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RNA Interference: From Tools to Therapies


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Wednesday, February 3

7:00 AM Registration and Morning Coffee

Plenary Keynote Session

8:10 When Drug Research is Personal

John F. Crowley, Founder, Novazyme Pharmaceuticals, Inc.

Mr. Crowley’s emotion-packed presentation will focus on his personal struggle to find a cure for Pompe disease, a rare and fatal illness that is caused by a defective or missing enzyme. Pompe disease affects fewer than 10,000 people world-wide, including Mr. Crowley’s two small children. Mr. Crowley, a Harvard educated businessman, created and built a pharmaceutical company devoted expressly to finding a cure for the disease. He will detail his journey through the labyrinth of scientific and business fronts, which lead up to a first-round clinical trial.

8:55 Technology, Aging, and the Brain

Gary W. Small, M.D., Parlow-Solomon Professor on Aging, Professor of Psychiatry & Biobehavioral Sciences, Director, UCLA Center on Aging, Director, Memory & Aging Research Center, Director, Geriatric Psychiatry Division, Semel Institute for Neuroscience & Human Behavior, David Geffen School of Medicine at UCLA

New neuroimaging and other technologies are teaching us about how the brain ages and what we can do about it. Although memory declines as we age, medical and nonpharmacological strategies may protect brain health and improve memory performance. At the same time, innovation in digital technology is not only changing the way we live and communicate, it appears to be altering how our brains function. As a consequence of this high-tech stimulation, we are witnessing the beginning of a new form of the generation gap – a brain gap dividing younger digital natives, immersed in the technology early in life, from older digital immigrants, who adapt to the new technology more reluctantly. This lecture will describe this current pivotal point in brain evolution and how we can harness the new technology and lifestyle choices to improve memory and brain function so we can live better and longer.

9:40 Grand Opening Refreshment Break in the Exhibit Hall



11:00 Chairperson’s Remarks

Christophe J. Echeverri, Ph.D., CEO and CSO, Cenix BioScience GmbH

11:10 Genome-wide RNAi Screen in Drosophila Cells Identifies G Protein-coupled Receptor Kinase 2 as an Evolutionarily Conserved Regulator of the NFКB Signaling

Mika Rämet, M.D., Ph.D., Professor of Experimental Pediatric Immunology and Infectious Diseases, University of Tampere, Finland

We have carried out an RNA interference-based genome-wide  in vitro reporter assay screen in   Drosophila for components of NF-КB pathways. We analyzed 16, 025 dsRNA-treatments and identified ten novel NF-КB regulators. Of these, Gprk2 was shown to be evolutionary conserved regulator of NF-КB signalling. siRNA-silencing of human ortholog GRK5 in HeLa cells impaired NF-КB reporter activity. Morpholino-silencing of zebrafish GRK5 homolog in fish embryos caused impaired IL-1β and TNF-α expression after   E. coli infection. Gprk2/GRK5 was identified as an evolutionarily conserved modulator of NF-КB signaling.

11:40 Alexander Bishop, Ph.D., Associate Professor, UT San Antonio


12:10 PM Generation And Integration Of HT-RNAi Screening Data

Pedro Aza-Blanc, Ph.D., Director, Functional Genomics Resources, The Burnham Institute for Medical Research

Sponsored by

12:40 Luncheon Presentation I
Using siRNA to Investigate Non-Coding RNA (ncRNA) Function in Control of Mitosis and Apoptosis in Cells

Susan Magdaleno, Ph.D., Senior Manager, Scientist, RNAi Technologies Research & Development, Applied Biosystems
Long non-coding RNAs (ncRNA) are critical to biology and disease. Life Technologies has now developed a suite of integrated tools and workflows to discover, validate, and knock-down ncRNA which will accelerate understanding of the function of ncRNA in the cell. We will describe the special requirements for using siRNAs to knock down ncRNA and will highlight the application of siRNAs to investigate ncRNA function in regulating mitosis and apoptosis in normal and cancer cells.

1:10 Luncheon Presentation II (Sponsorship Opportunity Available)

1:45 Dessert in the Exhibit Hall



2:15 Chairperson’s Remarks

Paul Kassner, Ph.D., Scientific Director, Lead Discovery, Amgen, Inc.

2:20 Use of Fluorescence Microscopy to Track Protein Localization in siRNA Screening

Paul Kassner, Ph.D., Scientific Director, Lead Discovery, Amgen, Inc.

2:50 Comparative Analysis of RNAi Screening Performance Across Multiple Kinase-Focused Libraries: How Good is a Good Kinase Targeting Sequence?

Hakim Djaballah, Ph.D., Director, HTS Core Facility, Memorial Sloan Kettering Cancer Center

We have assembled and obtained several kinase focused libraries for use in the comparative analysis of RNAi knockdown performance. We have performed a systematic RNAi screening of the Kinome represented in several siRNA and shRNA libraries for gene inactivation that modulate apoptotic events in an isogenic pair of cell lines, namely HeLa/B5 and HeLa/N10. HeLa B5 is stably transfected and over expresses Bcl-XL, a member of the anti-apoptotic Bcl-2 family, whereas the HeLa N10 contains an empty expression vector as a control. We have employed a high content assay measuring real time induction of apoptosis in live cells together with an end point measure of nuclear count and morphological changes post fixation and staining. We will present and discuss our findings.

3:20 Speaker to be Announced

3:50 Sponsored Presentation (Sponsorship Opportunity Available)

4:20 Reception in the Exhibit Hall (Sponsorship Available)

5:20 BREAK-OUT DISCUSSIONS in the Exhibit Hall

Design of HT-RNAi Screens for Target Identification and Validation
Moderators: Paul Kassner, Ph.D., Scientific Director, Lead Discovery, Amgen Inc.
Christopher Miller, Ph.D., Group Director, Applied Genomics, Bristol-Myers Squibb Co.

Topics for discussion:

  • Choice of screening format, libraries and reagents
  • Design of positive and negative controls
  • Addressing the issue of off-target effects
  • Statistical approaches for prioritizing hits

Perspectives on siRNA Delivery Systems

Moderators: Ian MacLachlan, Ph.D., Chief Scientific Officer, Tekmira Pharmaceuticals Inc.
Antonin de Fougerolles, Ph.D., Vice President, Research, Alnylam Pharmaceuticals Inc.

Topics for discussion:

  • Key attributes necessary for delivery
  • Pharmaceutical aspects of siRNA formulations
  • Formulation-specific safety questions
  • Immune activation and RNAi delivery

Progress in Developing RNAi Therapeutics

Moderator: Bob Brown, Ph.D., Senior Vice President, Research, Dicerna Pharmaceuticals Corp.

Topics for discussion:

  • Transitioning from the lab to the clinic
  • Challenges in the clinic
  • RNAi therapies for acute versus chronic conditions
  • Lessons learnt from gene therapy and antisense

6:20 Close of Day


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