Ground state naïve pluripotent stem cells and CRISPR/Cas9 gene correction for β-thalassemia
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The β-thalassemias are a group of hereditary diseases caused by more than 300 mutations of the adult β-globin gene, leading to low or absent production of adult hemoglobin. The management of β-thalassemia patients is mostly based on blood transfusion, chelation therapy and, alternatively, on bone marrow transplantation. This review summurize novel therapeutic options which have been explored, such as gene therapy and fetal hemoglobin induction.