A novel CUG(exp)·MBNL1 inhibitor with therapeutic potential for myotonic dystrophy type 1.
about
A journey in bioinspired supramolecular chemistry: from molecular tweezers to small molecules that target myotonic dystrophyStructural studies of CNG repeatsStructure based approaches for targeting non-coding RNAs with small moleculesApproaches to Validate and Manipulate RNA Targets with Small Molecules in CellsBiological Efficacy and Toxicity of Diamidines in Myotonic Dystrophy Type 1 Models.Targeting toxic RNAs that cause myotonic dystrophy type 1 (DM1) with a bisamidinium inhibitor.MBNL proteins and their target RNAs, interaction and splicing regulation.Modifications to toxic CUG RNAs induce structural stability, rescue mis-splicing in a myotonic dystrophy cell model and reduce toxicity in a myotonic dystrophy zebrafish model.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.RNA Structures as Mediators of Neurological Diseases and as Drug Targets.Defining RNA-Small Molecule Affinity Landscapes Enables Design of a Small Molecule Inhibitor of an Oncogenic Noncoding RNAIn silico discovery of substituted pyrido[2,3-d]pyrimidines and pentamidine-like compounds with biological activity in myotonic dystrophy models.A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a Drosophila Model of Myotonic Dystrophy.Reduction of Huntington's Disease RNA Foci by CAG Repeat-Targeting Reagents.Finding needles in a basestack: recognition of mismatched base pairs in DNA by small molecules.Developmental insights into the pathology of and therapeutic strategies for DM1: Back to the basics.RNA mis-splicing in disease.Faulty RNA splicing: consequences and therapeutic opportunities in brain and muscle disorders.Small molecule kinase inhibitors alleviate different molecular features of myotonic dystrophy type 1.Small Molecule Recognition Triggers Secondary and Tertiary Interactions in DNA Folding and Hammerhead Ribozyme Catalysis.Small Molecules Which Improve Pathogenesis of Myotonic Dystrophy Type 1.
P2860
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P2860
A novel CUG(exp)·MBNL1 inhibitor with therapeutic potential for myotonic dystrophy type 1.
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
A novel CUG(exp)·MBNL1 inhibit ...... for myotonic dystrophy type 1.
@en
type
label
A novel CUG(exp)·MBNL1 inhibit ...... for myotonic dystrophy type 1.
@en
prefLabel
A novel CUG(exp)·MBNL1 inhibit ...... for myotonic dystrophy type 1.
@en
P2093
P2860
P356
P1433
P1476
A novel CUG(exp)·MBNL1 inhibit ...... for myotonic dystrophy type 1.
@en
P2093
Amin Haghighat Jahromi
Anne M Baranger
Kali A Miller
Lien Nguyen
Steven C Zimmerman
P2860
P304
P356
10.1021/CB400046U
P577
2013-03-20T00:00:00Z