USA
Nat Methods 6:343-5. 2009..This assembly method can be used to seamlessly construct synthetic and natural genes, genetic pathways and entire genomes, and could be a useful molecular engineering tool...
- A gateway cloning vector set for high-throughput functional analysis of genes in plantaMark D Curtis
Institute of Plant Biology and Zurich Basel Plant Science Centre, University of Zurich, Zollikerstrasse 107, Ch 8008 Zurich, Switzerland
Plant Physiol 133:462-9. 2003..All vectors were derived from pCambia T-DNA cloning vectors, with the exception of a chemically inducible vector, for Agrobacterium sp.-mediated transformation of a wide range of plant species...
- Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vectorAndrea L Szymczak
Nat Biotechnol 22:589-94. 2004....
- Human induced pluripotent stem cells free of vector and transgene sequencesJunying 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...
- Adenovirus-associated virus vector-mediated gene transfer in hemophilia BAmit C Nathwani
Department of Haematology, University College London Cancer Institute, London, United Kingdom
N Engl J Med 365:2357-65. 2011..Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder...
- A simplified system for generating recombinant adenovirusesT C He
The Howard Hughes Medical Institute, 424 North Bond Street, Baltimore, MD 21231, USA
Proc Natl Acad Sci U S A 95:2509-14. 1998..Homogeneous viruses can be obtained from this procedure without plaque purification. This system should expedite the process of generating and testing recombinant adenoviruses for a variety of purposes...
- Safety and efficacy of gene transfer for Leber's congenital amaurosisAlbert M Maguire
Scheie Eye Institute, University of Pennsylvania, USA
N Engl J Med 358:2240-8. 2008..Although the follow-up was very short and normal vision was not achieved, this study provides the basis for further gene therapy studies in patients with LCA...
- A variant of yellow fluorescent protein with fast and efficient maturation for cell-biological applicationsTakeharu Nagai
Laboratory for Cell Function and Dynamics, Advanced Technology Development Center, Brain Science Institute, RIKEN, 2 1 Hirosawa, Wako City, Saitama, 351 0198, Japan
Nat Biotechnol 20:87-90. 2002..With the improved speed and efficiency of maturation and the increased resistance to environment, Venus will enable fluorescent labelings that were not possible before...
- A lentiviral RNAi library for human and mouse genes applied to an arrayed viral high-content screenJason Moffat
Broad Institute of MIT and Harvard, Cambridge, MA 02139, USA
Cell 124:1283-98. 2006..This work provides a widely applicable resource for loss-of-function screens, as well as a roadmap for its application to biological discovery...
- Gateway-compatible vectors for plant functional genomics and proteomicsKeith W Earley
Biology Department, Washington University, 1 Brookings Drive, St Louis, MO 63130, USA
Plant J 45:616-29. 2006..Antibodies detecting the FLAG, HA, cMyc and AcV5 tags show relatively little cross-reaction with endogenous proteins in a variety of monocotyledonous and dicotyledonous plants, suggesting broad utility for the tags and vectors...
- P[acman]: a BAC transgenic platform for targeted insertion of large DNA fragments in D. melanogasterKoen 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...
- A third-generation lentivirus vector with a conditional packaging systemT Dull
Cell Genesys, Foster City, California 94404, USA
J Virol 72:8463-71. 1998..While the actual biosafety of the vector will ultimately be proven in vivo, the improved design presented here should facilitate testing of lentivirus vectors...
- A one pot, one step, precision cloning method with high throughput capabilityCarola Engler
Icon Genetics GmbH, Biozentrum Halle, Halle, Germany
PLoS ONE 3:e3647. 2008....
- Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectorsCarlos 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...
- LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1S Hacein-Bey-Abina
INSERM Unit 429, Cedex 15, France
Science 302:415-9. 2003..Thus, retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter...
- Tight regulation, modulation, and high-level expression by vectors containing the arabinose PBAD promoterL M Guzman
Department of Microbiology and Molecular Genetics, Harvard Medical School, Boston, Massachusetts 02115, USA
J Bacteriol 177:4121-30. 1995..We have exploited the tight regulation of the PBAD promoter to study the phenotypes of null mutations of essential genes and explored the use of pBAD vectors as an expression system...
- Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophyNathalie 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...
- Filamentous fusion phage: novel expression vectors that display cloned antigens on the virion surfaceG P Smith
Science 228:1315-7. 1985..Fusion phage may provide a simple way of cloning a gene when an antibody against the product of that gene is available...
- Generation of mouse induced pluripotent stem cells without viral vectorsKeisuke Okita
Center for iPS Cell Research and Application CiRA, Institute for Integrated Cell Material Sciences, Kyoto University, Kyoto 606 8507, Japan
Science 322:949-53. 2008..The production of virus-free iPS cells, albeit from embryonic fibroblasts, addresses a critical safety concern for potential use of iPS cells in regenerative medicine...
- Inducible gene targeting in miceR Kuhn
Institute for Genetics, University of Cologne, Germany
Science 269:1427-9. 1995..Deletion was complete in liver and nearly complete in lymphocytes within a few days, whereas partial deletion was obtained in other tissues. This method can be used for the inducible inactivation of any other gene in vivo...
- Efficient transposition of the piggyBac (PB) transposon in mammalian cells and miceSheng Ding
Institute of Developmental Biology and Molecular Medicine, School of Life Sciences, Fudan University, 220 Handan Road, Shanghai 200433, China
Cell 122:473-83. 2005..These data provide a first and critical step toward a highly efficient transposon system for a variety of genetic manipulations including transgenesis and insertional mutagenesis in mice and other vertebrates...
- Induced pluripotent stem cells generated without viral integrationMatthias 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...
- Virus-induced gene silencing in tomatoYule Liu
Department of Molecular, Cellular and Developmental Biology, OML 451, Yale University, PO Box 208104, New Haven, CT 06520 8104, USA
Plant J 31:777-86. 2002..Using this vector, we have silenced RbcS and an endogenous gene homologous to the tomato EST cLED3L14. In the future, this modified vector system will facilitate large-scale functional analysis of tomato ESTs...
- Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemiaMarina Cavazzana-Calvo
Clinical Investigation Center in Biotherapy, Groupe Hospitalier Universitaire Ouest, Inserm Assistance Publique Hôpitaux de Paris, Paris 75015, France
Nature 467:318-22. 2010..The clonal dominance that accompanies therapeutic efficacy may be coincidental and stochastic or result from a hitherto benign cell expansion caused by dysregulation of the HMGA2 gene in stem/progenitor cells...
- Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytesKevin 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...
- Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune responseCatherine S Manno
The Children s Hospital of Philadelphia, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania, 19104, USA
Nat Med 12:342-7. 2006..We conclude that rAAV-2 vectors can transduce human hepatocytes in vivo to result in therapeutically relevant levels of F.IX, but that future studies in humans may require immunomodulation to achieve long-term expression...
- GATEWAY vectors for Agrobacterium-mediated plant transformationMansour Karimi
Dept of Plant Systems Biology, Flanders Interuniversity Institute for Biotechnology VIB, Ghent University, K L Ledeganckstraat 35, B 9000, Gent, Belgium
Trends Plant Sci 7:193-5. 2002..Because these vectors are generally large, cloning can be time-consuming and laborious. Recently, the GATEWAY conversion technology has provided a fast and reliable alternative to the cloning of sequences into large acceptor plasmids...
- DNA cloning using in vitro site-specific recombinationJ L Hartley
Life Technologies, Inc, Rockville, Maryland 20850, USA
Genome Res 10:1788-95. 2000..In this paper, we outline the concepts of this approach and provide several examples that highlight some of its potential...
- Golden gate shuffling: a one-pot DNA shuffling method based on type IIs restriction enzymesCarola Engler
Icon Genetics GmbH, Biozentrum Halle, Halle, Germany
PLoS ONE 4:e5553. 2009....
- Induction of immunity to human immunodeficiency virus type-1 by vaccinationM Juliana McElrath
Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue N, D3 100, Seattle, WA 98109, USA
Immunity 33:542-54. 2010....
- A system for stable expression of short interfering RNAs in mammalian cellsThijn R Brummelkamp
Division of Molecular Carcinogenesis, Division of Tumor Biology, The Netherlands Cancer Institute, Plesmanlaan 121, 1066 CX Amsterdam, Netherlands
Science 296:550-3. 2002..Therefore, the pSUPER vector constitutes a new and powerful system to analyze gene function in a variety of mammalian cell types...
- Phase Ia clinical evaluation of the safety and immunogenicity of the Plasmodium falciparum blood-stage antigen AMA1 in ChAd63 and MVA vaccine vectorsSusanne H Sheehy
Centre for Clinical Vaccinology and Tropical Medicine, Churchill Hospital, Oxford, United Kingdom
PLoS ONE 7:e31208. 2012..falciparum. The development of a vaccine that could induce both cell-mediated and humoral immune responses would enable important proof-of-concept efficacy studies to be undertaken to address this question...
- A time- and cost-efficient system for high-level protein production in mammalian cellsA Radu Aricescu
Cancer Research UK Receptor Structure Research Group, Division of Structural Biology, Wellcome Trust Centre for Human Genetics, University of Oxford, Oxford, England
Acta Crystallogr D Biol Crystallogr 62:1243-50. 2006..The system is suitable for use in conventional laboratories or can be implemented in a medium- or high-throughput pipeline...
- Lentiviral vectors: basic to translationalToshie Sakuma
Department of Molecular Medicine, Mayo Clinic College of Medicine, 200 First Street SW, Rochester, MN 55905, USA
Biochem J 443:603-18. 2012....
- Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challengesFederico Mingozzi
Center for Cellular and Molecular Therapeutics, The Children s Hospital of Philadelphia, 3501 Civic Center Boulevard, 5th Floor CTRB, Philadelphia, Pennsylvania 19104, USA
Nat Rev Genet 12:341-55. 2011..Recent exciting results have raised hopes for the treatment of many other diseases. As we discuss here, the prospects and challenges for AAV gene therapy are to a large extent dependent on the target tissue and the specific disease...
- Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector deliveryAngelo Lombardo
San Raffaele Telethon Institute for Gene Therapy, Via Olgettina, 58, 20132 Milan, Italy
Nat Biotechnol 25:1298-306. 2007....
- A nonviral minicircle vector for deriving human iPS cellsFangjun Jia
Departments of Medicine and Radiology, Stanford University School of Medicine, Stanford, California, USA
Nat Methods 7:197-9. 2010..Here we use a single minicircle vector to generate transgene-free iPSCs from adult human adipose stem cells...
- Sequential expression of pluripotency markers during direct reprogramming of mouse somatic cellsTobias Brambrink
Whitehead Institute for Biomedical Research, Massachusetts Institute of Technology, 9 Cambridge Center, Cambridge, MA 02142, USA
Cell Stem Cell 2:151-9. 2008..Importantly, the virally transduced cDNAs needed to be expressed for at least 12 days in order to generate iPS cells. Our results are a step toward understanding some of the molecular events governing epigenetic reprogramming...
- Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 yearsSamuel G Jacobson
Scheie Eye Institute, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, USA
Arch Ophthalmol 130:9-24. 2012..To determine the safety and efficacy of subretinal gene therapy in the RPE65 form of Leber congenital amaurosis using recombinant adeno-associated virus 2 (rAAV2) carrying the RPE65 gene...
- Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapyGuang Ping Gao
Institute for Human Gene Therapy and Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA
Proc Natl Acad Sci U S A 99:11854-9. 2002....
- Potent CD8+ T-cell immunogenicity in humans of a novel heterosubtypic influenza A vaccine, MVA-NP+M1Tamara K Berthoud
The Jenner Institute, Oxford University, Oxford, United Kingdom
Clin Infect Dis 52:1-7. 2011..A vaccine providing protective immunity to the highly conserved internal antigens could provide longer-lasting protection against multiple influenza subtypes...
- Generation of transgene-free induced pluripotent mouse stem cells by the piggyBac transposonKosuke Yusa
Wellcome Trust Sanger Institute, Wellcome Trust Genome Campus, Hinxton, Cambridge, UK
Nat Methods 6:363-9. 2009..iPSCs fulfilled all criteria of pluripotency, such as pluripotency gene expression, teratoma formation and contribution to chimeras. piggyBac transposon-based reprogramming may be used to generate therapeutically applicable iPSCs...
- A phage integrase directs efficient site-specific integration in human cellsA C Groth
Department of Genetics, Stanford University School of Medicine, Stanford, CA 94305 5120, USA
Proc Natl Acad Sci U S A 97:5995-6000. 2000..These observations form the basis for site-specific integration strategies potentially useful in a broad range of genetic engineering applications...
- Hexon-chimaeric adenovirus serotype 5 vectors circumvent pre-existing anti-vector immunityDiane M Roberts
Division of Viral Pathogenesis, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts 02215, USA
Nature 441:239-43. 2006..Such chimaeric viral vectors may have important practical implications for vaccination and gene therapy...
- Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous systemCorinna Burger
Department of Molecular Genetics and Microbiology, University of Florida, Gainesville 32610, USA
Mol Ther 10:302-17. 2004..Retrograde transport of rAAV1 and rAAV5 was also observed in particular CNS areas. These results suggest that vectors based on distinct AAV serotypes can be chosen for specific applications in the nervous system...
- Viral vectors as vaccine platforms: deployment in sightChristine S Rollier
Oxford Vaccine Group, Department of Paediatrics, Center for Clinical Vaccine and Tropical Medicine, University of Oxford, Churchill Hospital, Churchill Drive, Oxford OX3 7LJ, United Kingdom
Curr Opin Immunol 23:377-82. 2011....
- A versatile viral system for expression and depletion of proteins in mammalian cellsEric Campeau
Program in Gene Function and Expression, University of Massachusetts Medical School, Worcester, Massachusetts, United States of America
PLoS ONE 4:e6529. 2009..As new technologies emerge, the vectors in this collection can be easily modified and adapted without the need for extensive recloning...
- Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trialWilliam W Hauswirth
Department of Ophthalmology, University of Florida, Gainesville, FL 32610, USA
Hum Gene Ther 19:979-90. 2008..99). Comparisons are drawn between the present work and two other studies of ocular gene therapy for RPE65-LCA that were carried out contemporaneously and reported...
- A suite of Gateway cloning vectors for high-throughput genetic analysis in Saccharomyces cerevisiaeSimon Alberti
Whitehead Institute for Biomedical Research, Cambridge, MA, USA
Yeast 24:913-9. 2007..We have made this yeast Gateway vector kit available to the research community via the non-profit Addgene Plasmid Repository (http://www.addgene.org/yeast_gateway)...
- A versatile ligation-independent cloning method suitable for high-throughput expression screening applicationsNick S Berrow
The Oxford Protein Production Facility, Henry Wellcome Building for Genomic Medicine, University of Oxford, Oxford, UK
Nucleic Acids Res 35:e45. 2007..g. Sf9 cells). These high-throughput screening activities have prompted the development and validation of automated protocols for transfection of mammalian cells and Ni-NTA protein purification...
- MicroRNA sponges: competitive inhibitors of small RNAs in mammalian cellsMargaret S Ebert
Center for Cancer Research, E17 529B Massachusetts Institute of Technology, Cambridge, Massachusetts 02139, USA
Nat Methods 4:721-6. 2007..RNA polymerase II promoter (Pol II)-driven sponges contain a fluorescence reporter gene for identification and sorting of sponge-treated cells. We envision the use of stably expressed sponges in animal models of disease and development...
- Retrovirus-mediated gene transfer and expression cloning: powerful tools in functional genomicsToshio Kitamura
Divisions of Cellular Therapy and Hematopoietic Factors, The Institute of Medical Science, The University of Tokyo, Tokyo, Japan
Exp Hematol 31:1007-14. 2003..In this review, we describe retrovirus-mediated strategies used for investigation of gene functions and function-based screening strategies...
- 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 promoterChristophe Demaison
Molecular Immunology Unit, Institute of Child Health, UCL, 30 Guilford Street, London, WC1N 1EH, UK
Hum Gene Ther 13:803-13. 2002....
- Design and development of polymers for gene deliveryDaniel W Pack
Department of Chemical and Biomolecular Engineering, University of Illinois, Box C 3, 600 South Mathews Avenue, Urbana, IL 61801, USA
Nat Rev Drug Discov 4:581-93. 2005..With the growing understanding of polymer gene-delivery mechanisms and continued efforts of creative polymer chemists, it is likely that polymer-based gene-delivery systems will become an important tool for human gene therapy...
- Profound early control of highly pathogenic SIV by an effector memory T-cell vaccineScott G Hansen
Vaccine and Gene Therapy Institute, Department of Molecular Microbiology, Oregon Health and Science University, Beaverton, Oregon 97006, USA
Nature 473:523-7. 2011..Thus, persistent vectors such as CMV and their associated T(EM) responses might significantly contribute to an efficacious HIV/AIDS vaccine...
- Avoiding cytotoxicity of transposases by dose-controlled mRNA deliveryMelanie Galla
Department of Experimental Hematology, Institute of Transplantation Immunology, Institute of Cell and Molecular Pathology, Hannover Medical School, Hannover, Germany
Nucleic Acids Res 39:7147-60. 2011..Transposase-mediated gene transfer can thus be tuned to maintain high efficiency in the absence of overt cell damage...
- Progress and problems with the use of viral vectors for gene therapyClare E Thomas
Department of Pediatrics, Stanford University School of Medicine, Stanford, California 94305, USA
Nat Rev Genet 4:346-58. 2003..With the development of a leukaemia-like syndrome in two of the small number of patients that have been cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go...
- Many paths to many clones: a comparative look at high-throughput cloning methodsGerald Marsischky
Institute of Proteomics, Harvard Medical School, Department of Biological Chemistry and Molecular Pharmacology, Boston, Massachusetts 02115, USA
Genome Res 14:2020-8. 2004..Alternative approaches to creating clones involving mixing of cloning methods, including gap-repair cloning, are also explored...
- Comparative genomic integration profiling of Sleeping Beauty transposons mobilized with high efficacy from integrase-defective lentiviral vectors in primary human cellsBrian 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...
- Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower dosesLi Zhong
Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL 32610, USA
Proc Natl Acad Sci U S A 105:7827-32. 2008..These studies have led to the development of AAV vectors that are capable of high-efficiency transduction at lower doses, which has important implications in their use in human gene therapy...
- Effect of gene therapy on visual function in Leber's congenital amaurosisJames W B Bainbridge
Institute of Ophthalmology, University College London, London, United Kingdom
N Engl J Med 358:2231-9. 2008..These findings provide support for further clinical studies of this experimental approach in other patients with mutant RPE65. (ClinicalTrials.gov number, NCT00643747 [ClinicalTrials.gov].)...
- Efficient generation of transgene-free human induced pluripotent stem cells (iPSCs) by temperature-sensitive Sendai virus vectorsHiroshi Ban
DNAVEC Corporation, Tsukuba, Ibaraki 300 2611, Japan
Proc Natl Acad Sci U S A 108:14234-9. 2011..We suggest that generation of transgene-free iPSCs from cord blood cells should be an important step in providing allogeneic iPSC-derived therapy in the future...
- Induction of broadly neutralizing H1N1 influenza antibodies by vaccinationChih Jen Wei
Vaccine Research Center, National Institute of Allergy and Infectious Diseases NIAID, National Institutes of Health NIH, Bethesda, MD 20892 3005, USA
Science 329:1060-4. 2010..Cross-neutralization of H1N1 subtypes elicited by this approach provides a basis for the development of a universal influenza vaccine for humans...
- Transposon vectors containing non-antibiotic resistance selection markers for cloning and stable chromosomal insertion of foreign genes in gram-negative bacteriaM Herrero
GBF National Research Centre for Biotechnology, Braunschweig, Federal Republic of Germany
J Bacteriol 172:6557-67. 1990..g., in open systems and natural environments), and that do not carry antibiotic resistance markers characteristic of most available cloning vectors (as is currently required of live bacterial vaccines)...
- piggyBac transposition reprograms fibroblasts to induced pluripotent stem cellsKnut Woltjen
Samuel Lunenfeld Research Institute, Mount Sinai Hospital, Toronto, Ontario M5G 1X5, Canada
Nature 458:766-70. 2009..We anticipate that the unique properties of this virus-independent simplification of iPS cell production will accelerate this field further towards full exploration of the reprogramming process and future cell-based therapies...
- Innate immune sensing of modified vaccinia virus Ankara (MVA) is mediated by TLR2-TLR6, MDA-5 and the NALP3 inflammasomeJulie Delaloye
Department of Medicine, Infectious Diseases Service, Centre Hospitalier Universitaire Vaudois and University of Lausanne, Lausanne, Switzerland
PLoS Pathog 5:e1000480. 2009..Delineation of the host response induced by MVA is critical for improving our understanding of poxvirus antiviral escape mechanisms and for designing new MVA vaccine vectors with improved immunogenicity...
- Production and characterization of adeno-associated viral vectorsJoshua C Grieger
Curriculum in Genetics and Molecular Biology, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina 27599, USA
Nat Protoc 1:1412-28. 2006..This review provides an up-to-date, detailed description of essential methods such as production, purification and titering and their application to characterize current AAV vectors for preclinical and clinical use...
- A high-efficiency system for the generation and study of human induced pluripotent stem cellsNimet Maherali
Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute, Massachusetts General Hospital Center for Regenerative Medicine, Massachusetts General Hospital Cancer Center, Boston, MA 02114, USA
Cell Stem Cell 3:340-5. 2008..The ability to reprogram cells at high efficiency provides a unique platform to dissect the underlying molecular and biochemical processes that accompany nuclear reprogramming...
- Expression of heterologous proteins in Pichia pastoris: a useful experimental tool in protein engineering and productionRachel Daly
ARC Special Research Centre for Green Chemistry, Monash University, Building 23, Wellington Road, Clayton, Victoria 3800, Australia
J Mol Recognit 18:119-38. 2005..Procedures to prevent glycosylation through manipulation of cell culture conditions or via enzymatic and site-directed mutagenesis methods are also discussed...
- Refinement of tools for targeted gene expression in DrosophilaBarret 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...
- Overall survival analysis of a phase II randomized controlled trial of a Poxviral-based PSA-targeted immunotherapy in metastatic castration-resistant prostate cancerPhilip W Kantoff
Medical Director, BN ImmunoTherapeutics, 2425 Garcia Ave, Mountain View, CA 94043, USA
J Clin Oncol 28:1099-105. 2010..PROSTVAC-VF treatment was evaluated for safety and for prolongation of progression-free survival (PFS) and overall survival (OS) in a randomized, controlled, and blinded phase II study...
- Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administrationFrancesca 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...
- Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primatesLluis Samaranch
Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103, USA
Hum Gene Ther 23:382-9. 2012..Last, we have demonstrated that delivery of AAV9 into the CSF does not shield against AAV antibodies. This has obvious implications when developing and/or implementing any clinical trial studies...
- Induced pluripotent stem cell generation using a single lentiviral stem cell cassetteCesar A Sommer
Department of Medicine, Boston University School of Medicine, Massachusetts 02118, USA
Stem Cells 27:543-9. 2009..The use of a single lentiviral vector for reprogramming represents a powerful laboratory tool and a significant step toward the application of iPS technology for clinical purposes...
- Phase Ia clinical evaluation of the Plasmodium falciparum blood-stage antigen MSP1 in ChAd63 and MVA vaccine vectorsSusanne H Sheehy
Centre for Clinical Vaccinology and Tropical Medicine, The Jenner Institute, University of Oxford, Churchill Hospital, Oxford, UK
Mol Ther 19:2269-76. 2011..Further studies are required to assess whether this strategy can achieve protective efficacy against blood-stage malaria infection...
- Disruption of overlapping transcripts in the ROSA beta geo 26 gene trap strain leads to widespread expression of beta-galactosidase in mouse embryos and hematopoietic cellsB P Zambrowicz
Division of Basic Sciences, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA
Proc Natl Acad Sci U S A 94:3789-94. 1997..This third transcript potentially encodes a novel protein of at least 505 amino acids that is conserved in humans and in Caenorhabditis elegans...
- Cre reporter strains produced by targeted insertion of EYFP and ECFP into the ROSA26 locusS Srinivas
Department of Genetics and Development, Columbia University, New York, USA
BMC Dev Biol 1:4. 2001....
- New insights on adenovirus as vaccine vectorsMarcio O Lasaro
The Wistar Institute Vaccine Center, Philadelphia, Pennsylvania 19104, USA
Mol Ther 17:1333-9. 2009..In summary, although Ad vectors have seen their share of setbacks in recent years, they remain viable tools for prevention or treatment of a multitude of diseases...
- RF cloning: a restriction-free method for inserting target genes into plasmidsFusinita van den Ent
MRC Laboratory of Molecular Biology, Hills Road, Cambridge CB2 2QH, UK
J Biochem Biophys Methods 67:67-74. 2006..The absence of any alterations to the protein as well as the simplicity of both the primer design and the procedure itself makes it suitable for high-throughput expression and ideal for structural genomics...
- Analysis of mutation in human cells by using an Epstein-Barr virus shuttle systemR B DuBridge
Mol Cell Biol 7:379-87. 1987..A total of 33 of 34 lacI nonsense mutations and 26 of 27 missense mutations involve G X C to A X T transitions. These data provide support for the mutational theory of cancer...
- DNA sequence quality trimming and vector removalH H Chou
Department of Zoology and Genetics, Department of Computer Science, Iowa State University, Ames, IA 50011, USA
Bioinformatics 17:1093-104. 2001..This step is necessary before other genomic data processing stages can be carried out, such as fragment assembly or EST clustering. A specialized tool is therefore needed to solve this apparent dilemma...
- Conditional-replication, integration, excision, and retrieval plasmid-host systems for gene structure-function studies of bacteriaA Haldimann
Department of Biological Sciences, Purdue University, West Lafayette, Indiana 47907, USA
J Bacteriol 183:6384-93. 2001..These helper plasmids facilitate integration, excision ("curing"), or retrieval of the CRIM plasmids...
- Reprogramming of murine and human somatic cells using a single polycistronic vectorBryce 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...
- Adenovirus-5-vectored P. falciparum vaccine expressing CSP and AMA1. Part B: safety, immunogenicity and protective efficacy of the CSP componentCindy Tamminga
U S Military Malaria Vaccine Program, Naval Medical Research Center, Silver Spring, Maryland, United States of America
PLoS ONE 6:e25868. 2011..As adenovirus vaccine vectors induce both these responses in humans, a Phase 1/2a clinical trial was conducted to evaluate the efficacy of an adenovirus serotype 5-vectored malaria vaccine against sporozoite challenge...
- Prime-boost vectored malaria vaccines: progress and prospectsAdrian V S Hill
The Jenner Institute, University of Oxford, Oxford, UK
Hum Vaccin 6:78-83. 2010..These viral vectors now provide a major option for inclusion in a high efficacy multi-stage malaria vaccine that should achieve deployable levels of efficacy in endemic settings...
- A Tn7-based broad-range bacterial cloning and expression systemKyoung Hee Choi
Department of Microbiology, Immunology and Pathology, Colorado State University, Fort Collins, Colorado 80523, USA
Nat Methods 2:443-8. 2005..pestis. This system will thus have widespread biomedical and environmental applications, especially in environments where plasmids and antibiotic selection are not feasible, namely in plant and animal models or biofilms...
- Baculovirus as a gene delivery vector: recent understandings of molecular alterations in transduced cells and latest applicationsChi Yuan Chen
Department of Chemical Engineering, National Tsing Hua University, Hsinchu 30013, Taiwan
Biotechnol Adv 29:618-31. 2011....
- Production and purification of lentiviral vectorsGustavo Tiscornia
The Salk Institute for Biological Studies, Laboratory of Genetics, 10010 North Torrey Pines Road, La Jolla, California 92037, USA
Nat Protoc 1:241-5. 2006....
- MVA and NYVAC as vaccines against emergent infectious diseases and cancerCarmen E Gomez
Department of Molecular and Cellular Biology, Centro Nacional de Biotecnologia, CSIC, Ciudad Universitaria Cantoblanco, Madrid, Spain
Curr Gene Ther 11:189-217. 2011....
- Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectorsSylvie Boutin
Laboratoire d Immunologie, Genethon R and D, Evry Cedex, France
Hum Gene Ther 21:704-12. 2010..Better characterization of the preexisting humoral responses to the AAV capsid and cross-reactivity will allow development of new strategies to circumvent AAV acquired immune responses...
- Optogenetic interrogation of neural circuits: technology for probing mammalian brain structuresFeng Zhang
Department of Bioengineering, Stanford University, Stanford, California, USA
Nat Protoc 5:439-56. 2010..Together, these methods may help in providing circuit-level insight into the dynamics underlying complex mammalian behaviors in health and disease...
- Development of series of gateway binary vectors, pGWBs, for realizing efficient construction of fusion genes for plant transformationTsuyoshi Nakagawa
Department of Molecular and Functional Genomics, Center for Integrated Research in Science, Shimane University, 1060 Nishikawatsu, Matsue, Japan
J Biosci Bioeng 104:34-41. 2007..Moreover, tissue-specific and inducible gene expression using a promoter was also monitored with pGWBs. It is expected that, the pGWB system will serve as a powerful tool for plasmid construction in plant research...
- Effective gene therapy with nonintegrating lentiviral vectorsRafael J Yáñez-Muñoz
Molecular Immunology Unit, Institute of Child Health, University College London, 30 Guilford Street, London WC1N 1EH, UK
Nat Med 12:348-53. 2006..For therapeutic application to postmitotic tissues, this system substantially reduces the risk of insertional mutagenesis...
- Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 yearArtur V Cideciyan
Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA
Hum Gene Ther 20:999-1004. 2009..The safety and efficacy of human retinal gene transfer with rAAV2-RPE65 vector extends to at least 1 year posttreatment...
- Systemic Agrobacterium tumefaciens-mediated transfection of viral replicons for efficient transient expression in plantsSylvestre Marillonnet
Icon Genetics, Biozentrum Halle, Weinbergweg 22, D 06120 Halle Saale, Germany
Nat Biotechnol 23:718-23. 2005..tumefaciens, the high expression yield obtained with viral vectors, and the post-translational capabilities of a plant), does not require genetic modification of plants and is faster than other existing methods...
- The role of heterologous chloroplast sequence elements in transgene integration and expressionTracey 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....
- Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infectionBoris Kantor
Gene Therapy Center, University of North Carolina School of Medicine, Chapel Hill, NC 27514, USA
Proc Natl Acad Sci U S A 106:18786-91. 2009..Finally, we propose a mechanism describing the role of episomal HIV-1 forms in the viral life cycle in a SCFA-rich gut environment...
- Oncolytic (replication-competent) adenoviruses as anticancer agentsKaroly Toth
Department of Molecular Microbiology and Immunology, Saint Louis University School of Medicine, St Louis, Missouri, USA
Expert Opin Biol Ther 10:353-68. 2010..One option is genetically-engineered oncolytic adenovirus (Ad) 'vectors'. These kill cancer cells via the viral replication cycle, and amplify the anti-tumor effect by producing progeny virions able to infect neighboring tumor cells...
- Functional expression of secreted proteins from a bicistronic retroviral cassette based on foot-and-mouth disease virus 2A can be position dependentDominic G Rothwell
Cancer Research UK Department of Medical Oncology, School of Cancer and Imaging Sciences, University of Manchester, Manchester Academic Health Science Centre, Christie NHS Trust, Manchester M20 4BX, United Kingdom
Hum Gene Ther 21:1631-7. 2010..These results highlight that to achieve functional expression of secreted proteins consideration must be taken of the transgenic protein's posttranslational modification and trafficking when using 2A-based bicistronic cassettes...
- Gene therapy for immunodeficiency due to adenosine deaminase deficiencyAlessandro 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...
- Frequent endonuclease cleavage at off-target locations in vivoLisa M Petek
Department of Pediatrics, Division of Genetic Medicine, University of Washington, Seattle, Washington, USA
Mol Ther 18:983-6. 2010....
- Small Arteries as a Therapeutic Target in ADNienwen Chow; Fiscal Year: 2004..Potential products to be ultimately developed are new drugs or genetic vectors that would affect SRF/myocardin function in small arteries in AD with a goal to improve CBF regulations by ..
- NONRADIOACTIVE NUCLEIC ACID LABELING TECHNOLOGYMEAD MCCABE; Fiscal Year: 1993..b>Genetic Vectors has developed a chemical procedure for incorporation of detectable labels into nucleic acids...
- CNS Cellular Senescence/Targeting Endothelial SIPSNienwen Chow; Fiscal Year: 2004..The potential products ultimately to be developed are therapeutic drugs and genetic vectors to selectively correct dysfunctions of senescent brain endothelium in neurodegenerative disorders associated ..
- HSV-1 AMPLICON DELIVERED STABLE GENE THERAPY FOR THE CNSDavid Jacoby; Fiscal Year: 1999The development of genetic vectors that can deliver therapeutic DNA sequences to the central nervous system (CNS) in situ promises to be a powerful means of intervention in neurologic diseases, many of which have not current form of ..
- Target Assessment in Alzheimer's Neurovasculature/BBBJan Sallstrom; Fiscal Year: 2004..The potential products include new diagnostics DNA chips and brain imaging agents, therapeutic genetic vectors and new drugs to treat neurovascular AD disorder.
- Cellular physical properties in relation to cancerWilliam Craelius; Fiscal Year: 2002..to standard cytometers that will be useful for researchers screening potential anti- angiogenic drugs or genetic vectors, and for clinicians to biopsy specimens...
- Gene delivery to striated muscle by systemic AAV vectorsDwight D Koeberl; Fiscal Year: 2010..Efficacious muscle-targeted gene therapy in GSD-II will have implications for gene therapy in other muscular dystrophies and myopathies. ..
- INHIBITOR FORMATION IN GENE THERAPY FOR HEMOPHILIAKatherine High; Fiscal Year: 2001..These studies will involve a collaboration between the P.I.'s lab and the laboratory of Dr. Hildegund Ertl, an immunologist with experience in characterizing immune responses in the setting of viral vectors. ..
- Gene Therapy for hemophilia using muscle-expressed FVIIaKatherine A High; Fiscal Year: 2011..The proposed work will allow us to increase the therapeutic applicability of our approach and enhance its safety, prior to a clinical application. ..
- Mechanisms for immune tolerance in Pompe DiseaseDwight D Koeberl; Fiscal Year: 2010..These comparisons will guide preclinical experiments to further immunomodulatory gene therapy in Pompe disease and other lysosomal storage disorders. ..
- Nanoparticle & AAV Approaches to Rapid Onset, Stable Retinal Gene TherapyJijing Pang; Fiscal Year: 2008..Our ultimate goal is to establish a retinal gene delivery system that is fast, of long duration and can accommodate large therapeutic cDNAs. [unreadable] [unreadable] [unreadable]..
- REGULATION OF B LYMPHOMA GROWTH AND APOPTOSISDavid Scott; Fiscal Year: 2001..The results of these studies will provide information on regulation of neoplastic growth as well as mechanism for inducing apoptosis in a model for deletional tolerance. ..
- Development of an VEE Replicon Vaccine Against SmallpoxKurt Kamrud; Fiscal Year: 2005....
- MECHANISMS OF IMMUNOLOGIC TOLERANCE AND ITS BREAKDOWNDavid Scott; Fiscal Year: 2003..abstract_text> ..
- Mechanism of B-cell Delivered Tolerance in DiabetesDavid Scott; Fiscal Year: 2009..Our goal is to modulate pathogenic responses to GAD, for example, as a prelude to applying this protocol as a complementary ..
- Directed Evolution of Adeno-Associated Virus Vectors for Seizure Gene TherapyThomas 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]..
- NUCLEOTIDE DEPRIVATION-BASED SELECTIVE THERAPEUTICSRICHARD MORAN; Fiscal Year: 2008..These studies are a logical extension of past studies supported by this grant, but also represent new directions to therapeutics suggested by recent advances in cancer cell biology. ..
- Evaluation of High-Capacity Adenovectors in the EyeJoseph Bruder; Fiscal Year: 2006..The long-term goal of this proposal is the clinical testing and commercialization of PEDF expressing HC vector for the treatment of AMD and other ocular neovascular diseases. [unreadable] [unreadable]..
- Vaccination with Regulatory T Cell DepletionMichael Morse; Fiscal Year: 2007..Future studies will assess whether the greater magnitude and durability of the CEA-specific immune responses will translate into a long-term clinical benefit. [unreadable] [unreadable] [unreadable]..
- Engineering transgenic silkworms to produce spider silk fibersDonald Jarvis; Fiscal Year: 2008..These projects will exploit current knowledge of specific peptide motifs contributing tensile strength or elasticity to spider silks. [unreadable] [unreadable] [unreadable]..
- Gene Therapy for Usher Syndrome (USH1C)Jean Bennett; Fiscal Year: 2008..unreadable] [unreadable] [unreadable]..
- NATIONAL GENE VECTOR LABORATORY AT INDIANA UNIVERSITYKenneth Cornetta; Fiscal Year: 2007..abstract_text> ..
- Molecular Basis of Flavivirus NeurovirulenceWilliam Wold; Fiscal Year: 2007..abstract_text> ..
- HUMAN MITOCHONDRIAL FOLATE/ANTIFOLATE TRANSPORTRICHARD MORAN; Fiscal Year: 2008..abstract_text> ..
- Immunoglobulin Allotypes in Hepatitis C Virus InfectionJANARDAN PANDEY; Fiscal Year: 2008..Results of this investigation will advance our understanding of the role of host genetic factors in clearance and persistence of hepatitis C virus infection. ..
- A Biological Basis for Repair of the ACLChristopher Evans; Fiscal Year: 2009..MRI, histology and immunohistochemistry will also be used to evaluate the healed ligament. These studies will advance our knowledge of the biology of the injured ACL and suggest novel, biologically-based approaches to healing. ..
- MORPHOLOGIC/NEUROCHEMICAL CORRELATES OF DEPRESSION IN ADGEORGE ZUBENKO; Fiscal Year: 2009..abstract_text> ..
- PROFILING AUTOANTIGENS IN UVEITISWei Li; Fiscal Year: 2010..These efforts will advance our understanding of the disease etiology and prognosis, and improve the diagnosis and therapy. ..
- PATHOGENESIS OF DISEASE IN MUCOPOLYSACCHARIDOSIS I AND VIIKatherine P Ponder; Fiscal Year: 2010..These studies may demonstrate that the development of disease in the aorta and the cervical spine in MPS is due to a common mechanism, and may identify a therapy for these difficult-to-treat sites. ..
- Genetic Regulatory Network Controlling Vertebrate Eye FormationMichael E Zuber; Fiscal Year: 2010..Identification of the genes and genetic pathways in the developing eye will provide a much-needed understanding of the genetic mechanisms underlying eye formation and provide a window into understanding and treating human blindness. ..
- A novel transgenic silkworm system for recombinant glycoprotein productionDonald L Jarvis; Fiscal Year: 2010..Finally, these results will be of great interest to bioengineers working to overcome the evolutionary limitations of lower eukaryotic systems for recombinant glycoprotein production. ..
- Molecular Genetics of Cadmium ToxicityDaniel W Nebert; Fiscal Year: 2010....
- NEURAL REGULATION OF VASOPRESSIN RELEASEJ Thomas Cunningham; Fiscal Year: 2010..Benefits: The results will provide new information regarding the control of vasopressin secretion and how these systems contribute to pathophysiology. ..
- N-GLYCOSYLATION MECHANISM IN INSECT CELLSDonald L Jarvis; Fiscal Year: 2010....
- Tumor Sensitization to Purine Analogs by E. coli PNPEric J Sorscher; Fiscal Year: 2010....
- Hamster Model for Oncolytic Adenovirus VectorsWILLIAM SM WOLD; Fiscal Year: 2010..Although not proposed specifically in this application, these studies should also provide novel information on adenovirus pathogenesis. ..
- PAHs: Balance of Detoxication vs Metabolic ActivationDaniel Nebert; Fiscal Year: 2009..dissemination of ingested BaP and will be informative in clinical studies in which we would determine which haplotypes of these three human genes might be associated with resistance vs. sensitivity to PAH-induced toxicity and cancer. ..
- Transfer of IL-1Ra cDNA to Osteoarthritic KneeChristopher Evans; Fiscal Year: 2008..abstract_text> ..
- (SCOR) Mechanistic Studies of CF Pathogenesis and CL- S*Eric Sorscher; Fiscal Year: 2006..In summary, we propose an aggressive, multi-pronged attack aimed at developing a comprehensive and mechanistic understanding of CI- secretion in CF pathophysiology. ..
- Gene Therapy of Autosomal Recessive RPSimon Petersen Jones; Fiscal Year: 2006..abstract_text> ..
- GENE THERAPY FOR BLOOD PROTEIN DEFICIENCIESKATHERINE PONDER; Fiscal Year: 2005..Success in this project might lead to a safe, effective, and permanent therapy for Hemophilia B. ..
- CYP1A1 GENE AND ENVIRONMENTAL TOXICITYDaniel Nebert; Fiscal Year: 2005..abstract_text> ..
- Animal Model for Adenovirus Oncolytic VectorsWilliam Wold; Fiscal Year: 2004..We will inject subcutaneous tumors with vectors and evaluate the efficacy and safety of these vectors by gross pathology, immunohistochemistry, and histopathology. ..
- Animal Model for a Hereditary Macular DegenerationJean Bennett; Fiscal Year: 2004..These studies aim to reveal specific biological pathways relevant to the pathology in this animal model. Any such pathways could potentially be manipulated in the future to slow/prevent the disease process. ..
- Modulation of CD4+ T cells in autoimmune uveitisWei Li; Fiscal Year: 2003..These studies will not only yield new insights for the underlying causes and pathogenesis of autoimmune uveitis, but may also lead to a more selective immunotherapy for the disease. ..
- BIOLOGICAL MARKER FOR PRIMARY DEMENTIA IN THE ELDERLYGEORGE ZUBENKO; Fiscal Year: 2003....
- GENETICS OF RECURRENT EARLY-ONSET DEPRESSION (GENRED)GEORGE ZUBENKO; Fiscal Year: 2002..A timetable is proposed for rapid sharing of all biological materials, blinded clinical data, genotypes and linkage analyses with the scientific community through the NIMH-sponsored Center for Genetic Studies. ..
- GLYCOGEN STORAGE DISEASE TREATMENT: HEPATIC GENE THERAPYBarry Byrne; Fiscal Year: 2002..These studies will yield important new information in establishing a clinically relevant treatment for these fatal diseases and add new understanding to the basic pathophysiology of GSD. ..
- NADPH P450 REDUCTASE--THYROID HORMONE REGULATIONDavid Waxman; Fiscal Year: 2002....
- GENOTOXICITY IN CYP1A2 DEFICIENT TRANSGENIC MICEDaniel Nebert; Fiscal Year: 2002..S. population. ..
- DEXASOME BASED IMMUNOTHERAPY OF LUNG CANCERMichael Morse; Fiscal Year: 2002..This clinical trial will form the background for further trials designed to demonstrate both immunology and clinical benefits of dexosome-based immunotherapy. ..
- TARGETING ANGIOGENESIS WITH PROTEASE ACTIVATABLE VECTORSStephen Russell; Fiscal Year: 2002..abstract_text> ..
- GENE THERAPY FOR HURLERS DISEASEDonald Kohn; Fiscal Year: 2001..Successful tolerance induction in murine and canine models of MPS I will be an Important step toward application of gene therapy in human patients. ..
- CONTROL OF SODIUM INTAKE IN THE HINDLIMB UNWEIGHTED RATJ Cunningham; Fiscal Year: 2005..Additionally, these data may contribute to understanding of health problems associated with an aging population and with cardiovascular disease such as congestive heart failure. ..
- Syrian Hamster as a Permissive Model for Testing Anti-Adenovirus DrugsWilliam Wold; Fiscal Year: 2007..Success in this project could lead to the eventual approval of drugs to treat adenovirus infections in immunosuppressed patients. [unreadable] [unreadable] [unreadable]..
- NEUROTROPIC CYTOMEGALOVIRUS DURING IMMUNOSUPPRESSIONJON REUTER; Fiscal Year: 2006..abstract_text> ..
- BD Biosciences FACS Canto Flow CytometerLAWRENCE LAMB; Fiscal Year: 2006..The laboratory will require an instrument that will serve the laboratory in the development and execution of advanced cell therapy trials. [unreadable] [unreadable]..
- ADENOVIRUS E3 PROTEINS AND TUMOR NECROSIS FACTORWilliam Wold; Fiscal Year: 2001....
- Postsynthetic Modifications of Bacterial Membrane LipidsJohn Cronan; Fiscal Year: 2006..These processes will be studied by a combination of biochemical, genetic, and molecular biological approaches including tests of pathogenesis in a mouse model system ..
- Viral Mediated Gene Therapy for Retinal DiseasesJohn Flannery; Fiscal Year: 2005..In summary, this application supports key, initial "proof-of-principle" experiments to create retina-specific viral vectors and systems to transfer neurotrophic factors for gene therapy of retinal degeneration. ..
- Vaccine Induced Mucosal Protection Against TB InfectionDANIEL HOFT; Fiscal Year: 2005..Prime/boosting strategies found to prevent BCG infection after aerosol challenges will be studied for the ability to prevent infection with virulent M. tuberculosis by aerosol challenge. ..
- LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLSDonald Kohn; Fiscal Year: 2005..This "intracellular immunization" approach may provide a therapeutic measure against HIV-1 that adds to pre-existing therapies, such as anti-retroviral drugs. ..
- Novel Neuronal Transport Mechanisms of Lyosomal EnzymesMark Sands; Fiscal Year: 2004..abstract_text> ..
- TRYPANOSOMA CRUZI MUCOSAL & SYSTEMIC PROTECTIVE IMMUNITYDANIEL HOFT; Fiscal Year: 2008..Chemokine and/or integrin receptor expression will be studied by flow cytometry and RT-Real-Time PCR, and the in vivo effects of defective chemokine and integrin binding and/or signaling will be evaluated. [unreadable] [unreadable]..
- YELLOW FEVER 17D-BASED CHIMERIC FLAVIVIRUS VACCINESWilliam Wold; Fiscal Year: 2002....
- HSV Amplicon Vectors for Cerebral IschemiaEnnio Chiocca; Fiscal Year: 2004..abstract_text> ..
- AAV-mediated gene correction in retinaJean Bennett; Fiscal Year: 2007..unreadable] [unreadable]..
- GENE THERAPY AND SEIZURESThomas 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. ..
- REGULATION OF TRANSCRIPTION BY THE RB ANTIONCOGENEPaul Robbins; Fiscal Year: 2002..The experiments described in the proposal are expected to provide important insights into the mechanism of how RB and cyclin D1 function to regulate transcription directly, cell differentiation, and tumorigenesis. ..
- Effect of HIV-1 Vpr on Basic Cellular Functions (II)Richard Zhao; Fiscal Year: 2007..abstract_text> ..
- QUALITATIVE NATURE OF ANTIBODIES TO HCV GLYCOPROTEIN SUBUNIT VACCINE IN HUMANSRanjit Ray; Fiscal Year: 2009..Our proposed complimentary experimental approaches in this vaccinated cohort will help in developing successful vaccine strategies for the intervention of the first crucial step of the HCV life cycle. ..
- 2008 Viral Vectors for Gene Therapy, The Science of Gordon Research ConferencePaul Robbins; Fiscal Year: 2008..End of Abstract) [unreadable] [unreadable] [unreadable]..
- Inhibition of NF-KB to Facilitate Islet TransplantationPaul Robbins; Fiscal Year: 2003..The successful completion of the proposed studies should determine if NF-kB inhibition in islets by peptide and gene mediated transduction is able to improve islet viability prior to and post-transplantation. ..
- Study of Leptin Receptor Deficiency in FatAllan Zhao; Fiscal Year: 2007..We believe that these studies will provide a possible molecular linkage between adipocyte-selective leptin receptor deficiency and type 2 diabetes. ..
- S. typhimiurium Vaccine Against Bacterial EnteropathogensRoy Curtiss; Fiscal Year: 2007..abstract_text> ..
- Mesenchymal Stem Cells for Fetal Gene DeliveryAlice Tarantal; Fiscal Year: 2006..abstract_text> ..
- Hantavirus Vaccines Based On Nonreplicating AdenovirusesDavid Johnson; Fiscal Year: 2005..This will be a prelude to challenge experiments in which vaccinated hamsters will be challenged with Andes virus that causes a lethal pulmonary disease similar to that in humans. ..