Gene-edited stem cells help five blood disorder patients stop transfusions in clinical trial

MedicalXpress | 四月 08, 2026

Stem cell transplantation could be a rapid and effective way to restore hemoglobin production in individuals with the blood disorder β-thalassaemia. The treatment, presented in a phase 1 clinical trial, could reduce dependence on blood transfusions in some individuals with the disease in the future, although further trials are needed to confirm these findings.