Targeting nuclear RNA for in vivo correction of myotonic dystrophy
about
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 phenotypesFacioscapulohumeral dystrophy: the path to consensus on pathophysiologyRNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense interventionAdvanced In vivo Use of CRISPR/Cas9 and Anti-sense DNA Inhibition for Gene Manipulation in the BrainCongenital and childhood myotonic dystrophy: Current aspects of disease and future directionsModeling ALS and FTD with iPSC-derived neuronsRNA-Binding Proteins: Splicing Factors and DiseaseLong noncoding RNAs-related diseases, cancers, and drugsA chemical view of oligonucleotides for exon skipping and related drug applicationsNoncoding RNAs: emerging players in muscular dystrophiesRNA-binding proteins in neurodegeneration: Seq and you shall receiveCongenital muscular dystrophy: from muscle to brainThe unstable repeats--three evolving faces of neurological diseaseReversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides.Long non-coding RNA regulation of epithelial-mesenchymal transition in cancer metastasisNoncoding RNA in age-related cardiovascular diseasesG-quadruplexes: Emerging roles in neurodegenerative diseases and the non-coding transcriptomeOral administration of erythromycin decreases RNA toxicity in myotonic dystrophyCell membrane integrity in myotonic dystrophy type 1: implications for therapyApproaches to Validate and Manipulate RNA Targets with Small Molecules in CellsTWEAK/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 miceSingle-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 researchLomofungin 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 MiceIntracellular 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
P2860
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P2860
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
description
2012 nî lūn-bûn
@nan
2012 թուականի Օգոստոսին հրատարակուած գիտական յօդուած
@hyw
2012 թվականի օգոստոսին հրատարակված գիտական հոդված
@hy
2012年の論文
@ja
2012年論文
@yue
2012年論文
@zh-hant
2012年論文
@zh-hk
2012年論文
@zh-mo
2012年論文
@zh-tw
2012年论文
@wuu
name
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@ast
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@en
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@nl
type
label
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@ast
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@en
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@nl
prefLabel
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@ast
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@en
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@nl
P2093
P2860
P356
P1433
P1476
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
@en
P2093
A Robert MacLeod
Andrew J Leger
Bruce M Wentworth
C Frank Bennett
Charles A Thornton
Sanjay K Pandey
Seng H Cheng
Thurman M Wheeler
P2860
P2888
P304
P356
10.1038/NATURE11362
P407
P50
P577
2012-08-01T00:00:00Z
P5875
P6179
1046613614