[HIGHLIGHTS] Gene therapy works for bubble babies, long-term results are positive - Telethon for you

Gene therapy works for bubble babies, long-term results are positive - Telethon for you. Gene therapy on 'bubble' children, i.e.

those affected by the rare ADA-SCID immunodeficiency which forces them to live in sterile and isolated environments, also works in the long term. This is what emerges from a study published in Nature Medicine by the San Raffaele-Telethon Institute in Milan. The research involved 43 patients treated since 2000, 19 of which after the therapy was registered in the European Union in 2016.