TY - JOUR AU - Finotti, Alessia AU - Borgatti, Monica AU - Gambari, Roberto PY - 2016 TI - Ground state naïve pluripotent stem cells and CRISPR/Cas9 gene correction for β-thalassemia JF - Stem Cell Investigation; Vol 3, No 10 (October 2016): Stem Cell Investigation Y2 - 2016 KW - N2 - 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 (HbA) (1-3). Together with sickle cell anemia (SCA), thalassemia syndromes are among the most impactful diseases in developing countries, in which the lack of genetic counselling and prenatal diagnosis have contributed to the maintenance of a very high frequency in the population. The management of β-thalassemia patients is mostly based on blood transfusion, chelation therapy and, alternatively, on bone marrow transplantation (2). Recently, novel therapeutic options have been explored, such as gene therapy (3) and fetal hemoglobin (HbF) induction (4). UR - https://sci.amegroups.org/article/view/12099