Pérez-Luz, S.; Loria, F.; Katsu-Jiménez, Y.; Oberdoerfer, D.; Yang, O.-L.; Lim, F.; Muñoz-Blanco, J.L.; Díaz-Nido, J.; Int. J. Mol. Sci. 2020, 21, 6662. doi:10.3390/ijms21186662. Human olfactory ecto-mesenchymal stem cells represent a novel model that could prove useful due to their accessibility and neurogenic capacity. Here, we isolated and cultured these stem cells from Friedreich´s ataxia patients and healthy donors, characterizing their phenotype and describing disease-specific features such as reduced cell viability, impaired aconitase activity, increased ROS production and the release of cytokines involved in neuroinflammation. Importantly, we observed a positive effect on patient-derived cells, when frataxin levels were restored, confirming the utility of this in vitro model to study the disease. This model will improve our understanding of Friedreich´s ataxia pathogenesis and will help in developing rationally designed therapeutic strategies.
Saturday, September 12, 2020
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