in Trentino-Alto Adige Telethon Foundation appointed €159,500 to fund a research project on Duchenne muscular dystrophy. It is one of 22 research projects funded at the national level by the Telethon Foundation, which has allocated a total of nearly 4 million euros (specifically 3,904,094) to projects on rare genetic diseases raised through citizen donations.
The regionally funded project is carried out by a research team from the University of Trento led by a researcher. Stefano Biressi. Biressi and his team will investigate some aspects of Duchenne muscular dystrophy, a disease characterized by the progressive degeneration of muscle tissue and its replacement by fibrotic tissue. In addition to contributing to symptoms, fibrosis is a barrier to effective treatments, particularly those based on cell or gene therapy.
Recent observations by Biressi’s group suggested its overactivation The complement system is a part of the immune system, may play a role in the pathogenetic process leading to fibrosis. The aim of the study is to further explore these aspects and explore the potential beneficial effects of drugs and genetic approaches that can inhibit the activity of the complement system. In particular, we will investigate the effect of inhibiting C1q, a component of the complement protein complex.