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Abstract

Grant Number:	4R44CA097898-02
PI Name:	LEWIS, DAVID L.
PI Email:	david.lewis@genetransfer.com
PI Title:	SENIOR SCIENTIST
Project Title:	siRNA for Drug Target Validation in Mammalian Cells

Abstract: DESCRIPTION (provided by applicant): The tremendous progress made in the medical sciences and genomics has given rise to a plethora of new candidate drug targets. However, it is becoming increasingly apparent that a major bottleneck in the drug discovery process is not always drug design per se, but rather identification of which of the candidate targets most contributes to the particular diseased state. Recently, a revolutionary new method termed RNA interference (RNAi) has been shown to be highly effective at inhibiting the expression of specific target genes in mammalian cells in vitro. The overall objective of the studies proposed in this SBIR Fast Track grant application is to develop the use of RNAi for the purposes of drug target validation in mammalian cells in vitro and in vivo. In Phase I studies, we will develop reagents for delivery of siRNA and explore the utility of siRNA for inhibiting reporter gene expression in mammalian cells in vitro. In Phase II studies, we will apply RNAi technology to components of the insulin signal transduction pathway as a proof-of-principle test. We will also explore the use of RNAi technology to inhibit expression of genes in small animals in order to validate drug targets in vivo.

Thesaurus Terms:
RNA, drug discovery /isolation, gene expression, reporter gene, technology /technique development
cytokine, drug delivery system, drug metabolism, gene targeting
laboratory mouse

Institution:	MIRUS CORPORATION
	505 S ROSA RD, #104
	MADISON, WI 53711
Fiscal Year:	2003
Department:
Project Start:	01-AUG-2002
Project End:	30-JUN-2005
ICD:	NATIONAL CANCER INSTITUTE
IRG:	ZRG1

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