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Agenda

Day 1 - 20 September

09:00

RNAi Technologies
Chaired by Volker Patzel, Group Leader, Max Planck Institute for Infection Biology

09:05

RNAi vs. K. O.: Phenotypic Comparison of shRNA-Expressing Animals and Knockout Mice Lacking TRPV1
Jens Kurreck, Group Leader, Berlin Free University
RNAi are currently employed to investigate the role of the vanilloid receptor TRPV1 in pain signalling. A comparative phenotypic characterization of knockout mice and transgenic animals continuously expressing shRNAs against TRPV1 will be presented.

09:35 

Formulations for Using Dicer-Substrate siRNA in vivo
Mark Behlke, Vice President, Molecular Genetics, Integrated DNA Technologies
Liposome formulations have been used to deliver Dicer-substrate siRNAs (DsiRNAs) by IV administration with knockdown of hepatic targets in mice. Even using lipid-based delivery, modified DsiRNAs do not activate immune responses and showed high potency in this in vivo application.

10:05 

LNA (Locked Nucleic Acid): Key Molecule Towards Realising Gene Silencing Drugs
Jesper Wengel, Professor, University of Southern Denmark
The gene silencing effect in cell cultures and in vivo of unmodified and LNA-modified siRNA and sisiRNA ("short internally segmented interfering RNA") constructs will be presented. Novel designs and results will be discussed. 

10:35

Coffee Break and Networking in Exhibition Hall 

11:20

Delivery Methods for Gene Silencing Experiments
Teresa Rubio, Group Leader, Bio-Rad
New methods to silence genes for a longer length of time when compared to standard siRNAs, to allow efficient transfection of siRNA into a wide variety of cultured mammalian cells with extremely low cytotoxicity will be described.

11:50

Using siRNAs to Elucidate Gene Function – New Tools and Techniques
Susan Magdaleno, Manager, Scientists – RNAi,  Ambion, An Applied Biosystems Business
One of the most critical aspects of siRNA experimental design is the selection of effective, potent and specific siRNAs for knocking down expression of the target(s) of interest. Presented advances and new tools will simplify the design and execution of RNAi experiments.

12:20

Genome-Wide Functional Analysis of Mitotic Genes in Live Human Cells Using RNAi in Combination with Time-Lapse Microscopy
Beate Neumann, Scientist, European Molecular Biology Laboratory
Results of genome-wide screen including bioinformatics analysis of the mitotic hits in respect to their functions and homologies to other organisms will be presented.

12:50

Lunch Compliments of Qiagen
Followed by Poster Viewing in Exhibition Hall

14:30

Reversible Gene Knockdown in Mice Using a Tight, Inducible shRNA Expression System
Jost Seibler, RNAi Group Leader, Artemis Pharmaceuticals
Artemis Pharmaceuticals have developed a doxycycline inducible system for the temporal control of shRNA expression in mice. A mouse model of reversible insulin resistance was generated. The progression of the disease correlates with the concentration of doxycycline, and the phenotype is reversible shortly after withdrawal of the inductor.

15:00 

MISSIONTM RNAi: Using siRNA and shRNA to Mediate Gene Silencing
Heather Holemon, R&D Principle Investigator, Functional Genomics Group, Sigma-Aldrich
Both siRNA and shRNA are used to address biologically relevant questions and for screening strategies. The presentation will show a proof-of-concept screen using shRNA delivered by lentiviral technology to identify genes that enhance cell sensitivity to the cancer therapy drug, Paclitaxel.

15:30 

Coffee Break and Networking in Exhibition Hall 

16:15

Emerging Themes in RNAi
Chaired by Jens Kurreck, Group Leader, Berlin Free University

16:20

Genome Wide Discovery of Various Classes of Small RNA Using the 454 Sequencing Technology
Peter Matthiesen, Global Marketing Director Genome Sequencing, Roche Applied Science
The Genome Sequencer FLX System from Roche Applied Science: Due to its outstanding combination of read length, throughput and sequence accuracy, a broad variety of break-through sequencing applications can be addressed. Based on its high throughput sequencing technology it is an ideal tool for the identification and characterization of small non coding RNAs or transcripts of unknown function (TUFs) on a genome wide basis.

16:50

Advanced RNAi Technology for Genome-Wide Screening
Bettina Haedrich, Global Product Manager Gene Silencing, QIAGEN
Feedback and recommendations on RNAi screening technologies from QIAGEN’s RNAi High Throughput User Forum will be summarized. Guidelines on best practices are provided which are based on the discussion with a panel of experts in RNAi screening.

17:20

RNA Silencing in Prokaryotes
Volker Patzel, Group Leader, Max Planck Institute for Infection Biology
We developed a technique of siRNA-mediated gene silencing in prokaryotes. This method was used to specifically silence chromosomal and episomal genes in Gram+ and Gram- bacteria as well as in mycobacteria opening promising perspectives regarding validation of prokaryotic gene functions.

17:50 

High Throughput RNAi Screening: Initial Steps Toward Creation of a Functional Map of the Human Genome
Devin Leake, Director of Research & Development, Thermo Fisher Scientific Dharmacon Products
Successful merging of RNAi into high throughput applications requires the identification of critical attributes that distort the outcome of gene silencing studies. To that end, results of a recent multi-laboratory high throughput RNAi phenotypic screen for apoptotic induction and cell viability will be reviewed.

18:20 

Drinks Reception Compliments of Thermo Scientific


Day 2 - 21 September

09:00

RNAi-Based Therapeutics  
Chaired by Judy Lieberman, Senior Investigator, Harvard Medical School and Dmitry Samarsky, RXi Pharmaceuticals

09:05 

Targeting APP with Chemically Modified siRNA (stealthRNAi™) Ameliorates Alzheimer’s Disease Neuropathology in a Transgenic Mouse Model
Xavier de Mollerat du Jeu, Scientist, Invitrogen
The presented results suggest that direct delivery of StealthRNAi™ against APP can specifically reduce neurodegeneration in vivo and indicate that this approach could have potential therapeutic value for the treatment of Alzheimer’s disease. 

09:35 

Silencing HIV-1 with RNA Interference: A Multiple shRNA Approach
Karin von Eije, PhD Student, University of Amsterdam
The future route towards an ex vivo gene therapy for HIV-AIDS patients will be discussed. 

10:05

Coffee Break and Networking in Exhibition Hall 

10:50

Making Therapeutic siRNA a Reality: Recent Advances in siRNA Manufacture
Paul McCormac, Director of Process and Analytical Development, Avecia Biotechnology Inc
This talk will discuss, via the use of a specific case study, recent work that has enabled the scale up of RNA manufacture from 1mmol to 50mmol to an extremely tight timeline.

11:20 

Combination of ckit Gene Silencing and Tocopherol Succinate: A Novel Therapy Against Human Mast Cell Leukaemia
Irene Ruano Solana, PhD Student, Centro de Biología Molecular Severo Ochoa
A combined system, based on down-regulation of ckit signalling pathway and a tocopherol derivate, promotes apoptosis of human mast cell leukaemia and avoids drug treatment disadvantages related to drug resistance development.

11:50

RNAi Therapeutics Against Cancer
Takahiro Ochiya, Head, National Cancer Centre Research Institute
The presented atelocollagen-mediated delivery system holds great potential in significantly advancing the practical application of gene suppression using siRNAs for cancer therapeutics.

12:20 

Lunch Compliments of Qiagen
Followed by Poster Viewing in Exhibition Hall

14:00 

Targeting of the RET/PTC1 Junction Oncogene in Papillary Thyroid Carcinoma by a siRNA Reverses in-vitro and Inhibits in-vivo Tumoural Features
Claude Paul Malvy, Director, Institute for Higher Biomedical Studies (IFSBM)
A siRNA targets the thyroid junction oncogene RET/PTC1 mRNA at the junction. In vitro it triggers the phenotypic reversion of RET/PTC1 dependent cells and vectorized by polymeric nanoparticles inhibits in vivo with specificity the growth of a tumour xenografted with these cells.

14:30

RNAi Intellectual Property Landscape
Kathleen Williams, Co-Chair Life Sciences Practice, Edwards Angell Palmer & Dodge LLP
An overall view of the siRNA landscape and look in particular at specific early filings will be presented. 

15:00

Coffee Break and Poster Award Ceremony 

15:30

The Silent Treatment: Delivering RNA Interference
Judy Lieberman, Senior Investigator, Harvard Medical School
A method for cell-specific systemic delivery of siRNAs by mixing siRNAs with an antibody fragment fused to protamine was designed to silence gene expression in vivo with exquisite specificity in cells bearing the receptor recognized by the antibody. 

16:00

Targeting c-Jun and JunB Proteins as Potential Anti-Cancer Cell Therapy
Marta Izquierdo, Professor of Biochemistry and Molecular Biology, Autonomous University of Madrid
Using melanoma-derived B16-F10 cancer cells the combination of JunB knockdown and c-Jun/JNK inactivation leads to cell cycle arrest and AIF-dependent apoptosis. Inactivation of both, c-Jun and JunB, provides a valuable strategy for anti-tumour intervention. 

16:30

Developing RNAi Therapeutics
Dmitry Samarsky, Vice President, Technology Development, RXi Pharmaceuticals
The design of therapeutic RNAi triggers will be discussed. Also, nanotransporter delivery of RNAi compounds to mouse tissues will be illustrated and silencing of SOD1 in an ALS model demonstrated. Lastly, validation of RIP140 as a target for obesity will be described.

17:00 

Close of Conference