Arnas G. Brotzu performs first gene therapy for thalassemia major in Sardinia

An adult patient with thalassemia major in Sardinia has successfully undergone a pioneering gene therapy at Arnas G. Brotzu. The procedure utilized genetically modified hematopoietic stem cells to treat the condition.

Following the treatment, the patient has shown positive results, including a progressive increase in total hemoglobin and the production of new hemoglobin from the modified cells. Notably, the patient has not required blood transfusions in the last month and has been discharged in good general condition. The therapy employs genomic editing to enable the production of fetal hemoglobin, which helps correct the anemia characteristic of thalassemia. This development offers a significant new therapeutic perspective for a region with a historically high prevalence of the disease.