Beta-thalassemia, 9 out of 10 people can recover thanks to gene therapy

Nine out of ten patients could recover from beta-thalassemia thanks to two advanced genetic engineering therapies: gene therapy and genome editing. To say it is Franco Locatelli, one of the leading experts in Hematology at the Bambino Gesù Hospital in Rome and President of the Superior Health Council: “Gene therapy consists in the addition of one or more normal copies of the altered gene that codes for the beta chains of hemoglobin. With the editing of the genome, on the other hand, another gene is modified which allows the reactivation of the gamma chains of hemoglobin, and therefore the production of that type of hemoglobin which is normally active only in the fetal period. Thanks to these two approaches there is a probability of a definitive cure in the order of 90% ”, explained Locatelli during the visit to the Hematology Campus of the Cervello Hospital in Palermo on May 7th.

The Campus is recognized as an international excellence, which combines research and assistance: “One of the best”, as also underlined Joshua of the SourcePediatric Director of the BMT program of the Hematology Center of Imperial College London, also visiting, on the sidelines of the scientific congress organized by the Piera Cutino Association together with United Onlus – Italian Federation of Thalassemias, Rare Hemoglobinopathies and Sickle Cell Disease, and Fasted Sicily (all united in the TD22 Committee).

Learn about beta-thalassemia and new therapies

In Italy it is estimated that there are about 7,000 people with beta-thalassemia: a number destined to increase, with over three million healthy carriers. Today, the life expectancy of patients has significantly increased, passing from a few years to decades, thanks to continuous transfusions and iron-chelation therapy which has made it possible to reduce the accumulation of iron in the organs. The new perspective opened by these therapies will further improve this scenario.

The activities of the Piera Cutino Association

To continue to raise awareness and provide information on beta-thalassemia, the Piera Cutino Association has organized a series of live streaming webinars on its Facebook profile throughout the month of May. “The next one will be Friday (May 13, from 18.30 to 19.00) Prof. Franco Locatelli will speak in live streaming about gene therapy, now considered no longer frontier, but a clinical practice to be extended and applied in the shortest possible time on broad scala ”, said Giuseppe Cutino, Founder of the association. Which also launched a fundraiser to finance research projects: anyone can contribute by purchasing Piera Cutino association t-shirts online.