Stem cells and muscular dystrophies

The research activity of the group is focused on the development of skeletal muscles, the cell population that contribute to muscle histogenesis and regeneration and on the use of a class of muscle progenitors, the mesoangioblasts for cell therapy protocols involved in muscular dystrophies. Mesoangioblasts can be expanded extensively in vitro and participate to muscle growth and regeneration. Because of these features both wild type or dystrophic, genetically corrected, mesoangioblasts were transplanted into the arterial circulation of dystrophic mice and dogs. Transplantation resulted in morphological, biochemical and functional amelioration of the dystrophic pathology. Based on these discoveries a phase I/II clinical trial is planned for Duchenne Muscular Dystrophy. Moreover, new strategies for improved cell therapies are being developed.

Unit members

Selected publications

Gargioli, C., Coletta, M., De Grandis, F., Cannata, S.M. and Cossu G. 2008.
PlGF-MMP9 expressing cells restore microcirculation and efficacy of cell
therapy in old dystrophic muscle. Nature Med. 14(9):973-8.

Berghella, L., De Angelis, L., De Buysscher, T., Mortazavi, A., Biressi, S.,
Forcales, S.V.,  Sirabella, D., Cossu, G. and Wold, B. 2008. A highly conserved molecular switch binds MSY-3 to regulate myogenin repression in post-natal muscle Genes & Dev. 22(15):2125-38.

Dellavalle, A, Sampaolesi, M, Tonlorenzi, R,  Tagliafico, E,  Sacchetti, B,  Perani, L,, Innocenzi, B,  Galvez, BG,  Messina, G,   Morosetti, R,  Li, S,  Belicchi, M,  Peretti, G,  Chamberlain, JS,  Wright, WE,  Torrente, Y,   Ferrari, S,   Bianco, P and Cossu, G. 2007. Pericytes of human skeletal muscle are myogenic precursors distinct from satellite cells. Nature Cell Biol. 9:255-67.

Galvez, B.G., Sampaolesi, M., Brunelli, S., Covarello, D., Gavina, M., Rossi, B., Costantin,  G., Torrente, Y. and Cossu G. 2006. Complete repair of dystrophic skeletal muscle by mesoangioblasts with enhanced migratory ability.  J. Cell Biol. 174: 231-43.

Sampaolesi, M., Blot, S., D’Antona, G., Granger, N., Tonlorenzi, R., Innocenzi, A., Mognol, P., Thibaud, J.L., Galvez, B.G., Barthélémy, I., Perani, L., Mantero, S., Guttinger, M., Pansarasa, O., Rinaldi, C., Cusella De Angelis, M.G., Torrente, Y., Bordignon, C., Bottinelli, R. and Cossu, G. 2006. Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs. Nature, 444:574-9.