Combination Treatment of Erythromycin and Furamidine Provides Additive and Synergistic Rescue of Mis-splicing in Myotonic Dystrophy Type 1 Models

[Image: see text] Myotonic dystrophy type 1 (DM1) is a multisystemic disease that presents with clinical symptoms including myotonia, cardiac dysfunction, and cognitive impairment. DM1 is caused by a CTG expansion in the 3′ UTR of the DMPK gene. The transcribed expanded CUG-repeat RNA sequester the...

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Bibliographic Details
Published in:ACS Pharmacol Transl Sci
Main Authors: Jenquin, Jana R., Yang, Hongfen, Huigens III, Robert W., Nakamori, Masayuki, Berglund, J. Andrew
Format: Artigo
Language:Inglês
Published: American Chemical Society 2019
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6726129/
https://ncbi.nlm.nih.gov/pubmed/31485578
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acsptsci.9b00020
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