News from Research
01 / 01
First gene therapy to receive full EU marketing authorization for eligible MLD patients.
Margaux discovering the world thanks to gene therapy
She was the first Belgian girl with the rare immunodeficiency ADA-SCID treated with the gene therapy approach developed by Telethon researchers. From the risk of a life of seclusion, she has conquered the possibility of doing whatever she likes.
In Italy for love and by choice
The story of Attya and her personal and professional journey that took her to the San Raffaele Telethon Institute for Gene Therapy in Milan.
Lorenzo wants to go far
“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.
Andrea Ditadi awarded an ERC Consolidator Grant worth 2 million euro
New frontiers for gene therapy
At the San Raffaele-Telethon Institute in Milan researchers are working to apply gene therapy, an approach that has already given important results, to even more diseases optimizing development times and costs.
Generating blood in vitro: a new piece of the puzzle
An important contribution to the elucidation of the mechanisms through which blood stem cells arise during embryonic life from a study conducted by the San Raffaele Telethon Institute for Gene Therapy.
Gene therapy targeted to the brain: a complex but not impossible challenge
Angela Gritti from the San Raffaele Telethon Institute of Milan talks about the efforts of applying gene therapy to one of the most difficult body organs to access.