Scientific leader
Dr. Pablo Bartolucci
Sickle cell disease (SCD) is the most prevalent monogenic disorder in France and in the world. SCD-related cerebral vasculopathy (CV), generally appearing during childhood, is responsible for lifelong risk of ischemic strokes with an unclear mechanism. This risk could be partially reduced by chronic blood exchange transfusion. However, some patients are not responder to transfusion, which requires a better understanding of the physiopathology to develop new therapeutic strategies. Among them, gene therapy holds promise for SCD patients as a curative treatment. However, evaluation tools to predict and evaluate the risk of CV after this treatment are lacking.
Therefore, in SCD-related CV field, the multidisciplinary SI-BRAIN project aims to:
- Develop personalized 3D vascular models in silico and 3D-printed
- Determine the CV pathophysiology
- Improve risk assessment for CV
- Estimate the protective effects of gene therapy for SCD-related CV
Laboratory
IMRB (Institut Mondor de Recherche Biomédicale)
5 rue Gustave Eiffel
94 017 Créteil
