Safety and Efficacy of The Stem Cell Transplantation in Friedreich’s Ataxia: A Report of Three Cases
Abstract
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...
Paper Details
Title
Safety and Efficacy of The Stem Cell Transplantation in Friedreich’s Ataxia: A Report of Three Cases
Published Date
Feb 1, 2021
Volume
8
Issue
1
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