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消除障碍:小干扰RNA递送技术的进展

Knocking down barriers: advances in siRNA delivery.

作者信息

Whitehead Kathryn A, Langer Robert, Anderson Daniel G

机构信息

Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts 02142, USA.

出版信息

Nat Rev Drug Discov. 2009 Feb;8(2):129-38. doi: 10.1038/nrd2742.

Abstract

In the 10 years that have passed since the Nobel prize-winning discovery of RNA interference (RNAi), billions of dollars have been invested in the therapeutic application of gene silencing in humans. Today, there are promising data from ongoing clinical trials for the treatment of age-related macular degeneration and respiratory syncytial virus. Despite these early successes, however, the widespread use of RNAi therapeutics for disease prevention and treatment requires the development of clinically suitable, safe and effective drug delivery vehicles. Here, we provide an update on the progress of RNAi therapeutics and highlight novel synthetic materials for the encapsulation and intracellular delivery of nucleic acids.

摘要

自RNA干扰(RNAi)这一获得诺贝尔奖的发现以来的十年间,数十亿美元已投入到人类基因沉默治疗应用的研究中。如今,在治疗年龄相关性黄斑变性和呼吸道合胞病毒的临床试验中已有颇具前景的数据。然而,尽管取得了这些早期成功,但要将RNAi疗法广泛用于疾病预防和治疗,仍需要开发出临床适用、安全有效的药物递送载体。在此,我们提供RNAi疗法的进展更新,并重点介绍用于核酸封装和细胞内递送的新型合成材料。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d45c/7097568/465d533355c7/41573_2009_Article_BFnrd2742_Fig1_HTML.jpg

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