2025 CASH丨Dr. Yongrong Lai: Advances in Gene Therapy for Thalassemia
Thalassemia, an inherited blood disorder, imposes a significant economic and resource burden on patients, families, and society at large. In recent years, advances in medical research have led to a growing range of treatment options for thalassemia, including traditional blood transfusions and iron chelation therapy, drugs to induce gamma-globin production, hematopoietic stem cell transplantation (HSCT), and emerging approaches like gene and cell therapies, as well as nanomedicine. Among these, gene therapy has garnered particular attention as a potential cure, offering renewed hope to patients worldwide.