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dc.contributor.authorPerez-Luz, Sara 
dc.contributor.authorLoria, Frida
dc.contributor.authorKatsu-Jiménez, Yurika
dc.contributor.authorOberdoerfer, Daniel
dc.contributor.authorYang, Oscar-Li
dc.contributor.authorLim, Filip
dc.contributor.authorMuñoz-Blanco, José Luis
dc.contributor.authorDíaz-Nido, Javier
dc.date.accessioned2020-09-28T19:25:33Z
dc.date.available2020-09-28T19:25:33Z
dc.date.issued2020-09-11
dc.identifier.citationInt J Mol Sci . 2020 Sep 11;21(18):E6662.es_ES
dc.identifier.urihttp://hdl.handle.net/20.500.12105/11077
dc.description.abstractFriedreich'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.sponsorshipThis 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.isoenges_ES
dc.publisherMultidisciplinary Digital Publishing Institute (MDPI) es_ES
dc.type.hasVersionVoRes_ES
dc.rights.urihttp://creativecommons.org/licenses/by/4.0/*
dc.subject.meshFrataxines_ES
dc.subject.meshFriedreich´s ataxiaes_ES
dc.subject.meshgene therapyes_ES
dc.subject.meshstem cells human olfactory mucosaes_ES
dc.titleAltered Secretome and ROS Production in Olfactory Mucosa Stem Cells Derived from Friedreich's Ataxia Patients.es_ES
dc.typejournal articlees_ES
dc.rights.licenseAtribución 4.0 Internacional*
dc.identifier.pubmedID32933002es_ES
dc.format.volume21es_ES
dc.format.number18es_ES
dc.identifier.doi10.3390/ijms21186662es_ES
dc.contributor.funderL’association Française de l’Ataxie de Friedreich
dc.contributor.funderFundación Ataxia en Movimiento
dc.contributor.funderSpanish National Research Plan
dc.description.peerreviewedes_ES
dc.identifier.e-issn1422-0067es_ES
dc.relation.publisherversionhttps://doi.org/10.3390/ijms21186662es_ES
dc.identifier.journalInternational journal of molecular scienceses_ES
dc.repisalud.centroISCIII::Instituto de Investigación de Enfermedades Rarases_ES
dc.repisalud.institucionISCIIIes_ES
dc.relation.projectIDinfo:eu_repo/grantAgreement/EC/713366
dc.relation.projectIDinfo:eu_repo/grantAgreement/ES/SAF 2015–69361-Res_ES
dc.rights.accessRightsopen accesses_ES


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Atribución 4.0 Internacional
Este Item está sujeto a una licencia Creative Commons: Atribución 4.0 Internacional