CORDIS Project
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The project aims to develop safe and effective gene-editing strategies for treating beta-thalassemia and sickle cell disease. By using targeted base-editing techniques, it seeks to correct genetic mutations and enhance fetal hemoglobin production without causing harmful DNA breaks.
Beta-thalassemia and sickle cell disease (SCD) are caused by mutations affecting the synthesis or the structure of the adult hemoglobin (Hb) beta-chain.
The only definitive cure is transplantation of allogeneic hematopoietic stem cells (HSCs) from an HLA-matched donor, an option available to <30% of the patients.
The clinical severity of beta-hemoglobinopathies is alleviated by the co-inheritance of mutations causing expression of fetal gamma-globin in adult life - a condition termed hereditary…
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
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