RNAi Gene Silencing Applications Advance

A Cornucopia of siRNA- and RNAi-based Therapeutics in Development Show Promise

Researchers from academia and industry will present the latest developments in the application of interference RNA (RNAi) as a tool for gene silencing at “RNAi2008 Functions and Applications of Non-coding RNAs” to be held in March in Oxford, U.K. The meeting will focus on advances in the field and current opportunities and challenges in developing RNAi-based strategies for use in understanding the molecular mechanisms underlying disease processes, identifying new drug targets, and developing therapeutic agents capable of silencing disease-related genes.

siRNA Therapeutics

“Refined Animal Models for Optimizing Delivery of Functional siRNAs to Skin,” is the title of the presentation to be given by Roger Kaspar, Ph.D., on behalf of TransDerm (www.transderm.org). The skin disorder on which the company is focusing its initial product development effort is pachyonychia congenital (PC), a rare autosomal dominant disease in which a mutation in the gene for keratin 6a (K6a) causes painful skin lesions to form. The K6a N171 mutation is a single-nucleotide replacement mutation in which an adenine is present in the mutant gene form in place of a cytosine, resulting in an amino acid change.

Dr. Kaspar, CEO of TransDerm, will describe how the company’s TD101 therapeutic small interfering RNA (siRNA) specifically targets the N171K mutant form of the gene without affecting the wild-type gene.

To assess the activity of TD101 and optimize a controlled-dose delivery system, TranDerm is developing transgenic mouse models. In collaboration with Christopher Contag, Ph.D., and colleagues in the molecular imaging program at Stanford University, the company is using molecular imaging in transgenic mice to demonstrate the effectiveness of siRNA knockdown of reporter genes.

http://www.genengnews.com/articles/chitem.aspx?aid=2343&nc=1