Once formed, no treatment is currently available to revert aneurysm other than surgery. Because we do not have the sufficient knowledge to understand why aneurysm is formed in the first place, the present proposal aims at understanding the molecular mechanism of aneurysm formation and prevention. What is not generally know is that aneurysm contributes more significantly to mortality in patients with abnormal function of cilia than in general population (11.7% versus 1.0%). We therefore propose a new model system of cilia to study aneurysm. We also propose a candidate drug that could improve cilia function. Of note is that no drug is currently available on the market to prevent aneurysm formation, although various anti-hypertensive drugs have been suggested to prevent aneurysm rupture. In addition, there is currently no specific cilia therapy available. We are therefore hopeful that our studies will offer the very first therapeutic target to the cilia and to prevent aneurysm formation. We propose to target a drug to a specific part of a cell; i.e. the cilium. Overall, our proposed studies will examine important dogmas of aneurysm, and it will pave our path in the search for personalized medicine for ciliopathic patients with aneurysm.
|Program type||Innovative Project Award|
|Effective start/end date||07/01/2019 → 06/30/2021|