Research Topics
| N J CaplenSummaryAffiliation: National Institutes of Health Country: USA Publications
| Collaborators
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Detail Information
Publications
Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivoUta Griesenbach
Department of Gene Therapy, Faculty of Medicine, National Heart and Lung Institute, Imperial College, London, UK
Respir Res 7:26. 2006..The cationic lipid Genzyme lipid (GL) 67 is the current "gold-standard" for in vivo lung gene transfer. Here, we assessed, if GL67 mediated uptake of siRNAs and asODNs into airway epithelium in vivo...- Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systemsN J Caplen
Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA
Proc Natl Acad Sci U S A 98:9742-7. 2001..These observations may open a path toward the use of siRNAs as a reverse genetic and therapeutic tool in mammalian cells... 
Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interferenceNatasha J Caplen
Medical Genetics Branch, National Human Genome Research Institute and Neurogenetics Branch, National Institute of Neurological Diseases, National Institutes of Health, Bethesda, MD 20892, USA
Hum Mol Genet 11:175-84. 2002..This study demonstrates the feasibility of targeting a transcript associated with an important group of genetic diseases by RNAi...- Inhibition of viral gene expression and replication in mosquito cells by dsRNA-triggered RNA interferenceNatasha J Caplen
National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, 20892, USA
Mol Ther 6:243-51. 2002.... - RNAi as a gene therapy approachNatasha J Caplen
Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, 10 Center Drive, 10C103, Bethesda, MD 20892, USA
Expert Opin Biol Ther 3:575-86. 2003..This review will describe the status of the science behind this novel mechanism and will illustrate the therapeutic potential of RNAi-based technologies, using examples from these critical clinical research areas... - dsRNA-mediated gene silencing in cultured Drosophila cells: a tissue culture model for the analysis of RNA interferenceN J Caplen
Clinical Gene Therapy Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA
Gene 252:95-105. 2000..This invertebrate tissue culture model should allow facile assays for loss of function in a well-defined cellular system and facilitate further understanding of the mechanism of RNAi and the genes involved in this process... - RNAi screening identifies TAK1 as a potential target for the enhanced efficacy of topoisomerase inhibitorsS E Martin
Gene Silencing Section, Genetics Branch, CCR, NCI, NIH, Bethesda, MD 20892, USA
Curr Cancer Drug Targets 11:976-86. 2011..These findings may offer avenues towards lowering the effective doses of Top1 inhibitors in cancer cells and, in doing so, broaden their application... - Gene therapy progress and prospects. Downregulating gene expression: the impact of RNA interferenceN J Caplen
Gene Silencing Section, Office of Science and Technology Partnerships, Office of the Director, Center for Cancer Research, NCI, Bethesda, MD 20892, USA
Gene Ther 11:1241-8. 2004..RNAi has been adapted as a functional genomics tool and it has potential as a therapeutic approach. This review will summarize our current understanding of the RNAi mechanism and the various applications of RNAi-based technologies... - Adenovirus vectors as transcomplementing templates for the production of replication defective retroviral vectorsW J Ramsey
Clinical Gene Therapy Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
Biochem Biophys Res Commun 246:912-9. 1998..This adeno-retroviral chimeric vector system could simplify the generation and testing of different retroviral vectors, particularly where assessment of vectors with alternative envelopes carrying novel targeting ligands is required... - Single-step doxorubicin-selected cancer cells overexpress the ABCG2 drug transporter through epigenetic changesA M Calcagno
Laboratory of Cell Biology, Center for Cancer Research, National Cancer Institute, NIH, DHHS, Bethesda, MD 20892, USA
Br J Cancer 98:1515-24. 2008..This is the first report to our knowledge of single-step, low-dose selection leading to overexpression of ABCG2 by epigenetic changes in multiple cancer cell lines... - Asparagine synthetase as a causal, predictive biomarker for L-asparaginase activity in ovarian cancer cellsPhilip L Lorenzi
Genomics and Bioinformatics Group, Room 5056B, 37 Convent Drive, Bethesda, MD 20892, USA
Mol Cancer Ther 5:2613-23. 2006..Overall, this pharmacogenomic/pharmacoproteomic study suggests the use of l-ASP for treatment of a subset of ovarian cancers (and perhaps other tumor types), with ASNS as a biomarker for patient selection... 
The identification of microRNAs in a genomically unstable region of human chromosome 8q24Konrad Huppi
Gene Silencing Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, NIH, Bethesda, MD 20892, USA
Mol Cancer Res 6:212-21. 2008..High levels of expression of the hsa-miR-1204 precursor is also seen in several epithelial cancer cell lines with MYC/PVT1 coamplification, suggesting a potentially broad role for these miRNAs in tumorigenesis...- Pvt1-encoded microRNAs in oncogenesisGabriele B Beck-Engeser
Department of Microbiology and Immunology, University of California, San Francisco, CA 94143 0414, USA
Retrovirology 5:4. 2008..The recent identification of non-coding microRNAs encoded within the PVT1 region has suggested a regulatory role for this locus... - Applications of RNA interference in mammalian systemsScott E Martin
Gene Silencing Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
Annu Rev Genomics Hum Genet 8:81-108. 2007..Here, we review many of the ongoing applications of RNAi in mammalian and human systems, and discuss how advances in our knowledge of the RNAi machinery have enhanced the use of these technologies... - Multiplexing siRNAs to compress RNAi-based screen size in human cellsScott E Martin
Gene Silencing Section, Office of Science and Technology Partnership, OD, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA
Nucleic Acids Res 35:e57. 2007..This approach is likely to be especially applicable where assay costs or platform limitations are prohibitive... - MicroRNAs and genomic instabilityKonrad Huppi
Gene Silencing Section, Genetics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA
Semin Cancer Biol 17:65-73. 2007..In this review we discuss the possible role of altered miRNA expression on human cancer and conduct an analysis correlating the physical location of murine miRNAs with sites of genetic alteration in mouse models of cancer... - Allele-specific silencing of the dominant disease allele in sialuria by RNA interferenceRiko D Klootwijk
Medical Genetics Branch, NHGRI, NIH, 10 Center Dr, MSC 1851, Bethesda, MD 20892, USA
FASEB J 22:3846-52. 2008..These findings indicate that allele-specific silencing of a mutated allele is a viable therapeutic strategy for autosomal dominant diseases, including sialuria... - Mismatched siRNAs downregulate mRNAs as a function of target site locationScott E Martin
Gene Silencing Section, Office of Science and Technology Partnerships, Office of the Director, CCR, NCI, NIH, Bethesda, MD 20892, USA
FEBS Lett 580:3694-8. 2006..Our findings demonstrate the importance of target site location within endogenous mRNAs for small RNAs associated with RNAi... - Selective toxicity of NSC73306 in MDR1-positive cells as a new strategy to circumvent multidrug resistance in cancerJoseph A Ludwig
Laboratory of Cell Biology, Center for Cancer Research, National Cancer Institute, NIH, Bethesda, Maryland 20892, USA
Cancer Res 66:4808-15. 2006..This article shows that NSC73306 kills cells with intrinsic or acquired P-gp-induced MDR and indirectly acts to eliminate resistance to MDR1 substrates... - A new approach to the inhibition of gene expressionNatasha J Caplen
Trends Biotechnol 20:49-51. 2002 - Efficient delivery of RNA interference effectors via in vitro-packaged SV40 pseudovirionsChava Kimchi-Sarfaty
Laboratory of Cell Biology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD 20892-4256, USA
Hum Gene Ther 16:1110-5. 2005..Our findings indicate that SV40 pseudovirions may be a useful addition to the delivery systems currently being used for the transfer of RNAi effector molecules... - RNAi microarray analysis in cultured mammalian cellsSpyro Mousses
Cancer Drug Development Laboratory, Translational Genomics Research Institute TGen, Gaithersburg, Maryland 20878, USA
Genome Res 13:2341-7. 2003..In summary, this RNAi microarray platform, together with ongoing efforts to develop large-scale human siRNA libraries, should facilitate genomic-scale cell-based analyses of gene function... - Defining and assaying RNAi in mammalian cellsKonrad Huppi
Gene Silencing Section, Office of Science and Technology Partnerships, Office of the Director, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Building 37, 3128B, 37 Convent Drive, Bethesda, MD 20892, USA
Mol Cell 17:1-10. 2005..This perspective will aim to consider some of the issues encountered when conducting and interpreting RNAi experiments in mammalian cells... - Short interfering RNA (siRNA)-mediated RNA interference (RNAi) in human cellsNatasha J Caplen
Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland 20892, USA
Ann N Y Acad Sci 1002:56-62. 2003.... - RNAi quashes polyQNatasha J Caplen
Nat Med 10:775-6. 2004 - In situ generation of pseudotyped retroviral progeny by adenovirus-mediated transduction of tumor cells enhances the killing effect of HSV-tk suicide gene therapy in vitro and in vivoTakashi Okada
Division of Genetic Therapeutics, Center for Molecular Medicine, Jichi Medical School, Tochigi, Japan
J Gene Med 6:288-99. 2004.... - SpliceCenter: a suite of web-based bioinformatic applications for evaluating the impact of alternative splicing on RT-PCR, RNAi, microarray, and peptide-based studiesMichael C Ryan
Genomics and Bioinformatics Group, Laboratory of Molecular Pharmacology, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA
BMC Bioinformatics 9:313. 2008..In addition, the critical roles of alternative splice forms in biological function and in disease suggest that assay results may be more informative if analyzed in the context of the targeted splice variant... - Short interfering RNAs can induce unexpected and divergent changes in the levels of untargeted proteins in mammalian cellsPeter C Scacheri
National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA
Proc Natl Acad Sci U S A 101:1892-7. 2004.... - Kinase-independent functions for Itk in TCR-induced regulation of Vav and the actin cytoskeletonDerek Dombroski
National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA
J Immunol 174:1385-92. 2005..These data suggest a kinase-independent scaffolding function for Itk in the regulation of Vav localization and TCR-induced actin polarization...