From disease to cure: the story of an outstanding scientific result

Related topics:
Immune Conditions Rare Diseases Current and Potential Uses Gene Editing Gene Therapy

ADA-SCID is a rare genetic disease which affects the immune system within the first months of life. This video from Fondazione Telethon describes how gene therapy is used to treat ADA-SCID.

Fondazione Telethon logo
Did you find the content on this page useful? If not, you can leave us a message so we can improve Send us your thoughts