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.
SOURCE: http://www.ansa.it/
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