Free-ordered CUG on Chemical Abstract Machine
نویسنده
چکیده
Sa~oshi Tojo Mitsub i sh i l{ese~rch In s t i t u t e , Inc. (e-ma,i]) t o jo@mri . co . jp
منابع مشابه
A novel CUG(exp)·MBNL1 inhibitor with therapeutic potential for myotonic dystrophy type 1.
Myotonic dystrophy type 1 (DM1) is caused by an expanded CUG repeat (CUG(exp)) that sequesters muscleblind-like 1 protein (MBNL1), a protein that regulates alternative splicing. CUG(exp) RNA is a validated drug target for this currently untreatable disease. Herein, we develop a bioactive small molecule (1) that targets CUG(exp) RNA and is able to inhibit the CUG(exp)·MBNL1 interaction in cells ...
متن کاملRationally designed small molecules that target both the DNA and RNA causing myotonic dystrophy type 1.
Single-agent, single-target therapeutic approaches are often limited by a complex disease pathobiology. We report rationally designed, multi-target agents for myotonic dystrophy type 1 (DM1). DM1 originates in an abnormal expansion of CTG repeats (CTG(exp)) in the DMPK gene. The resultant expanded CUG transcript (CUG(exp)) identified as a toxic agent sequesters important proteins, such as muscl...
متن کاملGenetic and Chemical Modifiers of a CUG Toxicity Model in Drosophila
Non-coding CUG repeat expansions interfere with the activity of human Muscleblind-like (MBNL) proteins contributing to myotonic dystrophy 1 (DM1). To understand this toxic RNA gain-of-function mechanism we developed a Drosophila model expressing 60 pure and 480 interrupted CUG repeats in the context of a non-translatable RNA. These flies reproduced aspects of the DM1 pathology, most notably nuc...
متن کاملRNA CUG repeats sequester CUGBP1 and alter protein levels and activity of CUGBP1.
An RNA CUG triplet repeat binding protein, CUGBP1, regulates splicing and translation of various RNAs. Expansion of RNA CUG repeats in the 3'-untranslated repeat of the mutant myotonin protein kinase (DMPK) mRNA in myotonic dystrophy (DM) is associated with alterations in binding activity of CUGBP1. To investigate whether CUGBP1 is directly affected by expansion of CUG repeats in DM tissues, we...
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