Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1.

scientific article

Developing bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1. is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1021/JM400794Z
P932PMC publication ID3925341
P698PubMed publication ID24188018

P50authorSteven ZimmermanQ7615410
Kali A MillerQ86641508
P2093author name stringLien Nguyen
Yuan Fu
Anne M Baranger
Long M Luu
Amin Haghighat Jahromi
P2860cites workInvestigating the binding mode of an inhibitor of the MBNL1·RNA complex in myotonic dystrophy type 1 (DM1) leads to the unexpected discovery of a DNA-selective binderQ36546738
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Polyvalent Interactions in Biological Systems: Implications for Design and Use of Multivalent Ligands and InhibitorsQ88519588
Structural insights into CUG repeats containing the 'stretched U-U wobble': implications for myotonic dystrophyQ27655444
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Colocalization of muscleblind with RNA foci is separable from mis-regulation of alternative splicing in myotonic dystrophyQ28257074
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New function for the RNA helicase p68/DDX5 as a modifier of MBNL1 activity on expanded CUG repeatsQ30427374
CUGBP1 overexpression in mouse skeletal muscle reproduces features of myotonic dystrophy type 1.Q30496276
Single-molecule study of the CUG repeat-MBNL1 interaction and its inhibition by small molecules.Q30541488
Bis-acridines as lead antiparasitic agents: structure-activity analysis of a discrete compound library in vitroQ33279371
Rational and modular design of potent ligands targeting the RNA that causes myotonic dystrophy 2.Q33427148
Rational design of ligands targeting triplet repeating transcripts that cause RNA dominant disease: application to myotonic muscular dystrophy type 1 and spinocerebellar ataxia type 3.Q33474085
Synthesis and biological evaluation of bivalent ligands for the cannabinoid 1 receptor.Q33693780
In vivo discovery of a peptide that prevents CUG-RNA hairpin formation and reverses RNA toxicity in myotonic dystrophy modelsQ33951409
Bivalent diketopiperazine-based tropomysin receptor kinase C (TrkC) antagonistsQ34010191
Fluorescence characteristics of 5-carboxytetramethylrhodamine linked covalently to the 5' end of oligonucleotides: multiple conformers of single-stranded and double-stranded dye-DNA complexesQ34040387
The structural basis of myotonic dystrophy from the crystal structure of CUG repeatsQ34133251
Design of a bioactive small molecule that targets the myotonic dystrophy type 1 RNA via an RNA motif-ligand database and chemical similarity searchingQ34149072
Rationally designed small molecules targeting the RNA that causes myotonic dystrophy type 1 are potently bioactiveQ34158462
Design strategies for bivalent ligands targeting GPCRsQ34180510
RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1Q34257340
Targeting nuclear RNA for in vivo correction of myotonic dystrophyQ34459257
Identification of MBNL1 and MBNL3 domains required for splicing activation and repressionQ34779359
Acridine derivatives as chemotherapeutic agentsQ34780011
Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophyQ34984541
RNA Foci, CUGBP1, and ZNF9 are the primary targets of the mutant CUG and CCUG repeats expanded in myotonic dystrophies type 1 and type 2Q35474587
Using modularly assembled ligands to bind RNA internal loops separated by different distancesQ36045311
From dynamic combinatorial 'hit' to lead: in vitro and in vivo activity of compounds targeting the pathogenic RNAs that cause myotonic dystrophyQ36107041
Increased steady-state levels of CUGBP1 in myotonic dystrophy 1 are due to PKC-mediated hyperphosphorylationQ36154872
P433issue23
P407language of work or nameEnglishQ1860
P921main subjectmyotonic dystrophyQ1860507
P304page(s)9471-9481
P577publication date2013-11-21
P1433published inJournal of Medicinal ChemistryQ900316
P1476titleDeveloping bivalent ligands to target CUG triplet repeats, the causative agent of myotonic dystrophy type 1
P478volume56

Reverse relations

cites work (P2860)
Q51832854A Ligand That Targets CUG Trinucleotide Repeats.
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Q45344868A general model for predicting the binding affinity of reversibly and irreversibly dimerized ligands.
Q26766064A journey in bioinspired supramolecular chemistry: from molecular tweezers to small molecules that target myotonic dystrophy
Q99553165Design of small molecules targeting RNA structure from sequence
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Q58548529Drugging the RNA World
Q36392989In silico discovery of substituted pyrido[2,3-d]pyrimidines and pentamidine-like compounds with biological activity in myotonic dystrophy models.
Q93039359Intrinsically cell-penetrating multivalent and multitargeting ligands for myotonic dystrophy type 1
Q33698593Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects
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Q26783672Modeling ALS and FTD with iPSC-derived neurons
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Q35868044RNA Structures as Mediators of Neurological Diseases and as Drug Targets
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Q37723428Reduction of Huntington's Disease RNA Foci by CAG Repeat-Targeting Reagents
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Q26858823Structure based approaches for targeting non-coding RNAs with small molecules
Q90153443Synthesis of Menthol Glycinates and Their Potential as Cooling Agents
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Q33588831Targeting toxic RNAs that cause myotonic dystrophy type 1 (DM1) with a bisamidinium inhibitor
Q64084474The TwistDock workflow for evaluation of bivalent Smac mimetics targeting XIAP
Q93074975Unveiling the druggable RNA targets and small molecule therapeutics

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