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
ALL RIGHTS RESERVED © Copyright ANSA