The Non-Malignant Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
The Sickle Cell Disease Channel on VJHemOnc is an independent medical education platform, supported with funding from Agios (Gold). Supporters have no influence on the production of content. The levels of sponsorship listed are reflective of the amount of funding given.
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.
