News
21.06.16
Preliminary results reporting the safety and effectiveness of gene therapy in patients with metachromatic leukodystrophy published in The Lancet
The gene therapy developed in the laboratories of the San Raffaele Telethon Institute for Gene Therapy in Milan (SR-Tiget) remains effective as a potential early treatment of metachromatic leukodystrophy (MLD).
30.05.16
Strimvelis receives European marketing authorisation to treat very rare disease, ADA-SCID
GlaxoSmithKline (GSK), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) today announced that the European Commission has approved Strimvelis, the first ex-vivo stem cell gene therapy to treat patients with a very rare disease called ADA-SCID
16.06.14
Gene therapy: a "molecular scalpel" corrects the genes of blood stem cells
A group of researchers at the San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan was able for the first time to rewrite the DNA of human blood stem cells by editing the genome.
16.06.14
Cardiomyopathies: a protein has been discovered that keeps the heart young
It's called Atrogin-1 and it's a protein that plays an important role in cardiac health: its task, as demonstrated by a study funded by Telethon, is to identify and dispose of the waste substances produced by cellular activity, which would be harmful to the heart.
10.02.14
Ipex syndrome: fresh hope for a cure from Telethon - San Raffaele researchers
From Telethon - San Raffaele research come new treatment prospects for a rare genetic diseases affecting the immune system, IPEX syndrome.
14.01.14
From Telethon-funded gene therapy comes a new weapon in the battle against cancer
The innovative technique developed in the laboratories of the Telethon San Raffaele Institute for Gene Therapy for the treatment of two extremely serious genetic diseases can also be effective in treating tumours.
14.01.14
TIGEM: a centre of excellence for the south
The new premises of the Telethon Institute for Genetics and Medicine (Tigem) is set to open in the former "Olivetti area" in Pozzuoli.
06.11.13
Research into a rare kidney disease reveals a mechanism responsible for hypertension
Research into a rare kidney disease has shed important light on a possible way to tackle high blood pressure, a risk factor for the circulatory system that in Italy alone affects a third of the adult population.
29.10.13
Polycystic kidney: a problem of tubule expansion lies at the base of polycystic kidney disease
New light has been shed on the molecular mechanisms that lie at the base of one of the most common genetic diseases, autosomal dominant polycystic kidney, which in Italy alone affects 60 thousand people.
