Applying small RNA molecules to the directed treatment of human diseases: realizing the potential

Authors: Pappas, Todd C¹; Bader, Andreas G¹; Andruss, Bernard F¹; Brown, David¹; Ford, Lance P²

Source: Expert Opinion on Therapeutic Targets, Volume 12, Number 1, January 2008 , pp. 115-127(13)

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Abstract:

Small interfering RNAs (siRNA) and microRNAs (miRNA) are gaining considerable attention in the pharmaceutical and biotechnology industries, as research has revealed their likely clinical and agricultural applications. The capacity of siRNAs to dramatically and specifically reduce the expression of targeted genes has spawned multiple clinical trials to establish the therapeutic potential of small RNAs targeting viral, cancer and other disease-related genes. The successful application of siRNAs will enable the development of therapeutic applications based on miRNAs that have been observed to contribute to a variety of human diseases. This article reviews advances that have been made to apply small RNAs as therapeutics.

Keywords: delivery; microRNA; RNA drugs; small interfering RNA; therapeutics

Document Type: Research article

DOI: http://dx.doi.org/10.1517/14728222.12.1.115

Affiliations: 1: 1Asuragen, Inc., 2150 Woodward Street, Suite 100, Austin, TX 78744, USA, Email: dbrown@asuragen.com 2: 2BIOO Scientifi c, 3913 Todd Lane, Suite 312, Austin, TX 78739, USA, Email: lford@biooscientific.com

Publication date: 2008-01-01

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