Registration

Lentivirus Enabled shRNA Drug Modifier Screens: Panoramic View of the Human Genome One Hairpin at a Time
David Shum, Assay Development Specialist, Memorial Sloan-Kettering Cancer Center, United States of America

This presentation describes the first drug modifier screen at the genome level to interrogate drug-gene interactions. We highlight the advantages of our arrayed shRNA screening approach to evaluate the genome one hairpin at a time and identified several pathways that influence drug responsiveness.

Locked Nucleic Acid: A Decade of Achievements
Bo Hansen, Director/Vice President, Santaris Pharma A/S, Denmark

Locked Nucleic Acid (LNA) is today established as one of the most potent inhibitors of RNA. The presentation will focus on some of the highlights of the past 10 years R/D and lessons learned. The advanced level of LNA antisense technology will be illustrated by the latest data.

Coffee and Networking in Exhibition Hall

Efficient Use of Accessibility in microRNA Target Prediction
Jiri Vanicek, Assistant Professor, EFPL, Switzerland

This presentation describes an efficient and precise microRNA target prediction algorithm. The predictions of the algorithm are selected by accessibility of the target mRNA and ranked according to the overrepresentation of the partially accessible binding sites.

microRNA-Binding Protein Argonaute Binds to mRNAs Without microRNAs
Anthony Leung, Assistant Professor, Johns Hopkins Bloomberg School of Public Health, United States of America

We analyzed genome-wide binding sites of Ago2 in wild-type mouse embryonic stem cells and derivatives that lack mature microRNAs, resulting in a highly validated set of microRNA targets. Unexpectedly, we also identified a motif associated with Ago2 independent of microRNAs.

Lunch

Free Workshop
The miRNA Revolution Tools for miRNA purification and expression profiling

Free Workshop
Experimental Workflows for Successful miRNA Research Applications

Poster Viewing Session 1

Genome-Wide Decoding of miRNA-mRNA Interactions by HITS-CLIP
Chi Wook, Postdoctoral Fellow, Cold Spring Harbor Laboratory, United States of America

Application of high-throughput sequencing of Argonaute-bound RNAs isolated by cross linking immunoprecipitation (Ago HITS-CLIP) platform to identify miRNA target sites in vivo and new rules of miRNA target recognition decoded by Ago HITS-CLIP analysis.

Strategies for Improving RNAi Screening Success: Streamlining Primary Screens Through Hit Stratification
James Goldmeyer, Technical Marketing Scientist, ThermoFisher, Canada

RNAi library screening presents unique challenges that must be addressed to ensure success. A cell-based assay using a ubiquitin-EGFP cell line to screen for drug target genes that inhibit proteasome function will be discussed; focusing on data analysis, hit identification and stratification methodologies.

Coffee and Networking in Exhibition Hall

MicroRNA-31 is Secreted by Senescent Endothelial Cells and Inhibits Osteogenic Differentiation of Mesenchymal Stem Cells
Johannes Grillari, Chief Scientific Officer/Co Founder, Evercyte GmbH, Austria

We have found that a microRNA is packaged into exosomes and secreted by endothelial cells and inhibits osteogenic differentiation after uptake in mesenchymal stem cells. Since the miRNA is also increased in the serum of elderly it might contribute to the age-related disease osteoporosis.

Targeted Delivery of PLK1-siRNA by Single-Chain Antibody Suppresses Her2+ Breast Cancer Growth and Metastasis
Erwei Song, professor, Sun-Yat-Sen Memorial Hospital, China

The intravenously injected siRNA complexes retarded Her2+ breast tumor growth, reduced metastasis and prolonged survival without evident toxicity. These data suggest that Her2-ScFv-protamine peptide fusion protein could be used to deliver siRNAs to treat Her2+ breast cancer.

Aberrancies of the miRNome in Human Cancer: Causes and Consequences
Muller Fabbri, Research Scientist, Ohio State University, United States of America

MicroRNAs are gene expression regulators. They are frequently de-regulated in tumors. This talk will highlight the state-of-the-art knowledge on the causes of this aberrant expression, and the consequences of these aberrancies that lead to the development of the malignant phenotype.

Human Glioma Growth is Controlled by MicroRNA-10b
Galina Gabriely, Postdoctoral fellow, Brigham and Women's Hospital, United States of America

The presentation focuses on microRNA miR-10b which is not detected in normal brain, but highly expressed in glioma – the most common and aggressive primary brain tumor. With various approaches, we show significant role for this miRNA in glioma survival.

Drinks Reception

siRNA Induces Synthetic Lethality In Vivo
Michael Goldberg, Post-doc, MIT, United States of America

This talk will discuss the application of a novel siRNA delivery system for the treatment of ovarian cancer with RNAi therapeutics.

Novel siRNA Formulations for Delivery to the Lung
Jason Fewell, Director, Expression Genetics Inc, United States of America

The lack of safe and efficient delivery systems remain a fundamental problem in the development of RNAi based therapeutics. We have developed a family of structurally flexibility lipopolyamines that are designed specifically for in vivo applications. Here we report on one system that is administered iv and achieves lung specific knockdown activity through increased siRNA retention.

Coffee and Networking in Exhibition Hall

Invivofectamine® 2.0, a New, Commercially Available, Reagent for In Vivo Delivery of siRNA
Xavier de Mollerat du Jeu, Associate R&D Director, Life Technologies, United States of America

Invivofectamine™ 2.0 is a new in vivo delivery reagent. After a single injection of siRNAs complexed with thisreagent, we observed 90% mRNA and protein level reduction in liver for more than 2 weeks.

Optimization of siRNA Drug-Like Properties
Elena Feinstein, Chief Scientific Officer, Quark Pharmaceuticals Inc, Israel

The presentation will describe Quark’s approaches to generation of lead siRNA molecules and the nature and sensitivity of the methods used for evaluation of siRNA activity and drug-like properties.

siRNA Eluting Surfaces as a Novel Concept for Local Gene Silencing
Andrea Nolte, Research Scientist, Tuebingen University Hospital, Germany

We designed a coating consisting of Polyethylenimine, siRNA and gelatin, for local RNAi application to reduce pathogenic gene expression. The coating mediates no toxicity when administered in adequate quantities and the release of siRNA occurs rapid.

Lunch

Free Workshop
Michelson Prize & Grants Information Session Learn about the $75 million challenge!

Poster Viewing Session 2

RNAi Nanomedicines: Challenges and Opportunities within the Immune System
Dan Peer, Head, Tel Aviv University, Israel

RNA interference (RNAi)-based approaches have greatly contributed to better understanding of gene expression and function in-vitro. The capability to apply these strategies in-vivo in order to validate the role of specific genes in normal or pathological conditions, and to induce therapeutic gene silencing, opened new avenues for utilising RNAi as a novel therapeutic modality. However, the translation of RNAi from an effective genomic tool into clinical applications has been hindered by the difficulty to deliver RNAi molecules into their target tissues by systemic administration, especially to hematopoietic cells. Here, we describe current systemic RNAi delivery platforms that are targeted to leukocytes. Major focus is given to the integrin-targeted stabilised nanoparticles (I-tsNP) strategy, which utilises leukocytes' integrins for the delivery of siRNAs exclusively to cells of the immune system. Examples from inflammatory bowel diseases, viral infections and blood cancers will be discussed.

Direct Detection and Identification of miRNA. No More Extension - Reverse Transcription and PCR
Mark Bradley, Professor, DestiNA Genomics Ltd, United Kingdom

With the explosion in miRNA research, new tools for profiling these small molecular gene silencers need to be developed. Current detection methods have focused on developing better ways of extending these small oligonucleotides to improve reverse transcription. Here we present a novel chemical approach and methodology, which enables miRNA profiling without the need of extension, reverse transcription and PCR. High affinity molecular probes combined with smart chemically customised nucleobases, taking advantage of dynamic chemistry thermodynamics and Watson-Crick bonding to enable error free direct detection and identification of miRNAs. Either mass spectrometry or colorimetric detection methods are possible.

Coffee and Networking in Exhibition Hall

Exclusionary Commerical Rights: Patent Protection on Short Functional RNAs
Kathleen Williams, Partner, Edwards Angell Palmer & Dodge LLP, United States of America

This presentation will provide an overview of the Patent Landscape for short RNAs, including SIRNAs, Antisense RNAs, and MicroRNAs, and identify the key interests in these patents, identifying key US patents as well as key European patents.

Close of Conference