News
17.12.22
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.
06.06.22
Mucopolysaccharidosis Type VI: positive results from gene therapy
Promising outcomes have emerged from an innovative therapy for this metabolic disease developed by TIGEM
29.04.22
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.
05.04.22
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.
09.03.22
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.
21.12.20
Orchard Therapeutics Receives EC Approval for the Treatment of Early-Onset MLD
First gene therapy to receive full EU marketing authorization for eligible MLD patients.
03.11.20
Potential adverse reaction to gene therapy in a patient treated with Strimvelis for the treatment of ADA-SCID
Fondazione Telethon has recently learned that a patient suffering from a rare immunodeficiency of genetic origin, ADA-SCID, and treated in 2016 with gene therapy has developed leukemia.
16.10.20
Orchard Therapeutics Receives Positive CHMP Opinion for the Treatment of Early-Onset MLD
First therapy recommended for EU approval for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants.
07.09.20
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.
