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March 19, 2010Current Chemical GenomicsOpen Access

HTS-Compatible Patient-Derived Cell-Based Assay to Identify Small Molecule Modulators of Aberrant Splicing in Myotonic Dystrophy Type 1

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Authors

DODebra A. O’LearySt Vincent's HospitalLVLeonardo VargasUniversidad de Santiago de ChileOSOrzala SharifTherapeutics Clinical Research

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O’Leary et al. (2010) studied this question.

synapsesocial.com/papers/6aa92e0215a756b5ce462494https://doi.org/10.2174/1875397301004010009
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Also Consider

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  1. 1MyoD1: A Nuclear Phosphoprotein Requiring a Myc Homology Region to Convert Fibroblasts to Myoblasts1988 · 869 citations
  2. 2Muscle Chloride Channel Dysfunction in Two Mouse Models of Myotonic Dystrophy2006 · 112 citations
  3. 3Disruption of Splicing Regulated by a CUG-Binding Protein in Myotonic Dystrophy1998 · 826 citations
  4. 4Overexpression of CUG Triplet Repeat-binding Protein, CUGBP1, in Mice Inhibits Myogenesis2004 · 204 citations
  5. 5Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA2009 · 389 citations