The promises and pitfalls of RNA-interference-based therapeutics.
The promises and pitfalls of RNA-interference-based therapeutics.
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DOI：
10.1038/nature07758
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发表时间：
2009-01-22
期刊：
影响因子：
64.8
通讯作者：
Rossi, John J.
中科院分区：
文献类型：
--
作者：
Castanotto, Daniela;Rossi, John J.
基因表达可以通过小RNA与含有互补序列的信使RNA的沃森-克里克碱基配对来控制-这一过程称为RNA干扰-这一发现显着提高了我们对真核基因调控和功能的理解。短RNA序列调节基因表达的能力为研究基因功能提供了强有力的工具，并将彻底改变疾病的治疗。值得注意的是，尽管距离发现只有十年，但这种现象已经被用于人类临床试验的治疗，专注于基于RNA干扰的治疗方法的生物技术公司已经上市。
The discovery that gene expression can be controlled by the Watson–Crick base-pairing of small RNAs with messenger RNAs containing complementary sequence — a process known as RNA interference — has markedly advanced our understanding of eukaryotic gene regulation and function. The ability of short RNA sequences to modulate gene expression has provided a powerful tool with which to study gene function and is set to revolutionize the treatment of disease. Remarkably, despite being just one decade from its discovery, the phenomenon is already being used therapeutically in human clinical trials, and biotechnology companies that focus on RNA-interference-based therapeutics are already publicly traded.
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