From Research
02.03.26
Retinal dystrophies: gene-agnostic therapy based on miRNAs
New miR-based gene-agnostic strategies target shared mechanisms in retinal dystrophies, opening new therapeutic options beyond single-gene therapy.
23.02.26
AAV gene therapy for large genes: two TIGEM platforms
How AAV gene therapy for large genes overcomes size limits through two TIGEM technology platforms for inherited retinal and rare diseases.
11.02.26
February 11: The Value of Women in Research
Bioinformatics and translational research at TIGEM, as told by two of its leading women researchers, on the International Day of Women and Girls in Science.
05.02.26
Gene therapy platform for lysosomal storage diseases
A new gene therapy platform for lysosomal storage diseases enables parallel development, reducing time, costs and improving sustainability for rare disease treatments.
19.12.25
The sustainability of gene therapies for rare diseases: a model born from ADA-SCID and WAS
Fondazione Telethon have obtained marketing authorisation for a gene therapy thanks to a sustainability model that integrates research with industry, patients and supporters. Read the full interview with Celeste Scotti, R&D Director at Fondazione Telethon.
16.12.25
From Wiskott–Aldrich Syndrome to a new model for gene therapies
Rare and ultra-rare genetic diseases pose profound scientific, human and economic challenges. The development of gene therapy for Wiskott–Aldrich syndrome (WAS) is a clear example of this complexity.
05.12.25
Engineering peripheral immune tolerance: Tregs and beyond
Tregs and beyond: discover how SR-TIGET scientists are engineering peripheral immune tolerance for tomorrow’s precision therapies.
13.11.25
Cracking the Code: IPEX, FOXP3 and the Path to Gene Therapy
Discover how SR-TIGET scientists advanced the genetics of peripheral immune tolerance, from FOXP3 and IPEX to gene therapy using lentiviral vectors.
04.11.25
The origins of peripheral immune tolerance: SR-TIGET's contribution to a Nobel-winning field
Discover how SR-TIGET scientists helped shape the science of peripheral immune tolerance, from the discovery of Treg and Tr1 cells to early gene therapy.
