Brothers in Arms: DNA Enzymes, Short Interfering RNA, and the Emerging Wave of Small-Molecule Nucleic Acid-Based Gene-Silencing Strategies

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Bhindi, Ravinay, Fahmy, Roger J, Lowe, Harry C, Chesterman, Colin N, Dass, Crispin R, Cairns, Murra J, Saravolac, Edward G, Sun, Lun-Quan and Khachigian, Levon M (2007) Brothers in Arms: DNA Enzymes, Short Interfering RNA, and the Emerging Wave of Small-Molecule Nucleic Acid-Based Gene-Silencing Strategies. American Journal of Pathology, 171 (4). pp. 1079-1088. ISSN 0002-9440

Abstract

The past decade has seen the rapid evolution of small-molecule gene-silencing strategies, driven largely by enhanced understanding of gene function in the pathogenesis of disease. Over this time, many genes have been targeted by specifically engineered agents from different classes of nucleic acid-based drugs in experimental models of disease to probe, dissect, and characterize further the complex processes that underpin molecular signaling. Arising from this, a number of molecules have been examined in the setting of clinical trials, and several have recently made the successful transition from the bench to the clinic, heralding an exciting era of gene-specific treatments. This is particularly important because clear inadequacies in present therapies account for significant morbidity, mortality, and cost. The broad umbrella of gene-silencing therapeutics encompasses a range of agents that include DNA enzymes, short interfering RNA, antisense oligonucleotides, decoys, ribozymes, and aptamers. This review tracks current movements in these technologies, focusing mainly on DNA enzymes and short interfering RNA, because these are poised to play an integral role in antigene therapies in the future.

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Item type Article
URI https://vuir.vu.edu.au/id/eprint/3187
DOI https://doi.org/10.2353/ajpath.2007.070120
Official URL http://amjpathol.highwire.org/cgi/content/short/17...
Subjects Historical > FOR Classification > 1103 Clinical Sciences
Historical > FOR Classification > 0601 Biochemistry and Cell Biology
Historical > Faculty/School/Research Centre/Department > School of Biomedical and Health Sciences
Keywords ResPubID18879, small-molecule gene-silencing, gene function, nucleic acid-based drugs, molecular signaling, gene-specific treatments, DNA enzymes, short interfering RNA, antisense oligonucleotides, decoys, ribozymes, aptamers, antigene therapies
Citations in Scopus 110 - View on Scopus
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