Safety and efficacy of the stem cell transplantation in friedreich's ataxia: a report of three cases

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dc.authoridRıza Azeri / 0000-0002-1785-3371
dc.authoridEda Sun / 0000-0003-0320-5784
dc.authoridErdal Karaöz / 0000-0002-9992-833X
dc.authoridDuygu Koyuncu Irmak / 0000-0002-3277-5535
dc.authorscopusidRıza Azeri / 57669699400
dc.authorscopusidEda Sun / 57225054414
dc.authorscopusidErdal Karaöz / 7003448087
dc.authorscopusidDuygu Koyuncu Irmak / 57219539042
dc.authorwosidRıza Azeri / EKW-6951-2022
dc.authorwosidEda Sun / AAO-1470-2020
dc.authorwosidErdal Karaöz / GER-0120-2022
dc.contributor.authorAzeri, Rıza
dc.contributor.authorIrmak, Duygu Koyuncu
dc.contributor.authorSun, Eda
dc.contributor.authorKaraöz, Erdal
dc.date.accessioned2021-06-01T06:06:50Z
dc.date.available2021-06-01T06:06:50Z
dc.date.issued2021en_US
dc.departmentİstinye Üniversitesi, Sağlık Bilimleri Fakültesi, Fizyoterapi ve Rehabilitasyon Bölümüen_US
dc.description.abstractBackground: Friedreich's ataxia is a progressive degenerative disorder caused by deficiency of the frataxin protein. Expanded GAA repeats in intron 1 of the FXN gene lead to its heterochromatinization and transcriptional silencing. Strategies being trialed to treat Friedreich's ataxia include drugs that improve mitochondrial function and reduce oxidative injury. It has been shown that mesenchymal stem cell (MSC)-derived factors can restore cellular homeostasis and function to frataxin deficient cells. Case Summary: Here, we report three FRDA cases treated with four consecutive allogeneic transplantations of umbilical cord-derived MSCs with 30 days interval, upon per patient regulatory approvals for advanced cellular therapy. Outcome Measures: The cases were followed up after the treatment in means of the therapeutic effect of the cellular treatment by attenuating the neurological findings and gene expression parameters. Conclusions: Closely followed promising safety and efficacy outcomes demonstrated that the MSC treatment for FRDA might positively affect the clinical results caused by the defect in this genetic-based disease.en_US
dc.identifier.citationRiza Azeri, M. D. (2021). Safety and efficacy of the stem cell transplantation in friedreich's ataxia: a report of three cases. INTERNATIONAL JOURNAL OF PHYSIOTHERAPY, 8(1).en_US
dc.identifier.doi10.15621/ijphy/2021/v8i1/903en_US
dc.identifier.endpage35en_US
dc.identifier.issn2349-5987en_US
dc.identifier.issn2348-8336en_US
dc.identifier.issue1en_US
dc.identifier.startpage31en_US
dc.identifier.urihttps://www.doi.org/10.15621/ijphy/2021/v8i1/903
dc.identifier.urihttps://hdl.handle.net/20.500.12713/1773
dc.identifier.volume8en_US
dc.identifier.wosWOS:000647604600006en_US
dc.identifier.wosqualityN/Aen_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.institutionauthorAzeri, Rıza
dc.institutionauthorKoyuncu Irmak, Duygu
dc.institutionauthorSun, Eda
dc.institutionauthorKaraöz, Erdal
dc.language.isoenen_US
dc.publisherIJPHY PUBLISHERSen_US
dc.relation.ispartofINTERNATIONAL JOURNAL OF PHYSIOTHERAPYen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectFriedreich's Ataxiaen_US
dc.subjectHereditary Ataxiaen_US
dc.subjectCellular Treatmenten_US
dc.subjectRegenerative Medicineen_US
dc.subjectStem Cellen_US
dc.titleSafety and efficacy of the stem cell transplantation in friedreich's ataxia: a report of three casesen_US
dc.typeArticleen_US

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