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.
17.07.26
Scientific careers at TIGEM: research into rare genetic diseases
At TIGEM, PhD students and postdocs grow in an international environment through mentoring, advanced training and European research programmes.
13.07.26
A new gene therapy clinical trial launched for patients with transfusion-dependent beta-thalassemia
Fondazione Telethon and IRCCS Ospedale San Raffaele, sponsors of the trial, together with Ospedale Pediatrico Bambino Gesù launch a multicenter study based on an optimized gene therapy protocol developed at the SR-Tiget Institute.
08.07.26
Genespire and SR-Tiget show durable preclinical efficacy of liver-directed gene therapy for methylmalonic acidemia
The spin-off of Fondazione Telethon and Ospedale San Raffaele reports positive results in preclinical models of this rare metabolic disorder, further supporting the path toward first-in-human application.
