Andrea Ditadi awarded an ERC Consolidator Grant worth 2 million euro
Category Archives: From Research
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.
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.
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.
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.
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.
Orchard Therapeutics Receives Positive CHMP Opinion for LibmeldyTM for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)
First therapy recommended for EU approval for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants.
Osteopetrosis: new opportunity of treatment thanks to a licensing agreement
SiSaf announces a research collaboration and licensing agreement with the University of L’Aquila for the treatment of the debilitating bone disease, Autosomal Dominant Osteopetrosis Type 2.
Discovery of a novel drug candidate to develop effective treatments for brain disorders
Researchers at IIT-Istituto Italiano di Tecnologia discovered a novel chemical compound, which has the potential to became a new drug for the treatment of core symptoms of brain disorders like Down syndrome and autism.