Researchers at IIT-Istituto Italiano di Tecnologia discovered a novel chemical compound, which has the potential to became a new drug for the treatment of core symptoms of brain disorders like Down syndrome and autism.
Category Archives: From Research
Study of genetic condition leads to discovery of mechanism that promotes the formation of renal cysts and tumours, thanks to the support of Telethon and AIRC
At the Telethon Institute of Genetics and Medicine (Tigem) in Pozzuoli, the team led by Andrea Ballabio has described in Nature* how the study of a rare genetic condition has provided the key to understanding a mechanism that leads to the formation of cysts and tumours in certain organs, in particular the kidneys. Supported by Fondazione Telethon, Fondazione AIRC for cancer research and Regione Campania, the study included the participation the European Institute of Oncology (IEO) and the Institute of Cell Biology at the University of Innsbruck
Beta Thalassemia: clinical trial provides encouraging evidence for efficacy of gene therapy
Gene therapy, especially if administered early, could be an effective treatment strategy for beta thalassemia, a genetic disease that is quite common in Mediterranean countries, affecting over 7000 patients in Italy only.
A new ingredient to potentiate gene therapy in stem cells
A new study published by the research team led by Anna Kajaste-Rudnitski at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, shows that a naturally occurring compound significantly increases the efficiency of lentiviral vector-mediated gene transfer in blood stem cells.
From epigenetics – new molecular switches to silence genes
It is possible to silence a desired gene by hiding it from the molecular machinery that has the task of reading it and expressing its function. This is the result of a research carried out at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget).
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).
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.
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.
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.
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.