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

Pub. Date : 2010 Mar 19

PMID : 20502647






1 Functional Relationships(s)
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Compound Name
Protein Name
Organism
1 The DMPK transcripts containing expanded CUG repeats accumulate in nuclear foci and ultimately cause mis-splicing of secondary genes through the dysregulation of RNA-binding proteins including Muscleblind 1 (MBNL1) and CUG binding protein 1 (CUGBP1). 3-Carboxyumbelliferyl beta-D-galactopyranoside DM1 protein kinase Homo sapiens