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Article

Altered Secretome and ROS Production in Olfactory Mucosa Stem Cells Derived from Friedreich’s Ataxia Patients

1
Centro de Biología Molecular Severo Ochoa (CSIC-UAM) and Departamento de Biología Molecular, Universidad Autónoma de Madrid, Nicolás Cabrera, 1, 28049 Madrid, Spain
2
Molecular Genetics Unit, Institute of Rare Diseases Research, Institute of Health Carlos III (ISCIII), Ctra. Majadahonda-Pozuelo Km 2,200, 28220 Madrid, Spain
3
Laboratorio de Apoyo a la Investigación, Hospital Universitario Fundación Alcorcón, Calle Budapest 1, 28922 Madrid, Spain
4
Karolinska Institutet, Department of Microbiology Tumor and Cell Biology, Solnaväjen 1, 171 77 Stockholm, Sweden
5
Department of Molecular Biology, Autonomous University of Madrid, Francisco Tomás y Valiente 7, 28049 Madrid, Spain
6
Department of Neurology, Hospital Universitario Gregorio Marañón, Dr. Esquerdo 46, 28007 Madrid, Spain
*
Author to whom correspondence should be addressed.
These authors contributed equally to this work.
Daniel Oberdoerfer is deceased. This paper is dedicated to his memory.
Int. J. Mol. Sci. 2020, 21(18), 6662; https://0-doi-org.brum.beds.ac.uk/10.3390/ijms21186662
Received: 26 August 2020 / Accepted: 8 September 2020 / Published: 11 September 2020
(This article belongs to the Collection Feature Papers in Molecular Neurobiology)
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. View Full-Text
Keywords: Frataxin; Friedreich´s ataxia; gene therapy; stem cells human olfactory mucosa Frataxin; Friedreich´s ataxia; gene therapy; stem cells human olfactory mucosa
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MDPI and ACS Style

Pérez-Luz, S.; Loria, F.; Katsu-Jiménez, Y.; Oberdoerfer, D.; Yang, O.-L.; Lim, F.; Muñoz-Blanco, J.L.; Díaz-Nido, J. Altered Secretome and ROS Production in Olfactory Mucosa Stem Cells Derived from Friedreich’s Ataxia Patients. Int. J. Mol. Sci. 2020, 21, 6662. https://0-doi-org.brum.beds.ac.uk/10.3390/ijms21186662

AMA Style

Pérez-Luz S, Loria F, Katsu-Jiménez Y, Oberdoerfer D, Yang O-L, Lim F, Muñoz-Blanco JL, Díaz-Nido J. Altered Secretome and ROS Production in Olfactory Mucosa Stem Cells Derived from Friedreich’s Ataxia Patients. International Journal of Molecular Sciences. 2020; 21(18):6662. https://0-doi-org.brum.beds.ac.uk/10.3390/ijms21186662

Chicago/Turabian Style

Pérez-Luz, Sara, Frida Loria, Yurika Katsu-Jiménez, Daniel Oberdoerfer, Oscar-Li Yang, Filip Lim, José L. Muñoz-Blanco, and Javier Díaz-Nido. 2020. "Altered Secretome and ROS Production in Olfactory Mucosa Stem Cells Derived from Friedreich’s Ataxia Patients" International Journal of Molecular Sciences 21, no. 18: 6662. https://0-doi-org.brum.beds.ac.uk/10.3390/ijms21186662

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