SINEUP non-coding RNAs rescue defective frataxin expression and activity in a cellular model of Friedreich's Ataxia

Friedreich's ataxia (FRDA) is an untreatable disorder with neuro- and cardio-degenerative progression. This monogenic disease is caused by the hyper-expansion of naturally occurring GAA repeats in the first intron of the FXN gene, encoding for frataxin, a protein implicated in the biogenesis of...

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Bibliographic Details
Published in:Nucleic Acids Res
Main Authors: Bon, Carlotta, Luffarelli, Riccardo, Russo, Roberta, Fortuni, Silvia, Pierattini, Bianca, Santulli, Chiara, Fimiani, Cristina, Persichetti, Francesca, Cotella, Diego, Mallamaci, Antonello, Santoro, Claudio, Carninci, Piero, Espinoza, Stefano, Testi, Roberto, Zucchelli, Silvia, Condò, Ivano, Gustincich, Stefano
Format: Artigo
Language:Inglês
Published: Oxford University Press 2019
Subjects:
Molecular Biology
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6847766/
https://ncbi.nlm.nih.gov/pubmed/31584077
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkz798
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