Supported projects

D201701- Development of new therapeutic strategies in sickle cell disease

Scientific leader
Pr. Pablo Bartolucci, CHU Henri Mondor UPEC

Despite the undeniable progress of gene therapy in recent years, the treatment of sickle cell disease, through this approach, requires key improvements to achieve functional correction of all red blood cells of patients. It is therefore essential to have tools to evaluate the efficiency of gene therapy at the red blood cell level.

This project, in collaboration with teams of Annarita Miccio (Imagine) and Anne Galy (Genethon) aims to invent new analyzes that will better assess the benefits of gene therapy for treated patients. These new techniques include measuring the different types of hemoglobins and evaluating the physical and biological properties of red blood cells. These will be used to monitor patients before and after gene therapy to demonstrate its beneficial effects.

Doc/post-doc
Nicolas Hébert (PhD)
Kim-Anh Nguyen-Peyre (post-doc)

Financement
Etablissement Français du Sang (co-financeur), DIM