Sorbonne Universités (SORB)
The multidisciplinary medicine campus of the Pitié-Salpêtrière Hospital - one of the largest teaching hospitals in Europe - has a strong track record of experimental medicine and excellence in basic research. In particular, the research clinicians in charge of the French registry of Pompe disease patients, who are leading the research on clinical aspects of the disease and enzyme replacement therapy, are located at the Research Center for Myology at the Pitié-Salpêtrière Hospital. The center was created by the Association Française contre les Myopathies (AFM) in 1966 and is an international reference center for diseases affecting the skeletal muscle. Most importantly, the Research Center for Myology supports an exceptional array of gene therapy research in collaboration with the Institute of Biotherapies of which Genethon is also a partner.
SORB will bring key expertise and reagents to the MYOCURE project to 1) develop and produce therapeutic AAV vectors to target multiple tissues to treat inherited and acquired diseases; 2) model and study immune responses in gene transfer in the context of muscle, liver, brain, and other tissues; 3) develop immunomodulatory strategies to overcome transgene immunogenicity in muscle gene transfer; 4) devise strategies to overcome humoral immunity to AAV vectors. Furthermore, UPMC has been closely involved in the translational process from bench to bedside applied to gene therapy, acquired through the participation in clinical gene therapy trials.