From epigenetics - new molecular switches to silence genes
A new gene silencing strategy has been developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) that allows permanent silencing of genes by acting on epigenetic processes that regulate their reading and translation into proteins.
Editas Medicine Announces Scientific Multi-Year Collaboration with Fondazione Telethon and Ospedale San Raffaele
Company to Collaborate with Leading Genomic Medicines Institutes to Progress Genome Edited Hematopoietic Stem Cell and T Cell Based Therapies
Preliminary results reporting the safety and effectiveness of gene therapy in patients with metachromatic leukodystrophy published in The Lancet
A gene therapy with vectors derived from HIV virus has been developed in the laboratories of the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan. Preliminary results reporting the safety and efficacy of this investigational gene therapy for early treatment of the early onset forms of a rare genetic disease, metachromatic leukodystrophy have been published in The Lancet.
Strimvelis receives European marketing authorisation to treat very rare disease, ADA-SCID
GSK, Fondazione Telethon and Ospedale San Raffaele gain approval to provide life-saving gene therapy to patients