CORDIS Project
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This project aims to improve gene therapy for β-thalassemias and sickle cell disease by enhancing the homing and engraftment of modified hematopoietic stem cells. By manipulating the expression of the CXCR4 receptor, it seeks to increase the efficiency of treatment using autologous stem cell transplantation.
β-thalassemias and sickle cell disease (SCD) are caused by mutations in the β-globin gene (HBB) that result in the defective synthesis (β-thalassemias) or the production of an abnormal variant (SCD) of hemoglobin.
Currently, the only curative option is allogeneic transplantation of hematopoietic stem cells (HSCs), but it is severely limited by conditioning toxicity and the restricted availability of compatible donors.
Alternative strategies, based on lentiviral vector (LV)-mediated introduction…
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
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