From Research
08.07.26
Genespire and SR-Tiget show durable preclinical efficacy of liver-directed gene therapy for methylmalonic acidemia
The spin-off of Fondazione Telethon and Ospedale San Raffaele reports positive results in preclinical models of this rare metabolic disorder, further supporting the path toward first-in-human application.
23.06.26
ESMRank: using AI to predict the effects of protein mutations
At TIGEM, ESMRank uses artificial intelligence to rank protein mutations and help researchers interpret genetic variants linked to disease.
10.06.26
TUDP: genomics, bioinformatics and knowledge for undiagnosed diseases
Fondazione Telethon’s TUDP combines genomics, bioinformatics and research to identify the genetic causes of rare paediatric diseases without a diagnosis.
03.06.26
New “SMArT” platform makes gene editing in hematopoietic stem cells more efficient and safer
Nature Biotechnology study shows near-pure selection of correctly edited blood stem cells while reducing dangerous genomic alterations.
19.05.26
SR-Tiget’s Development Facilities: where gene therapy takes shape
How SR-Tiget’s development facilities are key to turn research into advanced therapies, and why they represent a new professional frontier.
08.05.26
Lysosome Biology: From “Suicide Bag” to Metabolic Hub
Tigem research reveals the lysosome as a metabolic hub via TFEB and mTOR, enabling new treatments for lysosomal diseases, neurodegeneration and cancer.
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.
