Chemical reversal of the RNA gain of function in myotonic dystrophy.
Cooper
Thomas A
TA
Department of Pathology and Molecular, Baylor College of Medicine, Houston, TX 77030, USA. tcooper@bcm.tmc.edu
D017398:Alternative Splicing / Q000187:drug effects* / Q000235:genetics; D000818:Animals; D004195:Disease Models, Animal; D006801:Humans; D051379:Mice; D009223:Myotonic Dystrophy / Q000188:drug therapy / Q000235:genetics*; D010419:Pentamidine / Q000737:chemistry / Q000494:pharmacology* / Q000627:therapeutic use; D012322:RNA Precursors / Q000235:genetics*; D016601:RNA-Binding Proteins / Q000235:genetics / Q000378:metabolism; D019680:Trinucleotide Repeat Expansion / Q000187:drug effects / Q000235:genetics
C493663:MBNL1 protein, human; D012322:RNA Precursors; D016601:RNA-Binding Proteins; D010419:Pentamidine