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Oral administration of erythromycin decreases RNA toxicity in myotonic dystrophy

OBJECTIVE: Myotonic dystrophy type 1 (DM1) is caused by the expansion of a CTG repeat in the 3′ untranslated region of DMPK. The transcripts containing an expanded CUG repeat (CUG (exp)) result in a toxic gain‐of‐function by forming ribonuclear foci that sequester the alternative splicing factor mus...

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Bibliografische gegevens
Gepubliceerd in:Ann Clin Transl Neurol
Hoofdauteurs: Nakamori, Masayuki, Taylor, Katarzyna, Mochizuki, Hideki, Sobczak, Krzysztof, Takahashi, Masanori P.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: John Wiley and Sons Inc. 2015
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4704483/
https://ncbi.nlm.nih.gov/pubmed/26783549
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.271
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