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Day 1 - Tuesday 16th September 2008
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08:00 |
Registration |
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08:55 |
Chair: Enal Razvi, Biotechnology Analyst, Selectbiosciences |
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09:00 |
Combination Approaches to Contain Ongoing Coxsackievirus Infection
Jens Kurreck, Professor, Universität Stuttgart
We employ RNA interference to inhibit coxsackievirus B3, a major heart pathogen. Antiviral potency of multiple shRNAs as well as of a combination of a soluble variant of the virus receptor with an siRNA will be compared. |
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09:30 |
Therapeutic Applications of RNAi for HIV Infection
John Rossi, Professor, Beckman Research Institute |
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10:00 |
The Off-Target Specificity of siRNA on Near-Perfectly Matched Targets
Zicai Liang, Professor, Peking University
The off-target effects of single siRNAs on their single- and double mutated targeting sites were exmined and mechanism of mismatch tolerance in RNAi was explored. |
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10:30 |
Coffee and Networking |
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11:30 |
Pharmaceutical Development of Dicer-substrate siRNAs
Mark Behlke, Chief Scientific Officer, Integrated DNA Technologies
Dicer-substrate siRNA (DsiRNA) are synthetic oligonucleotides that are processed by Dicer prior to RISC loading. DsiRNA often show improved potency over traditional siRNA in vitro and show similar benefits in vivo. |
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12:00 |
Small RNAs and Cell Proliferation/Differentiation
Annick Harel-Belan, Director, Laboratory of Epigenetics and Cancer, Institute Andre Lwoff
Small RNAs are essential components in the control mammalian cell proliferation/differentiation and in tumorigenesis. Small RNAs can also be used to explore this control in normal and tumor cells. |
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12:30 |
Lunch & Networking |
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14:30 |
Development and Optimization of a Novel RNAi Therapeutics Platform
Dimitry Smarsky, Vice President, RXi Pharmiceuticals
The design and effective delivery of synthetic RNAi compounds are important factors for therapeutic applications. We will present data obtained with our proprietary rxRNA™ compounds, which can be up to 100 times more potent than conventional siRNAs, demonstrate nuclease resistance, and are potentially more specific for their intended targets. We will also discuss the possible mechanism of incorporation of chemically modified molecules into the RNAi machinery. |
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15:00 |
Analysis of the MicroRNA and Epigenetics Market Landscape
Enal Razvi, Biotechnology Analyst, Select Biosciences
In this presentation, I will present some vignettes from our continuing industry coverage of the microRNA research tools, diagnostics/therapeutics, as well as the epigenetics marketplace. The data reveal market trends that will be discussed in the broader context of the evolution of these fields, and the associated market opportunities that they create. |
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15:30 |
Coffee and Networking |
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16:00 |
RNAi: From Mechanism to Medicine
Craig Mello, Professor, University of Massachusetts*
RNAi offers astounding potential for understanding and manipulating the genetic basis of disease and yet there are still many mysteries regarding its underlying mechanism. This talk will describe the discovery of RNAi and explore those remaining mysteries.
*Video Presentation and Live Telephone Link |
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17:30 |
Title to be Confirmed |
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18:00 |
End of Day 1 |
Day 2 - Wednesday 17th September 2008
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08:00 |
Title to be Confirmed |
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08:30 |
RNAi as Molecular Therapeutics for Liver Cancer
John Luk, Associate Professor, Hong Kong University
Liver cancer is one of the most common and aggressive malignancies, and there is no effective drug today. By molecular profilings, we have identified the molecular determinants for HCC. Today we determine the clinical utility of RNAi as cancer therapeutics. |
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09:00 |
Identification of Targets for a Cartilage Specific MicroRNA
Tamas Dalmay, Professor, University of East Anglia
We identified target genes for miR140 by suppressing or over-expressing the miRNA in cells and profiling the expression of mRNAs. Different approaches to process the data will be discussed. |
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09:30 |
How Small RNAs Regulate Multiple Target mRNAs
Joerg Vogel, Max Plank Institute
Small non-coding RNAs (sRNAs) are an emerging class of post-transcriptional regulators of bacterial gene expression. Such RNAs are typically ~ 50 to 250 nucleotides in length, and are expressed from the intergenic regions of bacterial chromosomes. Where studied in detail, most sRNAs were found to act on trans-encoded mRNAs to modulate their translation and stability. |
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10:00 |
Quantification and Functional Analysis of miRNA in Mammalian Cells
Martin Kreutz, Scientist R&D – miRNA Research, Qiagen
Qiagen have developed a robust and accurate method for transcriptome-wide miRNA quantification using SYBR Green detection-based, real-time PCR. The miScript System is highly specific, sensitive, and requires very small amounts of input RNA. |
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10:30 |
Coffee & Networking in Main Exhibition Hall |
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11:30 |
Targeted Genome Editing in Mammalian Cells Using Engineered Zinc Finger Nucleases
Trevor Collingwood, Manager Strategic Development, Sigma Aldrich
Rational genome engineering in mammalian cells is of enormous potential across basic research, drug-discovery as well as cell-based medicines. To this end, Sangamo Biosciences and Sigma-Aldrich have recently partnered to commercialize a novel technology that enables high-frequency genome editing via the application of designed zinc finger nucleases (ZFNs). Within these chimeric proteins the DNA binding specificity of the zinc finger protein determines the site of nuclease action. Such engineered ZFNs are able to recognize and bind to a specified locus and evoke a double-strand break in the targeted DNA with high efficiency and base-pair precision. The cell then employs the natural DNA repair processes of either “homology-directed repair” or “non-homologous end joining” to heal the targeted break. These two pathways provide the investigator with the ability to provoke three unique outcomes in genome editing – gene correction, gene deletion and targeted gene addition. Furthermore, the speed and efficiency of this process enables us to knockout multiple genes in the same cell. Drawing from our work with transformed cell lines, primary human cells, and multi-potent stem cells, we will present several examples of single, double and triple gene knockout, as well as targeted gene insertion into native chromosomal loci. |
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12:00 |
To Be Confirmed |
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12:30
13:00 |
Lunch & Poster Presentations
Integromics - Free Workshop!
Real-Time qPCR data analysis: Where not to fail
Ramon Goni, Integromics’ qPCR specialist (Madrid) |
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14:30 |
Targeting microRNAs in Cardiovascular Disease
Thomas Thum, Professor, University of Wurzburg
MicroRNAs negatively regulate gene expression by promoting mRNA degradation and inhibiting mRNA translation. In the past a variety of microRNAs that regulate cardiac development and function were identified. We now focus on therapeutic manipulation of microRNAs in cardiovascular disease. |
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15:00 |
A Model System to Test the Effects of Design and Modification on siRNA Activity
Glen Reid, Head of RNAi Product Development, Genesis Research & Development Corporation Limited |
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15:30 |
Coffee & Networking in Main Exhibition Hall |
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16:30 |
Cooperative microRNA Targeting and Regulation
Pal Saterom, Professor, Norwegian University of Science and Technology
Multiple microRNA target sites within a 3' UTR give synergistic down-regulation, but only if the distance between the target sites is in an optimal range. This result has implications for miRNA target prediction and siRNA design. |
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17:00 |
Large Scale Manufacture of RNA Duplexes
Kevin Fettes, Process Development Group Leader, Avecia Biotechnology
The practicalities of manufacturing RNA duplexes at a scale required to provide materials for clinical trials and commercial launch will be discussed using case studies. |
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17:30 |
Dual Role for Argonaute Proteins in MicroRNA Processing
Sven Diederichs, Research Fellow, German Cancer Research Center
Argonaute proteins, known effectors of RNA interference, are newly characterized as post-transcriptional regulators of mature miRNA expression. Ago2, the RNase in RNAi, also cleaves the pre-miRNA into a novel precursor and thus actively participates in microRNA processing. |
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18:00 |
Drinks Reception - Sponsorship available, please contact paul.raggett@selectbiosciences.com |
Day 3 - Thursday 18th September 2008
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08:00 |
Sigma - Free Workshop!
Chemical Modifications in the Seed Region Backbone can Significantly Reduce siRNA Off-Target Effects
Erik Eastlund, Senior Scientist, Sigma Life Science
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08:30 |
SilenciX Cell Lines for Valuable Insights Into Cellular Pathways
Nadia Normand, Bioassay Manager, Tebu-Bio |
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09:00 |
Identification of Chemical Modifications that Enhance siRNA Specificity Without Compromising siRNA Potency
Susan Magdaleno, Sr. Manager, Scientist RNAi Technologies, Applied Biosystems
We will describe the experimental studies and results leading to the discovery of a novel arrangment of chemical modifications in an siRNA duplex that significantly reduce off-target effects as measured by microarray and cell based assays. |
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09:30 |
Enabling RNAi Therapeutics the Non-Viral Way
Andy Miller, Professor, Imperial College London
Synthetic, self-assembly ABCD nanoparticles represent a new nanotechnology concept for the delivery of therapeutic nucleic acids including siRNA and plasmid DNA (pDNA). These nanoparticles consist of AB core particles formed from nucleic acids (A-component) and cationic liposomes (B-component), which are then coated with variable amounts of a stealth/biocompatibility polymer (C-component) to provide stability in biological fluids and also with optional biological ligands (D-component) if required for active targeting. In this lecture, the development of purpose designed and assembled ABC nanoparticles will be described that enable successful delivery of siRNA to liver (for anti-HBV treatment), and delivery of pDNA to the lung opening up opportunities for lung therapies. We also describe a theranostic approach to siRNA delivery to tumour that opens up real promise for siRNA anti-cancer therapeutics in the future. |
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10:00 |
RNA-Mediated Epigenetic Heredity : From a White-Tipped Tail to Familial Diseases
Minoo Rassoulzadegan, Professor, University of Nice
Consistent with converging evidence of a role of RNA in the establishment of epigenetic states and with the detection of RNA in human spermatozoa, our results reveal an unexpected mode of epigenetic inheritance by the zygotic transfer of RNA molecules. |
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10:30 |
Coffee & Networking in Main Exhibition Hall |
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11:30 |
siRNA and 3pRNA for Tumor Therapy
Gunther Hartmann, Professor, University of Bonn
The cytosolic helicase RIG-I detects RNA containing a triphosphate group at the 5´end (3pRNA) leading to the induction of an antiviral immune response upon exposure to negative strand RNA viruses. Here we combine gene silencing (siRNA) and 3pRNA for tumor therapy in the B16 melanoma model. We demonstrate that both functional activities contribute to antitumor activity in vivo. |
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12:00 |
Functionally-Tested siRNAs Deliver Potent and Extended Gene Silencing Using Low Concentrations.
Eli Hefner, PhD, Bio-Rad
We will discuss dsRNA molecules that deliver effective gene silencing using low concentrations. They are functionally tested via RT-qPCR for ≥85% mRNA knockdown and demonstrate sustained silencing for up to 6-9 days. |
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12:30 |
Award Presentation:
Bio-Rad European RNAi Award |
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12:35
13:00 |
Lunch & Poster Presentations
Thermo Fisher - Free Workshop!
Access to a New World of RNAi-based Discovery
Stephanie Urschel, Senior Field Scientist, Thermo Fisher Scientific |
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14:00 |
Title to be Confirmed |
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14:30 |
miCHIP and miQPCR, A Suit for Expression Profiling of Mature microRNA
Martina Muckenthaler, Head of Molecular Medicine, University of Heidelberg |
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15:00 |
RNAi Against a Moving Target: HIV-1 Gene Therapy
Ben Berkhout, Professor, University of Amsterdam
The exquisite sequence-specificity of the RNAi mechanism is a major advantage in therapeutic applications. However, it also creates new problems when targeting the rapidly evolving HIV-1. This virus will easily escape by selecting mutations in the targeted sequence. Thus, new escape-proof antiviral strategies are warranted. |
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15:30 |
Coffee and Networking |
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16:00 |
A High-Throughput Bead-Based Flow Cytometric miRNA Assay, Based on the Luminex® Technology
Karel Fostier, Phd Student, Laboratory of Molecular and Cellular Therapy, Vrije Universiteit Brussel
MicroRNAs have emerged as a group of tiny non-coding regulatory molecules. To understand their role, a sensitive and specific profiling methodology is warranted. Here we present a high-throughput bead-based flow cytometric miRNA assay, based on the Luminex® technology. |
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16:30 |
Close of Conference |
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