Search Results - "Jenquin, Jana R."
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Mitigating RNA Toxicity in Myotonic Dystrophy using Small Molecules
Published in International journal of molecular sciences (17-08-2019)“…This review, one in a series on myotonic dystrophy (DM), is focused on the development and potential use of small molecules as therapeutics for DM. The complex…”
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Journal Article -
2
A CTG repeat-selective chemical screen identifies microtubule inhibitors as selective modulators of toxic CUG RNA levels
Published in Proceedings of the National Academy of Sciences - PNAS (15-10-2019)“…A CTG repeat expansion in the DMPK gene is the causative mutation of myotonic dystrophy type 1 (DM1). Transcription of the expanded CTG repeat produces toxic…”
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Journal Article -
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Combination Treatment of Erythromycin and Furamidine Provides Additive and Synergistic Rescue of Mis-splicing in Myotonic Dystrophy Type 1 Models
Published in ACS pharmacology & translational science (09-08-2019)“…Myotonic dystrophy type 1 (DM1) is a multisystemic disease that presents with clinical symptoms including myotonia, cardiac dysfunction, and cognitive…”
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Journal Article -
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Molecular characterization of myotonic dystrophy fibroblast cell lines for use in small molecule screening
Published in iScience (20-05-2022)“…Myotonic dystrophy type 1 (DM1) and type 2 (DM2) are common forms of adult onset muscular dystrophy. Pathogenesis in both diseases is largely driven by…”
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Journal Article -
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Furamidine Rescues Myotonic Dystrophy Type I Associated Mis-Splicing through Multiple Mechanisms
Published in ACS chemical biology (21-09-2018)“…Myotonic dystrophy type 1 (DM1) is an autosomal dominant, CTG•CAG microsatellite expansion disease. Expanded CUG repeat RNA sequester the muscleblind-like…”
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Journal Article -
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Small Molecule Approaches to Reduce the Effects of Toxic Repeat RNA in Myotonic Dystrophy Models
Published 01-01-2019“…Myotonic dystrophy type 1 (DM1) is an autosomal dominant, microsatellite expansion disease that presents with clinical symptoms including myotonia, cardiac…”
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Dissertation