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dc.contributor.author | Perez-Luz, Sara | |
dc.contributor.author | Loria, Frida | |
dc.contributor.author | Katsu-Jiménez, Yurika | |
dc.contributor.author | Oberdoerfer, Daniel | |
dc.contributor.author | Yang, Oscar-Li | |
dc.contributor.author | Lim, Filip | |
dc.contributor.author | Muñoz-Blanco, José Luis | |
dc.contributor.author | Díaz-Nido, Javier | |
dc.date.accessioned | 2020-09-28T19:25:33Z | |
dc.date.available | 2020-09-28T19:25:33Z | |
dc.date.issued | 2020-09-11 | |
dc.identifier.citation | Int J Mol Sci . 2020 Sep 11;21(18):E6662. | es_ES |
dc.identifier.uri | http://hdl.handle.net/20.500.12105/11077 | |
dc.description.abstract | Friedreich's ataxia is the most common hereditary ataxia for which there is no cure or approved treatment at present. However, therapeutic developments based on the understanding of pathological mechanisms underlying the disease have advanced considerably, with the implementation of cellular models that mimic the disease playing a crucial role. 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. | es_ES |
dc.description.sponsorship | This work was supported by grants of the Spanish National Research Plan (SAF 2015–69361-R),L’association Française de l’Ataxie de Friedreich and Fundación Ataxia en Movimiento. Frida Loria received funding from the Marie Skłodowska-Curie Action COFUND 2015 (EU project 713366—InterTalentum). | es_ES |
dc.language.iso | eng | es_ES |
dc.publisher | Multidisciplinary Digital Publishing Institute (MDPI) | es_ES |
dc.type.hasVersion | VoR | es_ES |
dc.rights.uri | http://creativecommons.org/licenses/by/4.0/ | * |
dc.subject.mesh | Frataxin | es_ES |
dc.subject.mesh | Friedreich´s ataxia | es_ES |
dc.subject.mesh | gene therapy | es_ES |
dc.subject.mesh | stem cells human olfactory mucosa | es_ES |
dc.title | Altered Secretome and ROS Production in Olfactory Mucosa Stem Cells Derived from Friedreich's Ataxia Patients. | es_ES |
dc.type | journal article | es_ES |
dc.rights.license | Atribución 4.0 Internacional | * |
dc.identifier.pubmedID | 32933002 | es_ES |
dc.format.volume | 21 | es_ES |
dc.format.number | 18 | es_ES |
dc.identifier.doi | 10.3390/ijms21186662 | es_ES |
dc.contributor.funder | L’association Française de l’Ataxie de Friedreich | |
dc.contributor.funder | Fundación Ataxia en Movimiento | |
dc.contributor.funder | Spanish National Research Plan | |
dc.description.peerreviewed | Sí | es_ES |
dc.identifier.e-issn | 1422-0067 | es_ES |
dc.relation.publisherversion | https://doi.org/10.3390/ijms21186662 | es_ES |
dc.identifier.journal | International journal of molecular sciences | es_ES |
dc.repisalud.centro | ISCIII::Instituto de Investigación de Enfermedades Raras | es_ES |
dc.repisalud.institucion | ISCIII | es_ES |
dc.relation.projectID | info:eu_repo/grantAgreement/EC/713366 | |
dc.relation.projectID | info:eu_repo/grantAgreement/ES/SAF 2015–69361-R | es_ES |
dc.rights.accessRights | open access | es_ES |