Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules.
about
Studying a Drug-like, RNA-Focused Small Molecule Library Identifies Compounds That Inhibit RNA Toxicity in Myotonic DystrophySmall Molecule Inhibition of miR-544 Biogenesis Disrupts Adaptive Responses to Hypoxia by Modulating ATM-mTOR SignalingStacking in RNA: NMR of Four Tetramers Benchmark Molecular DynamicsStructure of the myotonic dystrophy type 2 RNA and designed small molecules that reduce toxicity.Computational investigation of RNA CUG repeats responsible for myotonic dystrophy 1Autoregulation of MBNL1 function by exon 1 exclusion from MBNL1 transcript.Targeting 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.RNA-protein interactions in unstable microsatellite diseases.MBNL proteins and their target RNAs, interaction and splicing regulation.Splicing landscape of the eight collaborative cross founder strains.Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy.RNA Structures as Mediators of Neurological Diseases and as Drug Targets.On the Applicability of Elastic Network Models for the Study of RNA CUG Trinucleotide Repeat Overexpansion.Precise small-molecule recognition of a toxic CUG RNA repeat expansion.In silico discovery of substituted pyrido[2,3-d]pyrimidines and pentamidine-like compounds with biological activity in myotonic dystrophy models.Splicing misregulation of SCN5A contributes to cardiac-conduction delay and heart arrhythmia in myotonic dystrophy.A Potent Inhibitor of Protein Sequestration by Expanded Triplet (CUG) Repeats that Shows Phenotypic Improvements in a Drosophila Model of Myotonic Dystrophy.Rational design of chemical genetic probes of RNA function and lead therapeutics targeting repeating transcripts.Developmental insights into the pathology of and therapeutic strategies for DM1: Back to the basics.The Emerging Role of RNA as a Therapeutic Target for Small Molecules.Development of pharmacophore models for small molecules targeting RNA: Application to the RNA repeat expansion in myotonic dystrophy type 1.RNA mis-splicing in disease.Hypo- and Hyper-Assembly Diseases of RNA-Protein Complexes.Dynamic changes of nuclear RNA foci in proliferating DM1 cells.Development of a Drosophila melanogaster spliceosensor system for in vivo high-throughput screening in myotonic dystrophy type 1.Hybrid splicing minigene and antisense oligonucleotides as efficient tools to determine functional protein/RNA interactions.Design of a "Mini" Nucleic Acid Probe for Cooperative Binding of an RNA-Repeated Transcript Associated with Myotonic Dystrophy Type 1.Using Genome Sequence to Enable the Design of Medicines and Chemical Probes.Design of Bivalent Nucleic Acid Ligands for Recognition of RNA-Repeated Expansion Associated with Huntington's Disease.[Deregulation of pre-messenger RNA splicing and rare diseases].Daunorubicin reduces MBNL1 sequestration caused by CUG-repeat expansion and rescues cardiac dysfunctions in a Drosophila model of myotonic dystrophy.Morpholino Antisense Oligomers as a Potential Therapeutic Option for the Correction of Alternative Splicing in PMD, SPG2, and HEMS.Alteration of RNA Splicing by Small-Molecule Inhibitors of the Interaction between NHP2L1 and U4Insights into the development of chemical probes for RNA
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P2860
Induction and reversal of myotonic dystrophy type 1 pre-mRNA splicing defects by small molecules.
description
2013 nî lūn-bûn
@nan
2013 թուականի Յունուարին հրատարակուած գիտական յօդուած
@hyw
2013 թվականի հունվարին հրատարակված գիտական հոդված
@hy
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
name
Induction and reversal of myot ...... ng defects by small molecules.
@ast
Induction and reversal of myot ...... ng defects by small molecules.
@en
Induction and reversal of myot ...... ng defects by small molecules.
@nl
type
label
Induction and reversal of myot ...... ng defects by small molecules.
@ast
Induction and reversal of myot ...... ng defects by small molecules.
@en
Induction and reversal of myot ...... ng defects by small molecules.
@nl
prefLabel
Induction and reversal of myot ...... ng defects by small molecules.
@ast
Induction and reversal of myot ...... ng defects by small molecules.
@en
Induction and reversal of myot ...... ng defects by small molecules.
@nl
P2093
P2860
P50
P356
P1476
Induction and reversal of myot ...... ing defects by small molecules
@en
P2093
Catherine Z Chen
Charles A Thornton
Chris P Austin
HaJeung Park
Ilyas Yildirim
Jessica L Childs-Disney
Juan J Marugan
Krzysztof Sobczak
Matthew D Disney
Samarjit Patnaik
P2860
P2888
P356
10.1038/NCOMMS3044
P407
P577
2013-01-01T00:00:00Z
P5875
P6179
1018823268