RNA干扰
基因沉默
临床试验
小干扰RNA
药物发现
医学
生物信息学
生物
核糖核酸
计算生物学
基因
遗传学
作者
Kathryn A. Whitehead,Robert Langer,Daniel G. Anderson
摘要
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.
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