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.
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
30.07.25
Usher Syndrome: Promising Preliminary Results from Gene Therapy
An experimental gene therapy developed at the TIGEM has shown encouraging clinical results in patients with Usher syndrome type 1B.
