The potential of antisense as a CNS therapeutic

Expert Opinion on Therapeutic Targets
J Godfray, P Estibeiro

Abstract

Antisense offers a precise and specific means of knocking down expression of a target gene, and is a major focus of research in neuroscience and other areas. It has application as a tool in gene function and target validation studies and is emerging as a therapeutic technology in its own right. It has become increasingly obvious, however, that there are a number of hurdles to overcome before antisense can be used effectively in the CNS, most notably finding suitable nucleic acid chemistries and an effective delivery vehicle to transport antisense oligonucleotides (AS-ODNs) across the blood-brain barrier (BBB) to their site of action. Despite these problems, a number of potential applications of AS-ODNs in CNS therapeutics have been validated in vitro and, in some cases, in vivo. Here the authors outline available nucleic acid chemistries and review progress in the development of non-invasive delivery vehicles that may be applicable to CNS therapeutics. Further to this, they discuss a number of experimental applications of AS-ODNs to CNS research and speculate on the development of antisense techniques to treat CNS disease.

References

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Citations

Aug 28, 2003·Current Opinion in Chemical Biology·Christopher P Austin
Jun 26, 2009·Expert Opinion on Drug Delivery·Xiubo ZhaoJian R Lu
Dec 19, 2014·Angewandte Chemie·Korbinian BrunnerThomas Carell
Oct 1, 2004·Drug Discovery Today. Technologies·Jenny GodfrayPeter Estibeiro
May 4, 2004·Molecular and Cellular Neurosciences·Stefan IsenmannAlexandra Kretz
Jul 21, 2012·Journal of the American Chemical Society·Julian WillibaldThomas Carell

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