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Keystone Symposium: RNAi for Target Validation and as a Therapeutic

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Keystone Resort, Keystone, CO, U.S.A.
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Sponsored by Alnylam Pharmaceuticals, Inc. 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.