New gene treatment for thalassemia kids

(ANSA) - Rome, January 21 - Three children with the blood disorder thalassemia can look forward to a life without transfusions thanks to a new gene therapy which has proved particularly effective with young patients, a study in Nature Medicine said Monday.
    Thanks to the new treatment at Milan's San Raffaele Hospital, funded by Telethon, the three children under the age of six have been rendered "completely free" of the need for transfusions, the study said.
    In three adult patients a significant reduction of the dependence on transfusions was registered, it said.
    The patients were suffering from the beta form of thalassemia.
    Thalassemia is an inherited blood disorder in which the body makes an abnormal form of hemoglobin. Hemoglobin is the protein molecule in red blood cells that carries oxygen.
    The disorder results in excessive destruction of red blood cells, which leads to anemia. Anemia is a condition in which your body doesn't have enough normal, healthy red blood cells.
    Thalassemia is inherited, meaning that at least one of your parents must be a carrier of the disease. It's caused by either a genetic mutation or a deletion of certain key gene fragments.
    Thalassemia minor is a less serious form of the disorder. There are two main forms of thalassemia that are more serious. In alpha thalassemia, at least one of the alpha globin genes has a mutation or abnormality. In beta thalassemia, the beta globin genes are affected.
    Some 7,000 people suffer from the disorder in Italy.
    Thalassemia signs and symptoms may include: •Fatigue •Weakness •Pale or yellowish skin •Facial bone deformities •Slow growth •Abdominal swelling •Dark urine