Sodium Channel Inhibitors Reduce DMPK mRNA and Protein.
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ABSTRACT:
SUBMITTER: Witherspoon L
PROVIDER: S-EPMC5351028 | biostudies-literature | 2015 Aug
REPOSITORIES: biostudies-literature
Publications
Sodium Channel Inhibitors Reduce DMPK mRNA and Protein.
Witherspoon Luke L O'Reilly Sean S Hadwen Jeremiah J Tasnim Nafisa N MacKenzie Alex A Farooq Faraz F
Clinical and translational science 20150522 4
Myotonic dystrophy type 1 (DM1) is caused by an expanded trinucleotide (CTG)n tract in the 3' untranslated region (UTR) of the dystrophia myotonica protein kinase (DMPK) gene. This results in the aggregation of an expanded mRNA forming toxic intranuclear foci which sequester splicing factors. We believe down-regulation of DMPK mRNA represents a potential, and as yet unexplored, DM1 therapeutic avenue. Consequently, a computational screen for agents which down-regulate DMPK mRNA was undertaken, u ...[more]
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