Supported projects

D201801 – Study of erythropoiesis and circulating red blood cells in gene therapy for sickle cell disease

Scientific leader
Wassim El Nemer

Erythropoiesis is a complex process that results in the production of red blood cells from stem cells. This process is altered in several human pathologies, causing abnormalities in the circulating blood. This results in anemia, as is the case in thalassemia, or in an increase in blood volume, as is the case in polycythemia. Sickle cell disease is a severe hereditary genetic disease caused by abnormal hemoglobin that causes sickling of red blood cells. It is characterized by severe anemia and stress erythropoiesis that is not well characterized. Our project aims to investigate and better characterize (normal and pathological) erythropoiesis and to determine the effect of therapeutic approaches, primarily gene therapy of sickle cell disease, on erythropoiesis and circulating red blood cell properties. A better characterization of these phenomena will make it possible to optimize the therapeutic approaches improving the effectiveness of the treatments.


Institut National de la transfusion Sanguine



Team 1 UMR_S1134

INTS (Institut National de Transfusion Sanguine)

6 rue Alexandre Cabanel

75015 Paris