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Targeting 3′ and 5′ untranslated regions with antisense oligonucleotides to stabilize frataxin mRNA and increase protein expression

Friedreich’s ataxia (FRDA) is a severe multisystem disease caused by transcriptional repression induced by expanded GAA repeats located in intron 1 of the Frataxin (FXN) gene encoding frataxin. FRDA results from decreased levels of frataxin; thus, stabilization of the FXN mRNA already present in pat...

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Detalles Bibliográficos
Autores principales:	Li, Yanjie, Li, Jixue, Wang, Jun, Lynch, David R, Shen, Xiulong, R. Corey, David, Parekh, Darshan, Bhat, Balkrishen, Woo, Caroline, Cherry, Jonathan J, Napierala, Jill S, Napierala, Marek
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2021
Materias:	Gene regulation, Chromatin and Epigenetics
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8599914/ https://www.ncbi.nlm.nih.gov/pubmed/34718736 http://dx.doi.org/10.1093/nar/gkab954

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