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Progress towards drug discovery for Friedreich’s Ataxia: Identifying synthetic oligonucleotides that more potently activate expression of human frataxin protein

Friedreich’s Ataxia (FRDA) is an incurable genetic disease caused by an expanded trinucleotide AAG repeat within intronic RNA of the frataxin (FXN) gene. We have previously demonstrated that synthetic antisense oligonucleotides or duplex RNAs that are complementary to the expanded repeat can activat...

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Detalhes bibliográficos
Publicado no:Bioorg Med Chem
Main Authors: Shen, Xiulong, Wong, Johnathan, Prakash, Thahza P., Rigo, Frank, Li, Yanjie, Napierala, Marek, Corey, David R.
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7217746/
https://ncbi.nlm.nih.gov/pubmed/32279920
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bmc.2020.115472
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