A Stunning Gene-Therapy Cure for Beta Thalassemia, One of the World’s Most Devastating Blood Diseases
In 1976, my final year of medical school, I travelled abroad and spent several months working in the hematology clinic at Hadassah Hospital, in Jerusalem. Every day, I attended to children and teen-agers suffering from a blood disorder called beta thalassemia. They were easy to identify in the clinic waiting room. Their skin was a pale yellow, their skull and facial bones were distorted, and their abdomens bulged from an enlarged liver and spleen. Many were short of breath, with swollen legs and other signs of heart failure.