Successful gene therapy for transfusion-dependent α-thalassemia: a case report

Xiaolin Yin1, Linxia Qian2,3, Guoyi Dong2,3

  • 1Department of Hematology, The 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army, Nanning, China.

Blood Advances
|May 27, 2026
PubMed
Summary

This study shows lentiviral gene therapy can potentially cure transfusion-dependent alpha-thalassemia. A young patient achieved transfusion independence and sustained high hemoglobin levels after receiving modified stem cells.

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