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The potential of antisense as a CNS therapeutic.

机译:反义作为中枢神经系统治疗剂的潜力。

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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.
机译:反义提供了敲低靶基因表达的精确和特异性方法,并且是神经科学和其他领域研究的主要重点。它已在基因功能和靶标验证研究中用作工具,并正独立地作为一种治疗技术出现。然而,变得越来越明显的是,在反义可以有效地用于CNS中之前,有许多障碍需要克服,最显着的是找到合适的核酸化学物质和有效的转运载体来将反义寡核苷酸(AS-ODN)通过血脑屏障(BBB)的作用部位。尽管存在这些问题,但已经在体外和某些情况下在体内验证了AS-ODN在CNS治疗中的许多潜在应用。在这里,作者概述了可用的核酸化学,并回顾了可应用于CNS治疗剂的非侵入性运载工具的开发进展。除此之外,他们讨论了AS-ODN在CNS研究中的许多实验应用,并推测了反义技术治疗CNS疾病的发展。

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