Safety and efficacy of the stem cell transplantation in friedreich's ataxia: a report of three cases
Yükleniyor...
Dosyalar
Tarih
2021
Dergi Başlığı
Dergi ISSN
Cilt Başlığı
Yayıncı
IJPHY PUBLISHERS
Erişim Hakkı
info:eu-repo/semantics/openAccess
Özet
Background: 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.
Açıklama
Anahtar Kelimeler
Friedreich's Ataxia, Hereditary Ataxia, Cellular Treatment, Regenerative Medicine, Stem Cell
Kaynak
INTERNATIONAL JOURNAL OF PHYSIOTHERAPY
WoS Q Değeri
N/A
Scopus Q Değeri
Cilt
8
Sayı
1
Künye
Riza 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).
