French researchers report that they have successfully used gene therapy to treat beta-thalassemia in a 19-year-old male patient. Beta-thalassemia is a genetic blood disorder in which a defect in the gene coding for the beta-globin chain of hemoglobin results in persistent and life-threatening anemia and dangerously high blood iron levels. Two years after the treatment, according to the researchers, the young man no longer needs regular monthly blood transfusions and appears to be in good health.
The French team has the approval of French authorities to treat more patients with the same inherited disorder. The hope is that someday they’ll be able to successfully treat one of the most common of all genetic blood disorders – sickle cell anemia.
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