From Research
19.12.25
The sustainability of gene therapies for rare diseases: a model born from ADA-SCID and WAS
Fondazione Telethon have obtained marketing authorisation for a gene therapy thanks to a sustainability model that integrates research with industry, patients and supporters. Read the full interview with Celeste Scotti, R&D Director at Fondazione Telethon.
16.12.25
From Wiskott–Aldrich Syndrome to a new model for gene therapies
Rare and ultra-rare genetic diseases pose profound scientific, human and economic challenges. The development of gene therapy for Wiskott–Aldrich syndrome (WAS) is a clear example of this complexity.
05.12.25
Engineering peripheral immune tolerance: Tregs and beyond
Tregs and beyond: discover how SR-TIGET scientists are engineering peripheral immune tolerance for tomorrow’s precision therapies.
13.11.25
Cracking the Code: IPEX, FOXP3 and the Path to Gene Therapy
Discover how SR-TIGET scientists advanced the genetics of peripheral immune tolerance, from FOXP3 and IPEX to gene therapy using lentiviral vectors.
04.11.25
The origins of peripheral immune tolerance: SR-TIGET's contribution to a Nobel-winning field
Discover how SR-TIGET scientists helped shape the science of peripheral immune tolerance, from the discovery of Treg and Tr1 cells to early gene therapy.
16.10.25
Attya Omer Named Runner-Up in the Nature Inspiring Women in Science Awards
The scientist of SR-TIGET and Università Vita-Salute San Raffaele recognized internationally for her vision in advancing gene therapy and inspiring women in science.
23.09.25
The Armenise-Harvard Foundation invests one million dollars in brain research
Thanks to the Career Development Award, neuroscientist Gabriele Ciceri will establish a new laboratory at the San Raffaele-Telethon Institute for Gene Therapy.
28.08.25
How Newborn Liver Cells Shape Growth and Future Treatments
A discovery at SR-Tiget reveals the key liver cells driving organ growth and advancing pediatric gene therapy
06.08.25
New 3D imaging tech revolutionizes lysosomal disease research
A new HTFC method allows live 3D lysosome imaging, with applications in Niemann-Pick C1 diagnosis and monitoring
