The Sanfilippo syndrome type C is a severe neurodegenerative disease which appearws during the first years of life and for which there is no treatment yet. A recent study, published in Journal of Clinical Medicine, has created brain cell models of neurons and astrocytes that allow researchers to better know the mechanisms of this syndrome and assess potential therapies.
The study was coordinated by a team of the Faculty of Biology of the University of Barcelona and the Institute of Biomedicine of the University of Barcelona (IBUB), the Rare Diseases Networking Biomedical Research Centre (CIBERER), the Research Institute Sant Joan de Déu (IRSJD), in collaboration with a group from Lund University (Sweden). Researchers from the Hospital Clínic de Barcelona took part in the study too.
Reference article:
Benetó, N.; Cozar, M.; Castilla-Vallmanya, L.; Zetterdahl, O. G.; Sacultanu, M.; Segur-Bailach, E.; García-Morant, M.; Ribes, A.; Ahlenius, H.; Grinberg, D.; Vilageliu, L.; Canals, I. «Neuronal and astrocytic dfferentiation from Sanfilippo C Syndrome iPSCs for disease modeling and drug development». Journal of Clinical Medicine, February, 2020. Doi: 10.3390/jcm9030644
More information [UB News]
