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TIGEM, a Telethon Foundation organization, is a leading institution for research on rare genetic diseases. With the support of Telethon, the European Union and many other funding agencies, TIGEM's 14 research groups are engaged in numerous clinical trials and collaborative projects. In addition, TIGEM's alliances with various universities, two of which being the awarding bodies for TIGEM's PhD programs, offer the institute additional resources and opportunities for excellent students. 

With TIGEM's carefully selected group of internationally-renowned researchers,  the Institute's studies are able to span over many fields of study. Cell Biology, Systems Biology and Molecular Therapy are the principal disciplines involved in research, clinical trials and translational medicine at TIGEM. The Institute's success in biological research applied to genetic diseases has received international recognition; since 1994, TIGEM has published over 800 research articles in leading scientific journals and continues to be one of the most prolific research centers in Europe.