Frataxin deficiency alters gene expression in Friedreich ataxia derived IPSC‐neurons and cardiomyocytes

Frataxin deficiency alters gene expression in Friedreich ataxia derived IPSC‐neurons and cardiomyocytes

Abstract Background Friedreich's ataxia (FRDA) is an autosomal recessive disease, whereby homozygous inheritance of an expanded GAA trinucleotide repeat expansion in the first intron of the FXN gene leads to transcriptional repression of the encoded protein frataxin. FRDA is a progressive neuro...

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Bibliographic Details
Main Authors: Mariana B. Angulo, Alexander Bertalovitz, Mariana A. Argenziano, Jiajia Yang, Aarti Patel, Theresa Zesiewicz, Thomas V. McDonald
Format: Article
Language:English
Published: Wiley 2023-01-01
Series:Molecular Genetics & Genomic Medicine
Subjects:
extracellular matrix
frataxin
Friedreich's ataxia
glycolysis
lentivirus
Online Access:https://doi.org/10.1002/mgg3.2093

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