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Repeat RNA expansion disorders of the nervous system: post-transcriptional mechanisms and therapeutic strategies

Dozens of incurable neurological disorders result from expansion of short repeat sequences in both coding and non-coding regions of the transcriptome. Short repeat expansions underlie microsatellite repeat expansion (MRE) disorders including myotonic dystrophy (DM1, CUG(50–3,500) in DMPK; DM2, CCTG(...

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Bibliografische gegevens
Gepubliceerd in:Crit Rev Biochem Mol Biol
Hoofdauteurs: Schwartz, Joshua L., Jones, Krysten Leigh, Yeo, Gene W.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2020
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8192115/
https://ncbi.nlm.nih.gov/pubmed/33172304
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/10409238.2020.1841726
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