A new chapter in a 30-year history: passing on the torch is Andrea Ballabio, who directed the Institute since its foundation in 1994.
Author Archives: Flavia Balboni
Fondazione Telethon and Orchard Therapeutics complete transfer of marketing authorization of Strimvelis for ADA-SCID in Europe
Una nuova sfida per Telethon, che amplia i confini della propria missione
San Raffaele-Telethon Institute receives European funds to study how blood is formed
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.
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.
Fondazione Cariplo and Fondazione Telethon: 24 projects funded with more than 5 million euros for basic research in the field of rare diseases
Winners of the 2021 Call for Basic Research have been selected from all over Italy to study the still obscure portions of our genetic heritage.
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.
Aicardi-Goutières syndrome: when the immune system detects viruses where there are none
An interview with Anna Kajaste-Rudnitski from the San Raffaele Telethon Institute for Gene Therapy in Milan, author of a study that gives new insights into this rare syndrome and provides also useful information to investigate the interaction between the immune system and viruses, including Sars-CoV-2.
Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)
First gene therapy to receive full EU marketing authorization for eligible MLD patients. One-time treatment with Libmeldy has been shown to preserve motor and cognitive function. Achievement shared with research alliance partners Fondazione Telethon and Ospedale San Raffaele.
A novel finding on Kabuki syndrome, a rare genetic disease
The research, coordinated by the University of Trento, has recreated for the first time the pathological condition in a test tube discovering what happens in the cell nucleus and what hinders the formation of cartilages and bones.