The Prospect of Silencing Disease Using RNA Interference
Premlata Shankar, MD;
N. Manjunath, MD;
Judy Lieberman, MD, PhD
JAMA. 2005;293:1367-1373.
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.
Author Affiliations: CBR Institute for Biomedical Research and Department of Pediatrics, Harvard Medical School, Boston, Mass.
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