In the United States, a severe inherited condition—beta thalassemia—has been cured for the first time using personalized gene therapy with the drug Zynteglo. At Children’s Hospital of Philadelphia, a 12-year-old patient underwent treatment in which her own cells were modified to produce healthy hemoglobin. Until this point, the only available option had been a bone marrow transplant from a compatible donor—a rare and risky procedure.
The process included collecting cells, turning them into universal stem cells, genetically modifying them, and returning them to the body after preparatory chemotherapy. As a result, the girl was able to get rid of chronic pain and no longer needed regular blood transfusions.
Now, similar treatment is being carried out in 17 more patients. The novelty of the approach lies in its personalization: donors are no longer required, and there is no risk of rejection. This is intended to make treatment accessible to thousands of people worldwide.
