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.
02.04.26
N-of-1 therapies: personalised treatments for single patients
How N-of-1 therapies and antisense oligonucleotides enable personalised treatments for ultra-rare genetic diseases, from mutation to clinical use.
31.03.26
In memory of Claudio Bordignon, pioneer of gene therapy
His work combined scientific vision, experimental rigor and a constant focus on the potential impact of research for patients affected by severe inherited diseases.
23.03.26
Cytiva and San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) launch collaboration to advance next-generation genomic medicine platforms
New “Danaher Beacon,” first on the European continent, aims to accelerate translation, scalability, and reliability across cell and gene therapy workflows. Focus is on lentiviral gene transfer and targeted gene editing of hematopoietic stem cells.
