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Chem Biol. 2012 Aug 24;19(8):937-54. doi: 10.1016/j.chembiol.2012.07.011.

Designing chemically modified oligonucleotides for targeted gene silencing.

Author information

1
Department of Chemistry, McGill University, 801 Sherbrooke Street West, Montréal, QC H3A 0B8, Canada. glen.deleavey@mail.mcgill.ca

Abstract

Oligonucleotides (ONs), and their chemically modified mimics, are now routinely used in the laboratory as a means to control the expression of fundamentally interesting or therapeutically relevant genes. ONs are also under active investigation in the clinic, with many expressing cautious optimism that at least some ON-based therapies will succeed in the coming years. In this review, we will discuss several classes of ONs used for controlling gene expression, with an emphasis on antisense ONs (AONs), small interfering RNAs (siRNAs), and microRNA-targeting ONs (anti-miRNAs). This review provides a current and detailed account of ON chemical modification strategies for the optimization of biological activity and therapeutic application, while clarifying the biological pathways, chemical properties, benefits, and limitations of oligonucleotide analogs used in nucleic acids research.

PMID:
22921062
DOI:
10.1016/j.chembiol.2012.07.011
[Indexed for MEDLINE]
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