Supported projects

D201901 – Development of an in vitro model for cerebral vasculopathy monitoring in gene therapy of sickle cell disease

Sickle cell disease (SCD) is the most prevalent and severe inherited monogenic disorder which results from a mutation in the b-globin gene. Gene therapy shows promise for treating SCD patients as a definitive therapeutic strategy. Cerebral vasculopathy including arteriopathy appears in childhood and is responsible for ischemic stroke, making SCD the first etiology of stroke in children as well as in young adults.

Therefore, these complications are among the selection criteria to enroll patients in gene therapy trials. To monitor the cerebral vasculopathy in SCD patients received gene therapy in order to estimate the impact of this treatment, we develop an in vitro model by mathematical modelling followed by 3D-printing of patients’ carotid from MRI and importing their Doppler parameters.