From Research
20.04.26
Fondazione Telethon’s genomic program ends the diagnostic odyssey for hundreds of children
Eight years of the Undiagnosed Disease Program by Fondazione Telethon: nearly 1 in 2 children receive a molecular diagnosis. A new study in Genetics in Medicine Open reports the main outcomes and describes a model that can be adopted by national health systems.
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.
12.03.26
Reaching the CNS with lentiviral gene therapy
SR-TIGET ex vivo and in vivo lentiviral gene therapy aims to target the CNS, building on MLD success and expanding toward new therapeutic strategies.
09.03.26
Tigem: technological platforms serving research
TIGEM’s core facilities combine advanced technology and scientific mentoring, accelerating growth and research for PhD students and postdocs.
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.
