In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy models
Author:
Publisher
Proceedings of the National Academy of Sciences
Subject
Multidisciplinary
Reference35 articles.
1. Molecular Effects of the CTG Repeats in Mutant Dystrophia Myotonica Protein Kinase Gene
2. HnRNP H inhibits nuclear export of mRNA containing expanded CUG repeats and a distal branch point sequence
3. Recruitment of human muscleblind proteins to (CUG)n expansions associated with myotonic dystrophy
4. Three proteins, MBNL, MBLL and MBXL, co-localize in vivo with nuclear foci of expanded-repeat transcripts in DM1 and DM2 cells
5. RNA Leaching of Transcription Factors Disrupts Transcription in Myotonic Dystrophy
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1. NMR structures of small molecules bound to a model of a CUG RNA repeat expansion;Bioorganic & Medicinal Chemistry Letters;2024-10
2. NMR structures of small molecules bound to a model of an RNA CUG repeat expansion;2024-06-22
3. Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing;International Journal of Molecular Sciences;2022-04-21
4. Reversal of RNA toxicity in myotonic dystrophy via a decoy RNA-binding protein with high affinity for expanded CUG repeats;Nature Biomedical Engineering;2022-02-10
5. Brain Pathogenesis and Potential Therapeutic Strategies in Myotonic Dystrophy Type 1;Frontiers in Aging Neuroscience;2021-11-15
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