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.
28.11.24
Gene therapy: target liver
At the San Raffaele-Telethon Institute in Milan, an innovative liver-directed gene transfer platform applicable to various metabolic diseases has been developed thanks to the efforts of the research group led by Alessio Cantore, together with the biotech company Genespire.
18.11.24
Mucopolysaccharidosis 6 and gene therapy: present and future
A study just published by TIGEM researchers shows how the efficacy of this experimental therapy is maintained over time, at the highest doses. Study coordinator Brunetti-Pierri reports on possible future developments
16.09.24
Naples, world's first genere therapy for a rare retinal disease
The surgery, performed at AOU Vanvitelli, was made possible by an innovative gene therapy platform developed by Pozzuoli's TIGEM that allows for large gene transfer.
