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.
The Thalassemia 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.
ESH Erythro 2025 | An overview of gene therapy approaches in SCD & thalassemia: gene addition versus genome editing
In this video, Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, shares insights into the current state of gene therapy for thalassemia and sickle cell disease (SCD), highlighting the promise of gene addition and genome editing approaches. Prof. Cappellini notes that gene addition has shown convincing results in transfusion-dependent thalassemia, although the procedure is not currently available in Europe due to logistical issues. In contrast, genome editing has shown promise in both diseases, with a reduction in vaso-occlusive crises observed in SCD. This interview took place at the 4th ESH Translational Research Conference on Pathophysiology and Clinical Advances in Sickle Cell Disease and Thalassemia in Dublin, Ireland.
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