A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding.
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A journey in bioinspired supramolecular chemistry: from molecular tweezers to small molecules that target myotonic dystrophyModeling ALS and FTD with iPSC-derived neuronsStructure based approaches for targeting non-coding RNAs with small moleculesNMR spectroscopy and molecular dynamics simulation of r(CCGCUGCGG)₂ reveal a dynamic UU internal loop found in myotonic dystrophy type 1.Crystal structures of CGG RNA repeats with implications for fragile X-associated tremor ataxia syndromeMyotonic Dystrophy Type 1 RNA Crystal Structures Reveal Heterogeneous 1 × 1 Nucleotide UU Internal Loop ConformationsBiological Efficacy and Toxicity of Diamidines in Myotonic Dystrophy Type 1 Models.Therapeutics development in myotonic dystrophy type 1.Single-molecule study of the CUG repeat-MBNL1 interaction and its inhibition by small molecules.Examining the interactions of the splicing factor MBNL1 with target RNA sequences via a label-free, multiplex methodStructure of the myotonic dystrophy type 2 RNA and designed small molecules that reduce toxicity.Computational investigation of RNA CUG repeats responsible for myotonic dystrophy 1Targeting toxic RNAs that cause myotonic dystrophy type 1 (DM1) with a bisamidinium inhibitor.Methods to enable the design of bioactive small molecules targeting RNA.Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects.The role of flexibility in the rational design of modularly assembled ligands targeting the RNAs that cause the myotonic dystrophiesOligonucleotide-based strategies to combat polyglutamine diseases.Reducing levels of toxic RNA with small moleculesIn vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy modelsTwo high-throughput screening assays for aberrant RNA-protein interactions in myotonic dystrophy type 1.Chemical correction of pre-mRNA splicing defects associated with sequestration of muscleblind-like 1 protein by expanded r(CAG)-containing transcriptsDesign of a bioactive small molecule that targets the myotonic dystrophy type 1 RNA via an RNA motif-ligand database and chemical similarity searching.RNA-protein interactions in unstable microsatellite diseases.MBNL proteins and their target RNAs, interaction and splicing regulation.Novel insights of structure-based modeling for RNA-targeted drug discovery.Targeting the r(CGG) repeats that cause FXTAS with modularly assembled small molecules and oligonucleotidesFeatures of modularly assembled compounds that impart bioactivity against an RNA target.Aberrant RNA homeostasis in amyotrophic lateral sclerosis: potential for new therapeutic targets?High-content screening identifies small molecules that remove nuclear foci, affect MBNL distribution and CELF1 protein levels via a PKC-independent pathway in myotonic dystrophy cell lines.Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1.Small molecules that target the toxic RNA in myotonic dystrophy type 2.Sequence-based design of bioactive small molecules that target precursor microRNAs.A dynamic combinatorial approach for identifying side groups that stabilize DNA-templated supramolecular self-assemblies.Selective inhibition of MBNL1-CCUG interaction by small molecules toward potential therapeutic agents for myotonic dystrophy type 2 (DM2)Triplet repeat RNA structure and its role as pathogenic agent and therapeutic targetNaphthyridine tetramer with a pre-organized structure for 1:1 binding to a CGG/CGG sequence.RNA Structures as Mediators of Neurological Diseases and as Drug Targets.Identification of Plant-derived Alkaloids with Therapeutic Potential for Myotonic Dystrophy Type IFrom dynamic combinatorial 'hit' to lead: in vitro and in vivo activity of compounds targeting the pathogenic RNAs that cause myotonic dystrophy.Defining RNA-Small Molecule Affinity Landscapes Enables Design of a Small Molecule Inhibitor of an Oncogenic Noncoding RNA
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A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding.
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
@pt
bilimsel makale
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scientific article published on 08 September 2009
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
A simple ligand that selective ...... inhibits MBNL protein binding.
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A simple ligand that selective ...... inhibits MBNL protein binding.
@nl
type
label
A simple ligand that selective ...... inhibits MBNL protein binding.
@en
A simple ligand that selective ...... inhibits MBNL protein binding.
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prefLabel
A simple ligand that selective ...... inhibits MBNL protein binding.
@en
A simple ligand that selective ...... inhibits MBNL protein binding.
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P2860
P356
P1476
A simple ligand that selective ...... inhibits MBNL protein binding
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P2093
Anne M Baranger
Sreenivasa Rao Ramisetty
P2860
P304
16068-16073
P356
10.1073/PNAS.0901824106
P407
P577
2009-09-08T00:00:00Z