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Gene therapy: A promising approach for hereditary haemoglobinopathies

Presented By
Dr Haydar Frangoul, Sarah Cannon Research Institute, USA
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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