Project Overview: Understanding and Targeting Dilated Cardiomyopathy (DCM).
Dilated Cardiomyopathy (DCM) is a severe heart muscle disease characterized by the thinning and stretching of the heart's chambers, leading to impaired pumping of blood through the body. DCM presents a significant health challenge, affecting a substantial portion of the global population, with prevalence estimates reaching as high as 1 in 250 individuals. This disorder, often striking younger adults, may be associated with significant morbidity and mortality, primarily due to heart failure and sudden cardiac death. Despite its impact, key gaps in scientific knowledge exist, which hamper effective management of affected individuals. Firstly, our knowledge of what causes DCM and how genetics plays a role is incomplete, which limits the usefulness of genetic testing in clinical care. Additionally, treatment options for DCM are limited. Current therapies mainly focus on managing symptoms and are not specific to the disease. The only potential cure is a heart transplant, and there are no approved treatments that address the root causes of the condition.
Project Objectives:
The DCM-NEXT project aims at addressing critical gaps in DCM research and clinical care. The project seeks to achieve two primary objectives:
1)Genetic understanding: Enhance our understanding of the genetic factors underpinning DCM, bridging the gap between genetic causes and the clinical expression of the disease. The goal is to improve genetic testing to allow more accurate prediction of disease onset, progression, and the risk of major adverse cardiac events.
2)Therapeutic advancements: Accelerate the development of novel therapies by identifying and validating targets involved in the pathogenesis of DCM. The project aims to move beyond generic treatments that manage symptoms. By deciphering underlying molecular disease mechanisms, it will inform the development of therapies that target mechanisms of disease.
