Silencing Disease Using RNA Interference (RNAi update)

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Journal of American Medicine

Vol. 293, No 11, March 16, 2005

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.