Division of Regenerative Medicine, Stem Cells and Gene Therapy
DIRECTOR: Giulio Cossu

The Division of Regenerative Medicine was created at the end of year 2008, by joining the Telethon Institute of Gene Therapy (TIGET), that remains an independent institute directed by Luigi Naldini, with former Stem Cell Research Institute and also includes researchers with expertise in developmental and stem cell biology, inflammation, immunology, tissue regeneration as well as clinicians actively involved in clinical trials, mainly in hematology. TIGET contributes its expertise and international leadership in gene therapy, including the development of new pre-clinical protocols, vector design and new clinical trials, in addition to the one currently running of congenital immune deficiency do to a mutation in the Adenosine Deaminase gene. The Division currently comprises 10 Principal Investigators/Heads of Unit (Aiuti, Bonini, Clementi, Ciceri, Cossu, Ferrari, Manfredi, Naldini, Roncarolo, Villa) and 10 Group Leaders, (Bacchetta, Biffi, Brunelli, De Palma, Fleischauer, Gabellini, Galli, Gregori, Montini, Rovere,) and approximately 200 researchers, clinicians, post and pre-doctoral fellows, undergraduate students and technicians. Projects focus on stem cell biology and physiopathology of the hematopoietic system, skeletal muscle and of the nervous system aiming at elucidating the pathogenesis of certain diseases of these tissues (congenital immune deficiencies, thalassemias, hemophilias, leukemias, muscular dystrophies and tesaurismosis), at developing pre-clinical models and clinical protocols of gene and cell therapy. Several clinical trials for onco-hematologic pathologies are currently running.