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

Related topics:
Malattie autoimmuni Malattie rare Uso attuale e potenziale Editing genetico Terapia genica

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
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