Italy pioneers gene therapy to treat rare genetic blood disease

Two Italian women have become the first patients in Europe to receive advanced gene therapy for rare genetic blood disorders.

Two Italian women in their 20s have become the first patients in Europe to receive advanced gene therapy for rare genetic blood disorders.

At San Matteo Hospital in Pavia, south of Milan, two patients – one with β-thalassemia major and the other with severe sickle cell anemia – have officially received the Casgevy gene therapy, the first to be deployed outside the framework of a clinical trial in Europe.

Casgevy is a revolutionary treatment developed using advanced gene editing technology to correct mutations in the β-globin gene – the main cause of hemoglobin disorders such as β-thalassemia and sickle cell anemia.

The two diseases mentioned above belong to the group of hemoglobinopathy – serious genetic disorders related to hemoglobin – which often require lifelong blood transfusions, while also facing complications of multiple organ failure and severely reduced quality of life.

The breakthrough treatment is expected to create a turning point in the treatment of diseases in the hemoglobinopathy group./.

(Vietnam News Agency/Vietnam+)

Source: https://www.vietnamplus.vn/italy-tien-phong-trong-lieu-phap-gene-dieu-tri-benh-mau-di-truyen-hiem-gap-post1043643.vnp