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Curing Diseases

The importance of RNA for all living beings means that errors in RNA production can cause disease. One such error—a mistake in RNA processing that generates a messenger RNA molecule instructing the cell to make a protein with an aberrant function—underlies preeclampsia. Melissa Moore's laboratory is developing a treatment for this dangerous complication of pregnancy using technologies that promise to destroy the aberrant RNA. For the laboratory of Michael Green, RNA is the tool of choice to find genes that contribute to the development of metastatic cancer. The Green lab uses RNAi, a method of gene silencing whose discovery earned RTI co-director Craig Mello the 2006 Nobel Prize in Medicine or Physiology. RNAi screens in the Green lab may point the way to new strategies for fighting cancer by revealing novel genetic "targets": specific genes against which drugs could be developed to kill cancer cells or stop their spread in the body. In contrast, the Aronin, Czech, Flotte, Xu, and Zamore labs all seek to use RNAi technology to make new RNA-based drugs to fight devastating diseases such as Huntington's disease, diabetes, Alpha-1 antitrypsin deficiency, and ALS by turning off disease causing genes. Other labs seek to understand the role of RNA in viral diseases, such as AIDS, which is caused by the HIV virus whose genetic material is made of RNA rather than DNA. Finally, the laboratory of Victor Ambros seeks to detect disease by looking for specific sets of RNA molecules outside of cells, in blood for example, long before current methods can alert physicians that something has gone wrong.