Contempo Updates Section Editor: Sarah Ringold,
MD, Fishbein Fellow.
Author Affiliations: CBR Institute for Biomedical
Research and Department of Pediatrics, Harvard Medical School, Boston, Mass.
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.
Shankar P, Manjunath N, Lieberman J. The Prospect of Silencing Disease Using RNA Interference. JAMA. 2005;293(11):1367-1373. doi:10.1001/jama.293.11.1367