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RNAi for Target Validation and as a Therapeutic (J6)

Organizer(s): John M. Maraganore and Stephen H. Friend
January 28 - February 2, 2007
Keystone Resort  ·  Keystone, Colorado
Abstract Deadline: September 28, 2006
Late Abstract Deadline: October 27, 2006
Scholarship Deadline: September 28, 2006
Early Registration Deadline: November 28, 2006


Sponsored by Alnylam Pharmaceuticals, Inc.

Joint meeting: MicroRNAs and siRNAs: Biological Functions and Mechanisms (J5)
NOTE: Registration for meeting allows attendance at joint meeting (pending space availability).



This meeting took place in the 2007 season.

Listed below are current meetings that are similar to this meeting in nature/content:

For a complete list of the meetings for the upcoming/current season,
see our meeting list, or search for a meeting.
Summary of Meeting
RNA Interference (RNAi) remains a promising new approach for gene silencing both in the context of post-genomic research on target validation and for potential therapeutic applications. As a tool for target validation, synthetic and DNA-based RNAi strategies have been applied in both academia and industry, with significant recent advances in understanding selection of siRNA sequences, optimization for potency and specificity, mechanisms for activation of innate immunity, and vector-based methods for whole-genome siRNA libraries. Several applications of these technologies will be presented. As a therapeutic strategy, synthetic and DNA-based RNAi strategies have advanced to pre-clinical animal models and early clinical testing. These approaches have explored multiple strategies to address the delivery hurdle, and include both local and systemic administration opportunities. The goals of the meeting are to discuss the latest advances in RNAi technology as both a research tool and therapeutic strategy.

Sunday, January 28
3:00 - 7:30 PM Registration Longs Foyer
6:30 - 7:30 PM Refreshments Longs Peak Foyer
7:30 - 7:45 PM Welcoming Remarks
David P. Bartel, Massachusetts Institute of Technology
John M. Maraganore, Alnylam Pharmaceuticals Inc.
Stephen H. Friend, Sage Bionetworks
7:30 - 10:00 PM Opening Session (Joint) Grays Peak/Longs Peak
Victor R. Ambros, University of Massachusetts
Functions of microRNAs in Animal Development
Phillip A. Sharp, Massachusetts Institute of Technology
RNA Biology
Gregory J. Hannon, Cold Spring Harbor Laboratory
Unexpected Complexity in Plant and Animal Small RNAs
Monday, January 29
7:00 - 8:00 AM Breakfast Shavano/Torreys
8:00 - 11:30 AM RNAi for Target Validation: Technologies and Applications I Grays Peak
* Stephen W. Fesik, Vanderbilt University School of Medicine
Peter S. Linsley, Regulus Therapeutics LLC
Insights into the Design of siRNAs for Increased Specificity and Potency
Thijn Brummelkamp, Whitehead Institute for Biomedical Research
Identification of Cancer Relevant Genes using RNAi Library Screens
William S. Marshall, miRagen Therapeutics
The Use of Synthetic siRNA Libraries for Target Validation
Vivek Mittal, Cold Spring Harbor Laboratory
Blocking Angiogenesis-Mediated Tumor Growth and Metastasis by RNAi in Mice
Peng Jin, Emory University School of Medicine
Short Talk: A Small Molecule Enhances RNA Interference and Promotes the Processing of microRNAs
Christophe J. Echeverri, Cenix BioScience GmbH
Short Talk: RNAi-based discovery and validation of novel anti-malarial lead compounds
9:20 - 9:40 AM Coffee Break Longs Peak Foyer
11:45 AM - 1:00 PM Poster Setup Shavano/Torreys
1:00 - 10:00 PM Poster Viewing Shavano/Torreys
4:30 - 5:00 PM Coffee Available Longs Peak Foyer
5:00 - 7:00 PM RNAi for Target Validation: Technologies and Applications II Grays Peak
* Peter S. Linsley, Regulus Therapeutics LLC
Stephen W. Fesik, Vanderbilt University School of Medicine
Cancer Target Identification and Validation with RNAi
Jonathan Hall, Institute of Pharmaceutical Sciences
RNAi in Target Discovery and Validation
Klaus Giese, Silence Therapeutics AG
AtuPLEX a Novel RNAi-Based Delivery System for Cancer Therapies
Jost Seibler, TaconicArtemis
Short Talk: Reversible Gene Knockdown in Mice using a Tight, Inducible shRNA Expression System
7:00 - 8:00 PM Social Hour w/ Lite Bites Shavano/Torreys
7:30 - 10:00 PM Poster Session 1: RNAi Therapeutic Discovery and Development Shavano/Torreys
Tuesday, January 30
7:00 - 8:00 AM Breakfast Shavano/Torreys
8:00 - 11:30 AM Therapeutic RNAi: Direct Delivery Grays Peak
* John J. Rossi, Beckman Research Institute of the City of Hope
Beverly L. Davidson, University of Iowa
The use of Expression-based RNAi to Treat Neurological Disease
Neil Aronin, University of Massachusetts Medical School
Advances in the use of Synthetic siRNAs in the Treatment of Huntington's Disease Models
Ralph A. Tripp, University of Georgia
RNAi as a Therapeutic Strategy for the Treatment of Viral Respiratory Disease
Josephine Lai, University of Arizona
Treating Neuropathic Pain with RNA Interference
Joerg F. Rippmann, Boehringer Ingelheim Pharma GmbH & Co. KG
Short Talk: LXR Alpha Knock Down in an Obese Mouse Model Results in Improved Lipid Parameters but Organ Toxicity
Xavier J. de Mollerat du Jeu, Invitrogen
Short Talk: Targeting APP with Chemically Modified siRNA (stealthRNAi™) Ameliorates Alzheimer’s Disease Neuropathology in a Transgenic Mouse Model
Daniel G. Anderson, Massachusetts Institute of Technology
Short Talk: Combinatorial Libraries of Lipidoids for Systemic siRNA Delivery
9:20 - 9:40 AM Coffee Break Longs Peak Foyer
11:30 AM - 1:00 PM Poster Setup Shavano/Torreys
1:00 - 10:00 PM Poster Viewing Shavano/Torreys
4:30 - 5:00 PM Coffee Available Longs Peak Foyer
5:00 - 7:00 PM Therapeutic RNAi: Systemic Delivery I Grays Peak
* Beverly L. Davidson, University of Iowa
Mark A. Kay, Stanford University
Making Viruses to Attack Other Viruses
John J. Rossi, Beckman Research Institute of the City of Hope
RNAi Based Gene Therapy and the Treatment of HIV Infection
Tracy Zimmermann, Alnylam Pharmaceuticals Inc.
Achieving Therapeutic Gene Silencing in vivo with RNAi
Matthew C. Catley, UCB Celltech
Short Talk: Developing siRNA for Target Validation in the Rat lung: A Rational Approach
7:00 - 8:00 PM Social Hour w/ Lite Bites Shavano/Torreys
7:30 - 10:00 PM Poster Session 2: RNAi Toolkit: Sreening, Viral Vectors, and Delivery Technologies Shavano/Torreys
Wednesday, January 31
7:00 - 8:00 AM Breakfast Shavano/Torreys
8:00 - 11:30 AM Posttrancriptional Silencing Mechanism II (Joint) Grays Peak/Longs Peak
* V. Narry Kim, Seoul National University
Phillip D. Zamore, University of Massachusetts Medical School
RNAi and other Ancient Pathways Programmed by Small RNA
Thomas Tuschl, Rockefeller University
Mechanisms of Small RNA Mediated Mammalian Gene Silencing
Erik Sontheimer, Northwestern University
Biochemical Analysis of RNAi
Witold Filipowicz, Friedrich Miescher Institute for Biomedical Research
Role of Dicer in RNAi/miRNA Pathways and Regulation of miRNA Repression in Mammalian Cells
David P. Bartel, Massachusetts Institute of Technology
MicroRNA Target Recognition
9:20 - 9:40 AM Coffee Break Longs Peak Foyer
11:30 AM - 1:00 PM Poster Setup Shavano/Torreys
1:00 - 10:00 PM Poster Viewing Shavano/Torreys
4:30 - 5:00 PM Coffee Available Longs Peak Foyer
5:00 - 7:00 PM Therapeutic RNAi: Systemic Delivery II Grays Peak
* Judy Lieberman, Harvard Medical School
Akshay Vaishnaw, Alnylam Pharmaceuticals Inc.
Clinical Opportunities for RNAi Therapeutics
Barry A. Polisky, Sirna Therapeutics, Inc.
Advancing RNAi Drugs in the Clinic
C. Frank Bennett, ISIS Pharmaceuticals, Inc.
Oligonucleotide-Based Therapeutics: Oligonucleotides as Drugs
Isidore Rigoutsos, International Business Machines Corporation
Short Talk: Revisiting the Topics of MicroRNA Target Detection and MicroRNA Precursor Discovery
7:00 - 8:00 PM Social Hour w/ Lite Bites Shavano/Torreys
7:30 - 10:00 PM Poster Session 3: RNAi Biology: Mechanism of Action, Structure-Activity Relationship, and Other Topics Shavano/Torreys
Thursday, February 1
7:00 - 8:00 AM Breakfast Shavano/Torreys
8:00 - 11:00 AM MicroRNAs in Development and Disease (Joint) Grays Peak/Longs Peak
* Phillip A. Sharp, Massachusetts Institute of Technology
Elisa Izaurralde, MPI for Developmental Biology
Short Talk: Mechanisms of miRNA-Mediated Gene Silencing
Carlo M. Croce, Ohio State University
MicroRNAs in Cancer
Ronald H. Plasterk, Ministry of Education, Culture and Science
MicroRNAs in Animal Development
Jan Krützfeldt, ETH-Zurich
Therapeutic Strategies using the microRNA Pathway
Anna M. Krichevsky, Brigham and Women's Hospital, Harvard Medical School
MicroRNA in Brain and Brain Tumors
9:20 - 9:40 AM Coffee Break Longs Peak Foyer
4:30 - 5:00 PM Coffee Available Longs Peak Foyer
5:00 - 7:00 PM Therapeutic RNAi: Delivery Technologies Grays Peak
* C. Frank Bennett, ISIS Pharmaceuticals, Inc.
Judy Lieberman, Harvard Medical School
Delivering RNA Interference: Appliations to Viral Infection and Cancer
Dan McCallus, Nucleonics
Gene-Expressed RNAi for Hepatitis B Virus
Patrick Lu, Intradigm Corporation
Nanoparticle Delivery of siRNA for Targeted Therapeutics
Stephen Isaac Rudnick, Fox Chase Cancer Center
Short Talk: Effects of Secondary Structure on Short Interfering RNA and Antisense Oligonucleotide Mediated mRNA Cleavage
7:00 - 8:00 PM Social Hour w/ Lite Bites Shavano/Torreys
8:00 - 11:00 PM Entertainment Shavano/Torreys
Friday, February 2
Departure
*Session Chair   †Speaker invited, not yet responded.



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