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This meeting took place in 2007



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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 USA
Abstract Deadline: Sep 28, 2006
Late Abstract Deadline: Oct 27, 2006
Scholarship Deadline: Sep 28, 2006
Early Registration Deadline: Nov 28, 2006

Sponsored by Alnylam Pharmaceuticals, Inc.


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.

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No registration fees are used to fund entertainment or alcohol at this conference

Conference Program    Print  |   View meeting in 12 hr (am/pm) time


SUNDAY, JANUARY 28

15:00—19:30
Registration

Longs Foyer
18:30—19:30
Refreshments

Longs Peak Foyer
19:30—19:45
Welcoming Remarks

David P. Bartel, HHMI, MIT and Whitehead Institute, USA

John M. Maraganore, Alnylam Pharmaceuticals Inc., USA

Stephen H. Friend, Sage Bionetworks, USA

19:30—22:00
Opening Session (Joint)
Meeting has ended...abstracts no longer viewable online.

Grays Peak/Longs Peak
Victor R. Ambros, University of Massachusetts, USA
Functions of microRNAs in Animal Development

Phillip A. Sharp, Massachusetts Institute of Technology, USA
RNA Biology

Gregory J. Hannon, University of Cambridge, UK
Unexpected Complexity in Plant and Animal Small RNAs


MONDAY, JANUARY 29

07:00—08:00
Breakfast

Shavano/Torreys
08:00—11:30
RNAi for Target Validation: Technologies and Applications I
Meeting has ended...abstracts no longer viewable online.

Grays Peak
* Stephen W. Fesik, Vanderbilt University School of Medicine, USA

Peter S. Linsley, AVI BioPharma, USA
Insights into the Design of siRNAs for Increased Specificity and Potency

Thijn Brummelkamp, Netherlands Cancer Institute, Netherlands
Identification of Cancer Relevant Genes using RNAi Library Screens

William S. Marshall, miRagen Therapeutics, USA
The Use of Synthetic siRNA Libraries for Target Validation

Vivek Mittal, Weill Cornell Medical College, USA
Blocking Angiogenesis-Mediated Tumor Growth and Metastasis by RNAi in Mice

Peng Jin, Emory University School of Medicine, USA
Short Talk: A Small Molecule Enhances RNA Interference and Promotes the Processing of microRNAs

Christophe J. Echeverri, Cenix BioScience GmbH, Germany
Short Talk: RNAi-based discovery and validation of novel anti-malarial lead compounds

08:00—11:00
siRNAs and Chromatin Silencing
Meeting has ended...abstracts no longer viewable online.

Longs Peak
* James C. Carrington, Donald Danforth Plant Science Center, USA

Marjori Ann Matzke, Institute of Plant and Microbial Biology, Academy Sinica, Taiwan
RNA-Directed DNA Methylation: A Versatile Pathway for Transcriptional Gene Silencing

Shiv I. S. Grewal, NCI, National Institutes of Health, USA
RNAi and Heterochromatin Assembly in S. pombe

Steven E. Jacobsen, University of California, Los Angeles, USA
RNAi-mediated Chromatin Silencing in Arabidopsis

Martin A. Gorovsky, University of Rochester, USA
RNAi and Genome Rearrangements in Tetrahymena thermophila

Elissa P. Lei, NIDDK, National Institutes of Health, USA
Short Talk: Identification of Small RNAs Associated with the Gypsy Chromatin Insulator

09:20—09:40
Coffee Break

Longs Peak Foyer
11:45—13:00
Poster Setup

Shavano/Torreys
13:00—22:00
Poster Viewing

Shavano/Torreys
16:30—17:00
Coffee Available

Longs Peak Foyer
17:00—19:00
RNAi for Target Validation: Technologies and Applications II
Meeting has ended...abstracts no longer viewable online.

Grays Peak
* Peter S. Linsley, AVI BioPharma, USA

Stephen W. Fesik, Vanderbilt University School of Medicine, USA
Cancer Target Identification and Validation with RNAi

Jonathan Hall, Eth Zurich, Switzerland
RNAi in Target Discovery and Validation

Klaus Giese, Silence Therapeutics plc, Germany
AtuPLEX a Novel RNAi-Based Delivery System for Cancer Therapies

Jost Seibler, TaconicArtemis, Germany
Short Talk: Reversible Gene Knockdown in Mice using a Tight, Inducible shRNA Expression System

17:00—19:00
Endogenous siRNAs and Silencing II
Meeting has ended...abstracts no longer viewable online.

Longs Peak
* Marjori Ann Matzke, Institute of Plant and Microbial Biology, Academy Sinica, Taiwan

Andrew Fire, Stanford University, USA
Roles of RdRPs in Amplifying the RNAi Response and Distinct Modes of Primary and Secondary siRNA Biogenesis in C. elegans

Sarah C.R. Elgin, Washington University, USA
Heterochromatin Formation and Gene Silencing in Drosophila Melanogaster

Ramin Shiekhattar, University of Miami, USA
Biochemistry of RNAi Effector Complexes

Christophe Antoniewski, Institut Pasteur, France
Short Talk: Using Viral Suppressors of RNA Silencing to Explore the Diversity and Functions of Small RNAs in Drosophila

19:00—20:00
Social Hour with Lite Bites

Shavano/Torreys
19:30—22:00
Poster Session 1: RNAi Therapeutic Discovery and Development

Shavano/Torreys

TUESDAY, JANUARY 30

07:00—08:00
Breakfast

Shavano/Torreys
08:00—11:30
Therapeutic RNAi: Direct Delivery
Meeting has ended...abstracts no longer viewable online.

Grays Peak
* John J. Rossi, Beckman Research Institute of the City of Hope, USA

Beverly L. Davidson, Children’s Hospital of Philadelphia and The University of Pennsylvania, USA
The use of Expression-based RNAi to Treat Neurological Disease

Neil Aronin, University of Massachusetts Medical School, USA
Advances in the use of Synthetic siRNAs in the Treatment of Huntington's Disease Models

Ralph A. Tripp, University of Georgia, USA
RNAi as a Therapeutic Strategy for the Treatment of Viral Respiratory Disease

Josephine Lai, University of Arizona, USA
Treating Neuropathic Pain with RNA Interference

Joerg F. Rippmann, Boehringer Ingelheim Pharma GmbH & Co. KG, Germany
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, Life Techonologies, USA
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, USA
Short Talk: Combinatorial Libraries of Lipidoids for Systemic siRNA Delivery

08:00—11:30
MicroRNA Biogenesis and Function
Meeting has ended...abstracts no longer viewable online.

Longs Peak
* Thomas Tuschl, HHMI/Rockefeller University, USA

V. Narry Kim, Seoul National University, South Korea
Primary microRNA Processing: When Does It Take Place and How Are the Processing Factors Regulated?

James C. Carrington, Donald Danforth Plant Science Center, USA
Small RNA-Directed Pathways in Plants

Richard W. Carthew, Northwestern University, USA
MicroRNAs in Drosophila

Olivier Voinnet, Swiss Federal Institute of Technology, Zürich, Switzerland
Forward Genetic Approach of miRNA Biogenesis and Function in Arabidopsis

Mikiko C. Siomi, University of Tokyo, Japan
Piwi Subfamily Proteins and their Associated Small RNAs in Drosophila Germlines

Eric C. Lai, Sloan-Kettering Institute, USA
Short Talk: Drosophila miR-315 Regulates Wingless Signaling by Inhibiting Axin and Notum

09:20—09:40
Coffee Break

Longs Peak Foyer
11:30—13:00
Poster Setup

Shavano/Torreys
13:00—22:00
Poster Viewing

Shavano/Torreys
14:30—16:30
Workshop 1: miRNA and piRNA Function

Longs Peak
* Elisa Izaurralde, Max Planck Institute for Developmental Biology, Germany

Antonio J. Giraldez, Yale University School of Medicine, USA
Sex, Fish and MicroRNAs: the Regulation of the Germ Line

Chang-Zheng Chen, Stanford University, USA
MiR-181a As a T Cell Sensitivity Rheostat

Trinna Lee Cuellar, Genentech, USA
Development of a Mouse Model to Study Dicer Function in the Brain

Anindya Dutta, University of Virginia, USA
Muscle-Differentiation-Induced MicroRNA miR-206 Promotes Muscle Differentiation and Cell Quiescence by Post-Transcriptional Degradation of Target by mRNAs.

Sergey G. Shpiz, Institute of Molecular Genetics, Russia
RNAi in the Functioning of Drosophila Telomeres

16:30—17:00
Coffee Available

Longs Peak Foyer
17:00—19:00
Therapeutic RNAi: Systemic Delivery I
Meeting has ended...abstracts no longer viewable online.

Grays Peak
* Beverly L. Davidson, Children’s Hospital of Philadelphia and The University of Pennsylvania, USA

Mark A. Kay, Stanford University, USA
Making Viruses to Attack Other Viruses

John J. Rossi, Beckman Research Institute of the City of Hope, USA
RNAi Based Gene Therapy and the Treatment of HIV Infection

Tracy Zimmermann, Alnylam Pharmaceuticals Inc., USA
Achieving Therapeutic Gene Silencing in vivo with RNAi

Matthew C. Catley, AstraZeneca, Sweden
Short Talk: Developing siRNA for Target Validation in the Rat lung: A Rational Approach

17:00—19:00
Posttranscriptional Silencing Mechanism I
Meeting has ended...abstracts no longer viewable online.

Longs Peak
* Phillip D. Zamore, University of Massachusetts Medical School, USA

Craig C. Mello, University of Massachusetts Medical School, USA
RNAi and Related Pathways in C. elegans

David C. Baulcombe, University of Cambridge, UK
ARGONOMICS: The Roles of Argonaute Proteins in RNA Silencing

Xuemei Chen, University of California, Riverside, USA
Role of Methlation in microRNA Metabolism in Plants

Raul Andino, University of California, San Francisco, USA
Short Talk: Characterization of the Drosophila RNAi-based Antiviral Defense Mechanism

19:00—20:00
Social Hour with Lite Bites

Shavano/Torreys
19:30—22:00
Poster Session 2: RNAi Toolkit: Sreening, Viral Vectors, and Delivery Technologies

Shavano/Torreys

WEDNESDAY, JANUARY 31

07:00—08:00
Breakfast

Shavano/Torreys
08:00—11:30
Posttrancriptional Silencing Mechanism II (Joint)
Meeting has ended...abstracts no longer viewable online.

Grays Peak/Longs Peak
* V. Narry Kim, Seoul National University, South Korea

Phillip D. Zamore, University of Massachusetts Medical School, USA
RNAi and other Ancient Pathways Programmed by Small RNA

Thomas Tuschl, HHMI/Rockefeller University, USA
Mechanisms of Small RNA Mediated Mammalian Gene Silencing

Erik Sontheimer, University of Massachusetts Medical School, USA
Biochemical Analysis of RNAi

Witold Filipowicz, Friedrich Miescher Institute for Biomedical Research, Switzerland
Role of Dicer in RNAi/miRNA Pathways and Regulation of miRNA Repression in Mammalian Cells

David P. Bartel, HHMI, MIT and Whitehead Institute, USA
MicroRNA Target Recognition

09:20—09:40
Coffee Break

Longs Peak Foyer
11:30—13:00
Poster Setup

Shavano/Torreys
13:00—22:00
Poster Viewing

Shavano/Torreys
16:30—17:00
Coffee Available

Longs Peak Foyer
17:00—19:00
Therapeutic RNAi: Systemic Delivery II
Meeting has ended...abstracts no longer viewable online.

Grays Peak
* Judy Lieberman, Harvard University, USA

Akshay Vaishnaw, Alnylam Pharmaceuticals Inc., USA
Clinical Opportunities for RNAi Therapeutics

Barry A. Polisky, Sirna Therapeutics, Inc., USA
Advancing RNAi Drugs in the Clinic

C. Frank Bennett, Ionis Pharmaceuticals, Inc., USA
Oligonucleotide-Based Therapeutics: Oligonucleotides as Drugs

Isidore Rigoutsos, International Business Machines Corporation, USA
Short Talk: Revisiting the Topics of MicroRNA Target Detection and MicroRNA Precursor Discovery

17:00—19:00
Structural Biology of RNAi Machinery
Meeting has ended...abstracts no longer viewable online.

Longs Peak
* Xuemei Chen, University of California, Riverside, USA

Dinshaw J. Patel, Memorial Sloan Kettering Cancer Center, USA
Structural Biology of RNA Silencing

Marianthi Kiriakidou, University of Pennsylvania School of Medicine, USA
Short Talk: An mRNA m7G cap Binding Motif within Human Argonaute2 Represses Translation

Javier A. Martinez†, Institute of Molecular Biotechnology of the Austrian Academy of Sciences, Austria
Short Talk: hClp1 is a Novel Human RNA-Kinase that Phosphorylates siRNAs and 3' exons during tRNA Splicing

Renée Schroeder, Max F. Perutz Laboratories, University of Vienna, Austria
Short Talk: Mechanism of Risc-Mediated RNA Recognition and Cleavage is Revealed by Target RNA Requirements

Chris B. Burge, Massachusetts Institute of Technology, USA
Short Talk: Determinants of Targeting by Endogenous and Exogenous MicroRNAs

19:00—20:00
Social Hour with Lite Bites

Shavano/Torreys
19:30—22:00
Poster Session 3: RNAi Biology: Mechanism of Action, Structure-Activity Relationship, and Other Topics

Shavano/Torreys

THURSDAY, FEBRUARY 1

07:00—08:00
Breakfast

Shavano/Torreys
08:00—11:00
MicroRNAs in Development and Disease (Joint)
Meeting has ended...abstracts no longer viewable online.

Grays Peak/Longs Peak
* Phillip A. Sharp, Massachusetts Institute of Technology, USA

Elisa Izaurralde, Max Planck Institute for Developmental Biology, Germany
Short Talk: Mechanisms of miRNA-Mediated Gene Silencing

Carlo M. Croce, Ohio State University, USA
MicroRNAs in Cancer

Ronald H. Plasterk, Ministry of Education, Culture and Science, Netherlands
MicroRNAs in Animal Development

Jan Krützfeldt, ETH-Zurich, Switzerland
Therapeutic Strategies using the microRNA Pathway

Anna M. Krichevsky, Brigham and Women's Hospital, Harvard Medical School, USA
MicroRNA in Brain and Brain Tumors

09:20—09:40
Coffee Break

Longs Peak Foyer
16:30—17:00
Coffee Available

Longs Peak Foyer
17:00—19:00
Therapeutic RNAi: Delivery Technologies
Meeting has ended...abstracts no longer viewable online.

Grays Peak
* C. Frank Bennett, Ionis Pharmaceuticals, Inc., USA

Judy Lieberman, Harvard University, USA
Delivering RNA Interference: Appliations to Viral Infection and Cancer

Dan McCallus, Nucleonics, USA
Gene-Expressed RNAi for Hepatitis B Virus

Patrick Lu, Intradigm Corporation, USA
Nanoparticle Delivery of siRNA for Targeted Therapeutics

Stephen Isaac Rudnick, Fox Chase Cancer Center, USA
Short Talk: Effects of Secondary Structure on Short Interfering RNA and Antisense Oligonucleotide Mediated mRNA Cleavage

17:00—19:00
Silencing RNAs in Genetic Pathways
Meeting has ended...abstracts no longer viewable online.

Longs Peak
* Craig C. Mello, University of Massachusetts Medical School, USA

Nikolaus Rajewsky, Max Delbruck Center for Molecular Medicine, Germany
Natural Selection on microRNA Targets, and Some Other Stories

Jian-Kang Zhu, Purdue University, USA
Stress Regulated Small RNAs and DNA Demethylation in Plants

Kathryn N. Ivey, Tenaya Therapeutics, USA
Short Talk: MicroRNAs Regulate Cardiomyocyte Differentiation from Embryonic Stem Cells

19:00—20:00
Social Hour with Lite Bites

Shavano/Torreys
20:00—23:00
Entertainment

Shavano/Torreys

FRIDAY, FEBRUARY 2

 
Departure


*Session Chair †Invited, not yet responded.



We gratefully acknowledge additional support for this conference from:

Alnylam Pharmaceuticals, Inc.
EMD Millipore

Integrated DNA Technologies Isis Pharmaceuticals, Inc.

We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:



Abbott Laboratories


Isis Pharmaceuticals, Inc.


Thermo Fisher Scientific, Inc.


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Phone:+1 970-262-2690

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Phone:+1 970-262-2676