GENE THERAPY FOR ADA SCID

Adenosine (ADA)-deficient severe combined immunodeficiency (SCID) was the first inherited disease treated with gene therapy. ADA-SCID patients have an impaired immune system, are highly susceptible to severe infections, and display alterations in several organs. Our studies have provided crucial information on the efficacy and safety of gene therapy approaches with hematopoietic stem cells or peripheral blood lymphocytes using retroviral vectors. The goals of this proposal are to acquire new information on the causes of the immune and organ alterations of this disease, to better understand the functional properties of gene corrected cells and to explore new strategy of gene transfer with lentiviral vectors. These studies will contribute to improve current gene therapy approaches and advance patients’ care.