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
Category Archives: News
Groundbreaking Gene Therapies for Hereditary Diseases
Alessandro Aiuti, a physician and research scientist from Milan, receives the Else Kröner Fresenius Prize for Medical Research 2020 endowed with 2.5 million euros.
Sofinnova Partners Announces Third Investment from its Italian Fund
This investment brings the total financing by Sofinnova Partners to €25 million over the past six months.
Tigem and SR-Tiget involved in UPGRADE project, funded by the European Union
Fondazione Telethon announces the launch of the UPGRADE project, funded by the European Union with 15 million euros, aiming at developing more precise and effective gene therapies approaches.
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.
Restore Health – Therapies that cure
The European Commission (EC) announced that the FET Flagship RESTORE, which was initiated by the Berlin-Brandenburg Center for Regenerative Therapies (BCRT), has been selected to enter the next phase of the FET Flagship competition.
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).
Editas Medicine Announces Scientific Multi-Year Collaboration with Fondazione Telethon and Ospedale San Raffaele
Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, and Fondazione Telethon and Ospedale San Raffaele, have entered into a scientific collaboration to research and develop genome edited hematopoietic stem cell (HSC) and T cell therapies.
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).