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Development of a new RNAi therapeutics platform
J. Kamens, W. Salomon, H. Baigude, J. McCarroll, C.-S. Yang, P. Swain, T. Rana, T. Woolf, D. Samarsky, P. Pavco RXi Pharmaceutical, US
Keywords: obesity, ALS, SOD1, RIP140
Abstract: Parameters for the design of therapeutic synthetic RNAi triggers will be discussed, and the data will be presented demonstrating the delivery of active RNAi compounds to the mouse liver and some other tissues with nanotransporters. RNAi compounds are readily formulated with the nanotransporters and activity is obtained at relatively low doses. Data will also be presented demonstrating the knock down of SOD1 in the spinal column in a mouse ALS model. Lastly, the RNAi validation of RIP140 as a target gene for obesity will be described.
Nanotech 2008 Conference Program Abstract
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