“I can’t walk”. When you hear those words on the lips of a child below the age of three, it’s hard to turn your back and walk away. Imagine you’re the child’s parents. The words came out with the composed calmness of a fact of nature.
03.02.25
Fondazione Telethon announced that it has submitted the Marketing Authorization Application (MAA) for the gene therapy - etuvetidigene autotemcel - for the treatment of patients with Wiskott-Aldrich Syndrome (WAS), a rare genetic disease of the immune system to the European Medicines Agency (EMA).
03.12.24
Daniela Cesana and Alessio Cantore presented two projects, respectively to investigate the impact, safety and efficacy of gene therapy through the 'signatures' of circulating DNA and to investigate the biology of liver cells in order to exploit them in the field of gene therapy.
28.11.24
At the San Raffaele-Telethon Institute in Milan, an innovative liver-directed gene transfer platform applicable to various metabolic diseases has been developed thanks to the efforts of the research group led by Alessio Cantore, together with the biotech company Genespire.
