MDA awarded a grant totaling $282,630 to Albena Jordanova, professor in the department of genetics at the University of Antwerp, Belgium, for research into the molecular causes of, and potential drug targets for, a recently discovered form of Charcot-Marie-Tooth (CMT) disease known as dominant intermediate CMT type C (DI-CMTC).
Jordanova's research team was the first to describe the new CMT type and showed that the disease is caused by different mutations (genetic flaws) in the gene that carries instructions for an enzyme called tyrosyl-tRNA synthetase (YARS).
In their new work, Jordanova and colleagues aim to identify genes that modify the neurodegenerative symptoms of DI-CMTC present in a fruit fly research model of the disease. The team will then test the modifying genes to determine how well each is able to interact with drug-like chemical compounds, in an effort to pinpoint the best drug targets for DI-CMT and other types of the disease.
The "best gene hits" will be those that carry instructions for proteins that are both structurally favorable — that is, their protein folds are amenable to interactions with drug-like chemical components — and functionally favorable with a proven ability to affect the DI-CMTC disease process.
"MDA funding is absolutely essential to start this work," Jordanova said. "This is important seeding money for us to initiate a midterm research program and to perform the research we dream of."
Funding for this MDA grant began August 1, 2010.
Muscular Dystrophy Association — USA
222 S. Riverside Plaza, Suite 1500
Chicago, Illinois 60606
The Muscular Dystrophy Association (MDA) is a qualified 501(c)(3) tax-exempt organization.
©2015, Muscular Dystrophy Association Inc. All rights reserved.