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Gene therapy: A promising approach for hereditary haemoglobinopathies

Presented By
Dr Haydar Frangoul, Sarah Cannon Research Institute, USA
Conference
EHA 2021
Gene addition and gene editing approaches for sickle cell disease (SCD) and transfusion-dependent β-thalassaemia (TDT) have shown encouraging results in clinical studies. Long-term follow-up will be required to assess the long-term safety and durability of gene expression. Dr Haydar Frangoul (Sarah Cannon Research Institute, USA) presented gene therapy approaches for 2 haemoglobinopathies caused by mutations: SCD and TDT [1]. SCD is an inherited disorder of haemoglobin affecting 270,000 infants born yearly worldwide, leading to chronic haemolysis and vaso-occlusive crises (VOCs). Patients with SCD have a lower life expectancy (median 48 years) [2]. Current therapy includes supportive care, transfusion, and biologicals. Allogeneic stem cell transplantation is the only curative therapy; however, most of the patients lack an HLA-identical donor. β-thalassaemia affects 40,000 infants born each year, approximately half of them...


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