Triggerable RNA nanodevices.

RNA & disease (Houston, Tex.) Pub Date : 2017-01-01 Epub Date: 2017-06-21
Justin Halman, Emily Satterwhite, Jaclyn Smollett, Eckart Bindewald, Lorena Parlea, Mathias Viard, Paul Zakrevsky, Wojciech K Kasprzak, Kirill A Afonin, Bruce A Shapiro
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引用次数: 0

Abstract

The targeted and conditional activation of pharmaceuticals is an increasingly important feature in modern personalized medicine. Nucleic acid nanoparticles show tremendous potential in this exploit due to their programmability and biocompatibility. Among the most powerful nucleic acid specific treatments is RNA interference-based therapeutics. RNA interference is a naturally occurring phenomenon in which specific genes are effectively silenced. Recently we have developed two different strategies based on customized multivalent nucleic acid nanoparticles with the ability to conditionally activate RNA interference in diseased cells as well as elicit detectable fluorescent responses.[1,2] These novel technologies can be further utilized for the simultaneous delivery and conditional intracellular activation of multiple therapeutic and biosensing functions to combat various diseases.

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可触发的 RNA 纳米器件。
有针对性和有条件地激活药物是现代个性化医疗中一个日益重要的特征。核酸纳米粒子因其可编程性和生物相容性,在这方面显示出巨大的潜力。基于 RNA 干扰的疗法是最强大的核酸特异性疗法之一。RNA 干扰是一种有效抑制特定基因的自然现象。最近,我们基于定制的多价核酸纳米粒子开发出了两种不同的策略,它们能够有条件地激活病变细胞中的 RNA 干扰,并引起可检测的荧光反应。
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