Sickle cell disease is a disabling hemoglobin genetic disease and without efficient treatment so far. Gene therapy could improve this disease by providing a therapeutic gene in hematopoietic stem cells of patients. However, this approach is very complex. It is necessary to optimize the therapeutic gene, the obtaining of the cells to be treated and their delivery.
Our project explores the feasibility of using blood stem cells from umbilical cord to perform the gene therapy for sickle cell disease. Our objectives are to set up cord blood collection for research purposes, to develop an efficient protocol for cord blood stem cell transduction using lentiviral vectors and finally to obtain preclinical data. The project we are carrying out with the Sud-Francilien hospital in Evry, is part of the global program DIM « thérapie génique » to develop new therapeutic strategies for sickle cell disease.
Inserm, Généthon, DIM