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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

http://www.wikidata.org/entity/Q34459257

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Identification of sequences common to more than one therapeutic target to treat complex diseases: simulating the high variance in sequence interactivity evolved to modulate robust phenotypes Facioscapulohumeral dystrophy: the path to consensus on pathophysiology RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention Advanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the Brain Congenital and childhood myotonic dystrophy: Current aspects of disease and future directions Modeling ALS and FTD with iPSC-derived neurons RNA-Binding Proteins: Splicing Factors and Disease Long noncoding RNAs-related diseases, cancers, and drugs A chemical view of oligonucleotides for exon skipping and related drug applications Noncoding RNAs: emerging players in muscular dystrophies RNA-binding proteins in neurodegeneration: Seq and you shall receive Congenital muscular dystrophy: from muscle to brain The unstable repeats--three evolving faces of neurological disease Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides.Long non-coding RNA regulation of epithelial-mesenchymal transition in cancer metastasis Noncoding RNA in age-related cardiovascular diseases G-quadruplexes: Emerging roles in neurodegenerative diseases and the non-coding transcriptome Oral administration of erythromycin decreases RNA toxicity in myotonic dystrophy Cell membrane integrity in myotonic dystrophy type 1: implications for therapy Approaches to Validate and Manipulate RNA Targets with Small Molecules in Cells TWEAK/Fn14, a pathway and novel therapeutic target in myotonic dystrophy.Report on the 3rd Ottawa International Conference on Neuromuscular Biology, Disease and Therapy - September 24-26, 2015, Ottawa, Canada.Biological Efficacy and Toxicity of Diamidines in Myotonic Dystrophy Type 1 Models.Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy.The alternative heart: impact of alternative splicing in heart disease.Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness.Ultrasound-enhanced delivery of morpholino with Bubble liposomes ameliorates the myotonia of myotonic dystrophy model mice Single-molecule study of the CUG repeat-MBNL1 interaction and its inhibition by small molecules.Structure of the myotonic dystrophy type 2 RNA and designed small molecules that reduce toxicity.Antisense oligonucleotides delivered to the amniotic cavity in utero modulate gene expression in the postnatal mouse.Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice.Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds.Targeting toxic RNAs that cause myotonic dystrophy type 1 (DM1) with a bisamidinium inhibitor.The UK Myotonic Dystrophy Patient Registry: facilitating and accelerating clinical research Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects.RNA metabolism in neurodegenerative disease.Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice Intracellular Distribution and Nuclear Activity of Antisense Oligonucleotides After Unassisted Uptake in Myoblasts and Differentiated Myotubes In Vitro.Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

http://www.wikidata.org/entity/Q34459257

description

2012 nî lūn-bûn

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2012 թուականի Օգոստոսին հրատարակուած գիտական յօդուած

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2012 թվականի օգոստոսին հրատարակված գիտական հոդված

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2012年の論文

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2012年論文

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2012年論文

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2012年論文

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2012年論文

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2012年論文

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2012年论文

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name

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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type

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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prefLabel

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

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A Robert MacLeod

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Andrew J Leger

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Bruce M Wentworth

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C Frank Bennett

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Charles A Thornton

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Sanjay K Pandey

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Seng H Cheng

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Thurman M Wheeler

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Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy

Failure of MBNL1-dependent post-natal splicing transitions in myotonic dystrophy

Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS

3' end processing of a long nuclear-retained noncoding RNA yields a tRNA-like cytoplasmic RNA

Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

Translation of stable hepadnaviral mRNA cleavage fragments induced by the action of phosphorothioate-modified antisense oligodeoxynucleotides

Principal components analysis to summarize microarray experiments: application to sporulation time series

Regulating gene expression through RNA nuclear retention

A muscleblind knockout model for myotonic dystrophy

Nucleocytoplasmic shuttling: a novel in vivo property of antisense phosphorothioate oligodeoxynucleotides.

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111-115

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10.1038/NATURE11362

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