TREM-2 AGONISTS FOR THE TREATMENT OF MARFAN SYNDROME

Abstract

Marfan syndrome is caused by mutations in the FBN1 gene (15q21) that codes for Fibrillin-1, an essential connective tissue protein and is a pathology responsible for a high morbidity and mortality. Apart from surgery, treatment options are limited. It is therefore essential to develop new pharmacological approaches to limit aortic dilatation and/or rupture. The inventors have demonstrated a critical role for TREM-2 in the pathophysiology of ascending aortopathy related to Marfan disease. Deletion of TREM-2 indeed aggravates ascending aorta dilation and rupture. Stimulating TREM-2 receptor with peptide or agonistic monoclonal antibody represent a new therapeutic approach for Marfan syndrome.

CPC Classifications