Activating frataxin expression by single-stranded siRNAs targeting the GAA repeat expansion

Friedreich’s ataxia (FRDA) is an incurable neurodegenerative disorder caused by reduced expression of the mitochondrial protein frataxin (FXN). The genetic cause of the disease is an expanded GAA repeat within the FXN gene. Agents that increase expression of FXN protein are a potential approach to t...

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Gepubliceerd in:Bioorg Med Chem Lett
Hoofdauteurs: Shen, Xiulong, Kilikevicius, Audrius, O’Reilly, Daniel, Prakash, Thazha P., Damha, Masad J., Rigo, Frank, Corey, David R.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2018
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6129981/
https://ncbi.nlm.nih.gov/pubmed/30076049
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bmcl.2018.07.033
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