Triggering RNAi with multifunctional RNA nanoparticles and their delivery.
Triggering RNAi with multifunctional RNA nanoparticles and their delivery.
复制标题
用多功能RNA纳米颗粒及其输送触发RNAi。
DOI：
10.1515/rnan-2015-0001
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发表时间：
2015-01
期刊：
影响因子：
--
通讯作者：
Afonin KA
中科院分区：
文献类型：
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作者：
Dao BN;Viard M;Martins AN;Kasprzak WK;Shapiro BA;Afonin KA
蛋白质被认为是我们身体结构、功能和代谢调节的关键参与者。常规疗法中使用的机制通常依赖于用小分子抑制蛋白质，但另一种有希望的治疗疾病的方法是靶向相应的mRNA。1998年，克雷格·梅塞尔和安德鲁·法尔通过RNA干扰或RNAi发现了dsRNA介导的基因沉默。这一发现为新的基因沉默方法带来了几乎无限的可能性，从而为临床医学打开了新的大门。RNAi是一种通过靶向mRNA来抑制基因表达的生物学过程。基于RNAi的治疗剂具有几个潜在的优点：（i）靶向任何基因的先验能力，（ii）相对简单的设计过程，（iii）位点特异性，（iv）效力，和（v）靶向细胞的潜在安全和选择性敲低。然而，问题在于RNAi治疗剂的配制和递送，包括快速排泄、血流中的不稳定性、差的细胞摄取和低效的细胞内释放。为了解决这些问题，正在开发不同类型的RNAi治疗递送策略，包括多功能RNA纳米颗粒。在这篇简短的评论中，我们将简要描述一些当前的方法。
Proteins are considered to be the key players in structure, function, and metabolic regulation of our bodies. The mechanisms used in conventional therapies often rely on inhibition of proteins with small molecules, but another promising method to treat disease is by targeting the corresponding mRNAs. In 1998, Craig Mellow and Andrew Fire discovered dsRNA-mediated gene silencing via RNA interference or RNAi. This discovery introduced almost unlimited possibilities for new gene silencing methods, thus opening new doors to clinical medicine. RNAi is a biological process that inhibits gene expression by targeting the mRNA. RNAi-based therapeutics have several potential advantages (i) a priori ability to target any gene, (ii) relatively simple design process, (iii) site-specificity, (iv) potency, and (v) a potentially safe and selective knockdown of the targeted cells. However, the problem lies within the formulation and delivery of RNAi therapeutics including rapid excretion, instability in the bloodstream, poor cellular uptake, and inefficient intracellular release. In an attempt to solve these issues, different types of RNAi therapeutic delivery strategies including multifunctional RNA nanoparticles are being developed. In this mini-review, we will briefly describe some of the current approaches.