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.
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
