Abstract
Treating a genetic disease by directly targeting its genetic roots: this is the promise of gene therapy. Once just a hopeful idea, it has now become a reality for certain rare genetic diseases, thanks in large part to the groundbreaking work of Telethon researchers. But how exactly does gene therapy work? Which diseases (not just genetic ones) can be treated with gene therapy today? What new developments—and challenges—lie on the horizon? Join us for an hour of exploration into one of today's most astonishing therapies!