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.
27.07.26
ER-phagy: a new strategy to target cellular quality control
The TIGEM LEAD-ER project investigates whether ER-phagy can be pharmacologically activated to remove misfolded proteins involved in different diseases.
20.07.26
Beta-thalassaemia: from the first trial to a new gene therapy protocol
A new clinical trial is evaluating an optimised ex vivo gene therapy protocol for transfusion-dependent beta-thalassaemia developed at SR-Tiget.
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.
