A case of non-progressive congenital myopathy: Efficacy and clinical outcomes of the wharton's jelly derived mesenchymal stem cell transplantation

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dc.authoridRıza Azeri / 0000-0002-1785-3371en_US
dc.authoridEda Sun / 0000-0003-0320-5784en_US
dc.authoridErdal Karaöz / 0000-0002-9992-833Xen_US
dc.authorscopusidRıza Azeri / 57669699400en_US
dc.authorscopusidEda Sun / 57225054414en_US
dc.authorscopusidErdal Karaöz / 7003448087en_US
dc.authorwosidRıza Azeri / EKW-6951-2022
dc.authorwosidEda Sun / AAO-1470-2020
dc.authorwosidErdal Karaöz / GER-0120-2022
dc.contributor.authorAzeri, Rıza
dc.contributor.authorSun, Eda
dc.contributor.authorKaraöz, Erdal
dc.date.accessioned2022-05-23T06:16:56Z
dc.date.available2022-05-23T06:16:56Z
dc.date.issued2022en_US
dc.departmentİstinye Üniversitesi, Sağlık Bilimleri Fakültesi, Fizyoterapi ve Rehabilitasyon Bölümüen_US
dc.description.abstractNon-Progressive Congenital Myopathy is a disease characterized by muscle weakness, and unfortunately, there is no conventional treatment. In the last decade, regenerative medicine practices have become a rising value, and Mesenchymal Stem Cells (MSCs) have fascinating outcomes in regenerative medicine with their high regenerative capacities, their ability to regulate with paracrine secretions, and their immunological properties. Based on our experience in our previous clinical studies, Wharton's-Jelly-derived (WJ-)MSCs are the most suitable source for muscle diseases among all MSC sources. In this study, we evaluated the outcomes of 10 doses of WJ-MSC transplantation to the patient diagnosed with Non-Progressive Congenital Myopathy. A 17-year-old female with a SPEN-1 mutation, Non-Progressive Congenital Myopathy patient received 10 times as 1×10?/kg in the intra-arterial, intramuscular and intravenous administration of allogenic WJ-MSC. Before and after the treatment, the patient was followed-up with the upper extremity scale, Vignos lower extremity scale, muscle strength scale, functional independence measure, and evaluation of Serum creatine kinase (CK) levels. Improvement in both upper extremity scale and Vignos lower extremity scales, increasing in muscle strength, and decreasing in CK-level were detected. Although transplantation of WJ-MSC cannot treat any genetic-based diseases, they may benefit in alleviating clinical outcomes of disease. More importantly, WJ-MSC transplantation may offer a better quality of life by alleviating the symptoms of this rare disease with no treatment option that can be provided in conventional methods. © 2022 Tehran University of Medical Sciences. All rights reserved.en_US
dc.identifier.citationAzeri, R., Sun, E., & Karaoz, E. (2022). A case of non-progressive congenital myopathy: Efficacy and clinical outcomes of the wharton's jelly derived mesenchymal stem cell transplantation. Acta Medica Iranica, 60(4), 249-253. doi:10.18502/acta.v60i4.9270en_US
dc.identifier.doi10.18502/acta.v60i4.9270en_US
dc.identifier.endpage253en_US
dc.identifier.issn0044-6025en_US
dc.identifier.issue4en_US
dc.identifier.scopus2-s2.0-85129527134en_US
dc.identifier.scopusqualityQ4en_US
dc.identifier.startpage249en_US
dc.identifier.urihttps://doi.org/10.18502/acta.v60i4.9270
dc.identifier.urihttps://hdl.handle.net/20.500.12713/2698
dc.identifier.volume60en_US
dc.indekslendigikaynakScopusen_US
dc.institutionauthorAzeri, Rıza
dc.institutionauthorSun, Eda
dc.institutionauthorKaraöz, Erdal
dc.language.isoenen_US
dc.publisherMedical Sciences University of Teheranen_US
dc.relation.ispartofActa Medica Iranicaen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectMesenchymal Stem Cellsen_US
dc.subjectMuscle Fibrosisen_US
dc.subjectMuscle-Fiber Regenerationen_US
dc.subjectNon-Progressive Congenital Myopathyen_US
dc.titleA case of non-progressive congenital myopathy: Efficacy and clinical outcomes of the wharton's jelly derived mesenchymal stem cell transplantationen_US
dc.typeArticleen_US

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