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.
03.12.24
ERC: two researchers from San Raffaele-Telethon institute for gene therapy in Milan each receive 2 million euros for their prestigious funding together with IRCCS Ospedale San Raffaele and Università Vita-Salute San Raffaele
Daniela Cesana and Alessio Cantore presented two projects, respectively to investigate the impact, safety and efficacy of gene therapy through the 'signatures' of circulating DNA and to investigate the biology of liver cells in order to exploit them in the field of gene therapy.
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
