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Best Practices in Gene Therapy for Sickle Cell Disease and Transfusion-dependent β-Thalassemia

7 Mar 2025

Abstract excerpt

Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are inherited blood disorders caused by pathogenic variants of the β-globin gene. Historically, allogeneic hematopoietic stem cell transplantation (HSCT) from human leukocyte antigen (HLA)-matched donors has been the only curative option. However, as most patients with SCD or TDT lack HLA-matched donors, autologous or patient-derived HSCT can...

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PMID: 40058646
DOI: 10.1016/j.jtct.2025.02.025

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