利用RNA干扰使疾病沉默的前景。
The prospect of silencing disease using RNA interference.
作者信息
Shankar Premlata, Manjunath N, Lieberman Judy
机构信息
CBR Institute for Biomedical Research and Department of Pediatrics, Harvard Medical School, Boston, Mass 02115, USA.
出版信息
JAMA. 2005 Mar 16;293(11):1367-73. doi: 10.1001/jama.293.11.1367.
The discovery of RNA interference (RNAi), an endogenous cellular gene-silencing mechanism, has already provided a powerful tool for basic science researchers to study gene function. The subsequent finding that RNAi also operates in mammalian cells has generated excitement regarding potential therapeutic applications. In this article we discuss the basic mechanism of RNAi and the therapeutic opportunities and obstacles for harnessing RNAi for therapy of human disease.
RNA干扰（RNAi）这一内源性细胞基因沉默机制的发现，已经为基础科学研究人员提供了一个研究基因功能的强大工具。随后发现RNAi也在哺乳动物细胞中发挥作用，这引发了人们对其潜在治疗应用的兴奋之情。在本文中，我们将讨论RNAi的基本机制以及利用RNAi治疗人类疾病的治疗机会和障碍。
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