Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy - Phase I-II

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dc.authoridEda Sun / 0000-003-0320-5784
dc.authoridErdal Karaöz / 0000-0002-9992-833X
dc.authorscopusidErdal Karaöz / 7003448087
dc.authorscopusidEda Sun / 57225054414
dc.authorwosidErdal Karaöz / IXE-6677-2023
dc.authorwosidEda Sun / GER-0120-2022
dc.contributor.authorDai, Alper
dc.contributor.authorBaşpınar, Osman
dc.contributor.authorYeşilyurt, Ahmet
dc.contributor.authorSun, Eda
dc.contributor.authorAydemir, Çiğdem İnci
dc.contributor.authorÖztel, Olga Nehir
dc.contributor.authorKaraöz, Erdal
dc.date.accessioned2020-08-30T20:07:44Z
dc.date.available2020-08-30T20:07:44Z
dc.date.issued2018
dc.departmentİstinye Üniversitesi, Tıp Fakültesi, Temel Tıp Bilimleri Bölümüen_US
dc.descriptionPubMed: 30498389en_US
dc.description.abstractPurpose: Duchenne muscular dystrophy (DMD) is an X-linked recessive pediatric disorder that ultimately leads to progressive muscle degeneration. It has been known that cell-based therapies were used to promote muscle regeneration. The main purpose of this study was to investigate the effects of allogeneic Wharton jelly-derived mesenchymal stem cells therapy in Duchenne muscular dystrophy. Patients and methods: Four ambulatory and five nonambulatory male patients were assessed as having acceptance criteria. Gene expression and immunohistochemical analysis were performed for dystrophin gene expression. The fluorescent in situ hybridization method was used for detection of chimerism and donor-recipient compatibility. Complement dependent lymphocyto-toxic crossmatch test and detection of panel reactive antigen were performed. All patients were treated with 2 x 10(6) cells/kg dose of allogeneic Wharton jelly-derived mesenchymal stem cells via intra-arterial and intramuscular administration. Stability was maintained in patient follow-up tests, which are respiratory capacity tests, cardiac measurements, and muscle strength tests. Results: The vastus intermedius muscle was observed in one patient with MRI. Chimerism was detected by fluorescent in situ hybridization and mean gene expression was increased to 3.3-fold. An increase in muscle strength measurements and pulmonary function tests was detected. Additionally, we observed two of nine patients with positive panel reactive antigen result. Conclusion: All our procedures are well tolerated, and we have not seen any application-related complications so far. Our main purpose of this study was to investigate the effects of allogeneic mesenchymal stem cell therapy and determine its suitability and safety as a form of treatment in this untreatable disorder.en_US
dc.description.sponsorshipLiv Hospitalen_US
dc.description.sponsorshipWe thank Vasif Haftaci from Isparta City Hospital for technical support and comments that greatly improved the manuscript. Moreover, we would like to thank Bahadir Edizer from Gaziantep University, Faculty of Medicine, for his support during the project. Also, we thank Liv Hospital that sponsored this research.en_US
dc.identifier.citationDai, A., Baspinar, O., Yeşilyurt, A., Sun, E., Aydemir, Ç. İ., Öztel, O. N., ... & Karaöz, E. (2018). Efficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy–Phase I–II. Degenerative neurological and neuromuscular disease, 8, 63.en_US
dc.identifier.doi10.2147/DNND.S170087en_US
dc.identifier.endpage77en_US
dc.identifier.issn1179-9900en_US
dc.identifier.pmid30498389en_US
dc.identifier.startpage63en_US
dc.identifier.urihttps://doi.org/10.2147/DNND.S170087
dc.identifier.urihttps://hdl.handle.net/20.500.12713/822
dc.identifier.volume8en_US
dc.identifier.wosWOS:000467905300001en_US
dc.identifier.wosqualityN/Aen_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakPubMeden_US
dc.institutionauthorSun, Edaen_US
dc.institutionauthorKaraöz, Erdalen_US
dc.language.isoenen_US
dc.publisherDove Medical Press Ltden_US
dc.relation.ispartofDegenerative Neurological and Neuromuscular Diseaseen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectDuchenne Muscular Dystrophyen_US
dc.subjectMesenchymal Stem Cellsen_US
dc.subjectPanel Reactive Antigenen_US
dc.subjectTreatmenten_US
dc.titleEfficacy of stem cell therapy in ambulatory and nonambulatory children with Duchenne muscular dystrophy - Phase I-IIen_US
dc.typeArticleen_US

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