The Prospect of Silencing Disease Using RNA Interference
- Premlata Shankar, MD;
- N. Manjunath, MD;
- Judy Lieberman, MD, PhD
- Author Affiliations: CBR Institute for Biomedical Research and Department of Pediatrics, Harvard Medical School, Boston, Mass.
- Corresponding Author: Judy Lieberman, MD, PhD, CBR Institute for Biomedical Research, 200 Longwood Ave, Boston, MA 02115 (lieberman{at}cbr.med.harvard.edu).
Abstract
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.
