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.
03.08.26
Working at the intersection of Biology and Data Science at TIGEM
At TIGEM, biologists, bioinformaticians and statisticians combine data, computational models and laboratory research to advance rare disease science.
29.07.26
Epigenetic editing: a technology pioneered at SR-Tiget enters clinical trials for hepatitis B
SR-Tiget’s epigenetic editing technology can permanently silence genes without cutting DNA and has now entered clinical trials for hepatitis B.
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.
