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ASH 2021 | Gene therapy is the future of sickle cell disease

Abdullah Kutlar, MD, Augusta University, Augusta, GA, shares an overview of exciting gene therapy-based approaches being assessed as treatment options for patients with sickle cell disease (SCD). LentiGlobin gene therapy is at the most advanced stage of development, currently under investigation in an ongoing Phase III trial. The approach aims to add functional copies of a modified form of the β-globin gene into a patient’s hematopoietic stem cells. BCL11A knockout and CRIPSR/Cas9 gene editing are other promising approaches, but clinical data is needed. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.