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The RNA drug revolution -- a new approach to gene therapy
Date:1/23/2008

New Rochelle, NY, January 23, 2008RNA interference (RNAi) represents an innovative new strategy for using small RNA molecules to silence specific genes associated with disease processes, and a series of review articles describing the state-of-the-art and potential therapeutic applications of RNAi and microRNAs will begin with two review papers in the January 2008 issue (Volume 19, Number 1) of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. The papers are available free online.

At least six clinical trials using RNA interference (RNAi) have been approved, with many more coming down the pipeline, according to the Editorial by Mark A. Kay, MD, PhD, an Associate Editor of Human Gene Therapy and the Dennis Farrey Family Professor in Pediatrics and Professor of Genetics at Stanford University School of Medicine. One thing is clear, adds Kay, small RNAs as a therapeutic platform are here to stay.

The excitement surrounding RNAi and the two main approaches to delivering RNA-based therapeuticsas mature siRNA molecules or as short hairpin RNAs (shRNAs)relates to the discovery of native microRNA molecules in human cells and their intrinsic ability to block the expression of a target gene. siRNA therapeutic strategies in development aim to harness the cells natural RNAi pathway and specifically silence a mutant or dysregulated gene.

Traditionally, gene therapy has focused on supplying a normal copy of a faulty gene, whereas RNAi turns off a problematic gene. These contrasting approaches share some of the same techniques and challenges, including delivery of a therapeutic gene or siRNA into cells.

Human Gene Therapy will publish a series of review papers in four consecutive issues focusing on a range of topics related to therapeutic applications of small RNAs. The series begins in the January issue with a paper entitled, Behind the Scenes of a Small RNA Gene-Silencing Pathway, by Gregory Ku, MD, PhD, and Mich
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Contact: Vicki Cohn
vcohn@liebertpub.com
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert

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