要了解和治疗任何以基因为根本原因或诱因的疾病，科学家和临床医师需要使用基因和基因产品的有效方法。许多实验用生物体的基因修正的传统方法需要很高的技术和很多时间。五年前，我们发现一个可以让基因关闭的新机制，叫RNA干扰（RNAi）。另外，在这个令人陶醉的生物过程中，RNAi提供了一个已经改变生物医学研究方向甚至也许能够证明在临床课程中基因干扰的实用性的革命性技术。在此课程中，学生学习RNAi是怎样被发现，怎样工作和它在生理学中可能发挥怎样的现实意义。RNAi是怎样去调节基因表达和实行基因屏蔽，无论在细胞中还是不同生物体上，这个问题都没有得到解答。最后，这个课程审视用RNAi去治疗以动物为实验体的人体疾病模型的首次尝试。

To understand and treat any disease with a genetic basis or predisposition, scientists and clinicians need effective ways of manipulating the levels of genes and gene products. Conventional methods for the genetic modification of many experimental organisms are technically demanding and time consuming. Just over 5 years ago, a new mechanism of gene-silencing, termed RNA interference (RNAi), was discovered. In addition to being a fascinating biological process, RNAi provides a revolutionary technology that has already changed the way biomedical research is done and that may even prove useful for genetic interventions in a clinical context. In this course, students learn how RNAi was discovered, how it works, and what its physiological relevance might be. How RNAi can be harnessed to modulate gene expression and perform genetic screens, both in cells and in various organisms is also covered. Finally, this course examines the first attempts to use RNAi for the treatment of models of human diseases in experimental animals.