A Healthy Nervous System A Delicate Balance

Description: Huda A. Zoghbi, Howard Hughes Medical Institute Investigator, discusses her work in discovering the culprit in a disorder known as spinocerebellar ataxia type 1. Dr. Zoghbi found a genetic stutter that increases the size of a particular gene, disrupting neurons controlling movement. Researching compounds to address this genetic flaw could lead to treatments for neurodegenerative diseases and change the way we treat patients who have had mutations in critical genes that can slowly destroy the nervous system, rendering these patients unable to talk, swallow or breathe. Dr. Zoghbis work opens up the pathway to a new level of treatment.