Review

Gene Therapy (2006) 13, 496–502. doi:10.1038/sj.gt.3302654; published online 29 September 2005

Anti-miRNA oligonucleotides (AMOs): ammunition to target miRNAs implicated in human disease?

J Weiler1, J Hunziker1 and J Hall1

1Novartis Institutes for BioMedical Research, Genome and Proteome Sciences, Basel, Switzerland

Correspondence: Dr J Weiler, Novartis Institutes for BioMedical Research, Genome and Proteome Sciences, CH-4002 Basel, Switzerland. E-mail: jan.weiler@novartis.com

Received 29 June 2005; Revised 16 August 2005; Accepted 25 August 2005; Published online 29 September 2005.

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Abstract

MicroRNAs (miRNAs) are endogenous 19–25 nucleotide RNAs that have recently emerged as a novel class of important gene-regulatory molecules involved in many critical developmental and cellular functions. miRNAs have been implicated in the pathogenesis of several human diseases, such as neurodegenerative disorders, cancer, and more recently in viral and metabolic diseases. Unraveling the roles of miRNAs in cellular processes linked to human diseases will lead to novel opportunities for the regulation of protein function and will help to evaluate their potential for therapeutic intervention. Approaches to interfere with miRNA function in vitro and in vivo based on synthetic anti-miRNA oligonucleotides (AMOs) are discussed in this review.

Keywords:

miRNA, microRNA, RNAi, antisense oligonucleotides, anti-miRNA oligonucleotide, AMO

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