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Validating rnai targets

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RNA interference (RNAi) is being commonly used as a screening tool for identifying and validating potential drug targets, exploring unknown cellular pathways, and for performing whole-genome functional screens.The screens developed, using both small interfering RNA (si RNA) and short hairpin RNA (sh RNA), are now fairly robust and sensitive and can be performed in a reliable and high-throughput fashion.

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It is imperative to select a relevant 3D model when performing high-throughput screens.Given that micro RNAs select their targets by nucleotide base-pairing, it follows that it should be possible to find micro RNA targets computationally.There has been considerable progress, but assessing success and biological significance requires a move into the 'wet' lab.There is growing interest in combining RNAi screens with gene editing, chemical genomics, overexpression studies and phenotypic screens, which will all be discussed here.Screening experts from industry and academia will share their experiences leveraging the utility of these diverse screening platforms for a wide range of applications.Final Agenda Day 1 | Day 2 | Download Brochure Tuesday, September 22 am Registration and Morning Coffee EXPLOITING NEW SCREENING PARADIGMS Chairperson’s Opening Remarks Scott Martin, Ph.D., Group Lead, Functional Genomics, Genentech, Inc. Harnessing High-Throughput RNAi for Target Identification Utilizing 3D Cell Culture Models Geoffrey Bartholomeusz, Ph.D., Associate Professor and Director, si RNA Core Facility, Department of Experimental Therapeutics, Division of Cancer Medicine, The University of Texas M. Anderson Cancer Center It is now well accepted that 3D cell culture models although varied and complex, replicate clinically relevant outcomes associated with the tumor microenvironment.Using a robust self-assembly strategy, we develop a unique nanoparticle (NP) platform composed of a solid polymer/cationic lipid hybrid core and a lipid-poly(ethylene glycol) (lipid-PEG) shell for systemic si RNA delivery.The new generation lipid–polymer hybrid NPs are small and uniform, and can efficiently encapsulate si RNA and control its sustained release.