The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models.
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Molecular Mechanisms of Neurodegeneration in Spinal Muscular AtrophySpinal muscular atrophy: development and implementation of potential treatmentsCollaboration for rare disease drug discovery researchLoss of the scavenger mRNA decapping enzyme DCPS causes syndromic intellectual disability with neuromuscular defectsProtective effects of butyrate-based compounds on a mouse model for spinal muscular atrophy.The effect of diet on the protective action of D156844 observed in spinal muscular atrophy mice.A short antisense oligonucleotide ameliorates symptoms of severe mouse models of spinal muscular atrophyQuality control of assembly-defective U1 snRNAs by decapping and 5'-to-3' exonucleolytic digestion.Assays for the identification and prioritization of drug candidates for spinal muscular atrophyTranscriptome profiling of spinal muscular atrophy motor neurons derived from mouse embryonic stem cellsGEMINs: potential therapeutic targets for spinal muscular atrophy?Presynaptic localization of Smn and hnRNP R in axon terminals of embryonic and postnatal mouse motoneuronsSpinal Muscular Atrophy Therapeutics: Where do we Stand?Elimination of cap structures generated by mRNA decay involves the new scavenger mRNA decapping enzyme Aph1/FHIT together with DcpS.A cell system for phenotypic screening of modifiers of SMN2 gene expression and function.Spinal muscular atrophy: from tissue specificity to therapeutic strategiesAdvances in therapeutic development for spinal muscular atrophy.ECG in neonate mice with spinal muscular atrophy allows assessment of drug efficacy.A library approach to rapidly discover photoaffinity probes of the mRNA decapping scavenger enzyme DcpS.Disease mechanisms and therapeutic approaches in spinal muscular atrophy.DcpS is a transcript-specific modulator of RNA in mammalian cells.The effect of the DcpS inhibitor D156844 on the protective action of follistatin in mice with spinal muscular atrophy.A fluorescent HTS assay for phosphohydrolases based on nucleoside 5'-fluorophosphates: its application in screening for inhibitors of mRNA decapping scavenger and PDE-I.Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.Systemic, postsymptomatic antisense oligonucleotide rescues motor unit maturation delay in a new mouse model for type II/III spinal muscular atrophy.Genetic inhibition of JNK3 ameliorates spinal muscular atrophy.Spinal muscular atrophy: an update on therapeutic progressDeveloping therapies for spinal muscular atrophy.Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy.Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy.The human decapping scavenger enzyme DcpS modulates microRNA turnover.An excess of catalytically required motions inhibits the scavenger decapping enzyme.The effects of C5-substituted 2,4-diaminoquinazolines on selected transcript expression in spinal muscular atrophy cellsIn vitro and in vivo effects of 2,4 diaminoquinazoline inhibitors of the decapping scavenger enzyme DcpS: Context-specific modulation of SMN transcript levels.Genome-wide CRISPR-Cas9 Screen Identifies Leukemia-Specific Dependence on a Pre-mRNA Metabolic Pathway Regulated by DCPS.Cellular thermal shift and clickable chemical probe assays for the determination of drug-target engagement in live cells
P2860
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P2860
The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models.
description
2013 nî lūn-bûn
@nan
2013 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2013 թվականի հունիսին 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
The DcpS inhibitor RG3039 impr ...... ogies in two SMA mouse models.
@ast
The DcpS inhibitor RG3039 impr ...... ogies in two SMA mouse models.
@en
type
label
The DcpS inhibitor RG3039 impr ...... ogies in two SMA mouse models.
@ast
The DcpS inhibitor RG3039 impr ...... ogies in two SMA mouse models.
@en
prefLabel
The DcpS inhibitor RG3039 impr ...... ogies in two SMA mouse models.
@ast
The DcpS inhibitor RG3039 impr ...... ogies in two SMA mouse models.
@en
P2093
P2860
P356
P1476
The DcpS inhibitor RG3039 impr ...... ogies in two SMA mouse models.
@en
P2093
Carina Emery
Christine J DiDonato
Courtenay Barlow
Heather L Plasterer
Herminio Cardona
James Rusche
Jasbir Singh
Jill Jarecki
Madel Durens
P2860
P304
P356
10.1093/HMG/DDT258
P577
2013-06-04T00:00:00Z