A screen for drug leads for the treatment of Barth syndrome

The objective of the project is to identify drug leads for the treatment of Barth syndrome. Barth syndrome is an inherited disease that results in death during childhood due to heart failure. The type of heart problem inherited is called 'cardiomyopathy.' This term basically means that over time the heart does not pump blood very well. Hearts of Barth patients are also prone to irregular heart beats called arrhythmias. The hearts of Barth syndrome patients gradually weaken to a point where either a major cardiac event occurs. There is currently no treatment for Barth syndrome. … In 1996 the human gene that causes Barth syndrome, TAZ (formerly called G4.5) was discovered. This discovery allowed for the development of various 'models' of Barth syndrome whereby the Barth syndrome gene is inactivated in 'model organisms'. Study of these model organisms which include yeast, fruit fly, zebrafish, and most recently mouse, has led to many breakthroughs in our knowledge about Barth syndrome. In this grant we propose to use the yeast model of Barth syndrome to identify drugs leads that improve their health and fitness as a first step toward the identification of a drug for the treatment of Barth syndrome.