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Frataxin deficiency alters gene expression in Friedreich ataxia derived IPSC‐neurons and cardiomyocytes
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 neurodegenera...
Autores principales: | Angulo, Mariana B., Bertalovitz, Alexander, Argenziano, Mariana A., Yang, Jiajia, Patel, Aarti, Zesiewicz, Theresa, McDonald, Thomas V. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9834160/ https://www.ncbi.nlm.nih.gov/pubmed/36369844 http://dx.doi.org/10.1002/mgg3.2093 |
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