5 November 2019 - A new targeted gene therapy shows the promise and pitfalls of medical advances.
On a sweltering summer morning, Daniela Miccichè headed from her home in the center of Sicily to a hospital in the ancient city of Palermo on the coast. It’s a trip she repeats every month. A travel agent from the town of Caltanissetta, Miccichè has no choice but to make the two-hour journey to the Franco and Piera Cutino clinic.
Scores of others pass through its front doors, all with the same disease. She’s among an estimated 7,000 Italians, many of them Sicilian, who depend on transfusions to treat beta thalasseamia, a genetic disorder that hampers red blood cells’ ability to carry oxygen.