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.
19.10.26
Luigi Naldini elected to the U.S. National Academy of Medicine
The prestigious recognition honors a pioneer of lentiviral gene therapy whose discoveries have led to approved treatments for rare diseases, including the first gene therapy brought to the U.S. market through a non-profit development pathway.
30.09.26
Newly discovered pathway unlocks the therapeutic potential of TFEB
A study published in Nature reveals druggable regulators of TFEB, a key controller of cellular recycling, opening new opportunities for the development of therapies for rare genetic diseases, cancer and other disorders linked to lysosomal dysfunction.
29.09.26
New gut-liver link discovered in alpha-1 antitrypsin deficiency
A study led by Nunzia Pastore at TIGEM shows that the mutated protein responsible for the disease also alters intestinal cells, opening new avenues for identifying at-risk patients and understanding the variability of the disease.
