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Isis Pharmaceuticals Demonstrates The Potential Of Single-Stranded RNA-Like Antisense Technology To Activate The RNAi Pathway
Date:8/30/2012

CARLSBAD, Calif., Aug. 30, 2012 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) announced today the publication of two papers in the journal Cell demonstrating that single-stranded short interfering RNA (ss-siRNA) molecules distributed broadly, activated the RNA interference (RNAi) pathway and reduced expression of targeted genes in animal models.  The RNAi pathway is a mechanism that can be used to inhibit expression of genes.  The RNAi pathway has typically been engaged by short double-stranded RNAs (dsRNA).  Once engaged, the RNAi mechanism removes the 'sense' strand of the dsRNA leaving a functional 'antisense' single-stranded RNA to bind to the target mRNA and cause degradation.  In these studies, Isis scientists and collaborators demonstrated that ss-siRNA molecules can effectively trigger the RNAi pathway and selectively inhibit the expression of a targeted gene. 

In the published study titled 'Single-stranded siRNAs activate RNAi in animals', Isis scientists evaluated the absorption, distribution, metabolism and activity of the ss-siRNAs designed to utilize the RNAi pathway.   These data show that single-stranded RNA-like antisense oligonucleotides are broadly distributed to and active in multiple organs and effectively engage and trigger the RNAi pathway without the need for complex formulations or delivery vehicles typically necessary for double-stranded RNAi molecules. 

"Double-stranded siRNA drugs require complex formulations to achieve sufficient delivery for systemic activity.  This requirement has severely limited the development of safe and effective drugs that work through the RNAi pathway.  In contrast, single-stranded RNA-like antisense drugs can be administered subcutaneously and distribute to tissues without the need for formulations.  In addition, approaching RNAi with singl
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SOURCE Isis Pharmaceuticals, Inc.
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