The potential of antisense as a CNS therapeutic
| Autor: | Jenny Godfray, Peter Estibeiro |
|---|---|
| Rok vydání: | 2003 |
| Předmět: |
Polymers
Clinical Biochemistry Computational biology Pharmacology Biology Structure-Activity Relationship Drug Delivery Systems Alzheimer Disease Central Nervous System Diseases Drug Discovery Humans Gene Silencing Molecular Structure Delivery vehicle Brain Neoplasms Mental Disorders Oligonucleotides Antisense Oxidative Stress Biodegradation Environmental Drug Design Antisense oligonucleotides Liposomes Nucleic acid Molecular Medicine Target gene Pharmaceutical Vehicles Functional genomics Site of action Central Nervous System Agents |
| Zdroj: | Expert opinion on therapeutic targets. 7(3) |
| ISSN: | 1744-7631 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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