For Patients & Families > Specific Diseases > Movement Disorders > RNAi

A completely novel approach to treating a wide range of genetic diseases is emerging from recent research advances using a technique called RNA interference (RNAi). The basic idea behind RNAi, which was Science magazine's Breakthrough of the Year two years in a row, is to shut down, or “silence” the expression of mutant genes to prevent them from doing their dirty work. Some of the most promising research on RNAi as a therapy for neurological disease is being done in models of movement disorders.

RNA molecules form the critical bridge between genes and the activity they generate in cells. Mutant genes, with the assistance of RNA, produce proteins that are in some way toxic to the cell, setting the disease process in motion.

Precisely how RNA facilitates this transfer of DNA bits into protein activity is the focus of Farber Institute scientist Ya-Ming Hou's lab. Hou, an associate professor of biochemistry and molecular pharmacology at Thomas Jefferson University, and her colleagues have contributed fundamental understandings about the structure and function of class of RNAs called transfer RNAs (tRNAs), which are critical to the process.

These and other scientific advances have enabled scientists to create small RNA molecules that can slip through a cell's membrane and interfere with tRNAs, like a well-targeted monkey wrench in the system. Hou's team has created modified tRNA molecules targeted at the gene that causes Huntington's disease. Like other familial movement disorders, the mutation in the Huntington's gene generates toxic proteins that kill motor neurons. Similar techniques are currently being tested in laboratory and animal models of ALS at other labs, with promising preliminary results.