15.11.24
Dawn's reflection: hope from Tigem research
The child, born with mucopolysaccharidosis type 6, was treated with gene therapy in 2020 and to date her liver is able to produce the missing enzyme that causes the disease.
11.10.24
A hope twelve thousand kilometres long
The story of Amari, who came from the small Caribbean Island of Trinidad to Milan to receive gene therapy for his rare genetic disorder of the immune system.
02.08.24
Violet's story: between research and outreach
The Canadian girl was born with mucopolysaccharidosis type 6. With help from the Isaac Foundation, she received important therapies in her country and participated in Italy in the trial developed by the Telethon Institute of Genetics and Medicine.
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.
14.11.25
CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)
Announced the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), recommending marketing authorisation in the European Union for Waskyra™, an ex vivo gene therapy for Wiskott-Aldrich Syndrome.
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.
