Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.
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Development of a single vector system that enhances trans-splicing of SMN2 transcriptsModeling spinal muscular atrophy in DrosophilaDrug treatment for spinal muscular atrophy types II and IIIDrug treatment for spinal muscular atrophy types II and IIIThe splicing regulator Sam68 binds to a novel exonic splicing silencer and functions in SMN2 alternative splicing in spinal muscular atrophyCholinergic-associated loss of hnRNP-A/B in Alzheimer's disease impairs cortical splicing and cognitive function in miceDifferential 3' splice site recognition of SMN1 and SMN2 transcripts by U2AF and U2 snRNPSpinal muscular atrophy: from gene discovery to clinical trialsSynthetic biology and therapeutic strategies for the degenerating brain: Synthetic biology approaches can transform classical cell and gene therapies, to provide new cures for neurodegenerative diseasesOligonucleotide Therapies: The Past and the PresentAntisense oligonucleotides: treating neurodegeneration at the level of RNAMolecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Splicing therapy for neuromuscular diseaseA splice site mutation in laminin-α2 results in a severe muscular dystrophy and growth abnormalities in zebrafishProgress toward therapy with antisense-mediated splicing modulationSpinal Muscular AtrophyApplications of Cas9 as an RNA-programmed RNA-binding proteinA short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophyRescue of a severe mouse model for spinal muscular atrophy by U7 snRNA-mediated splicing modulationEvaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical studySynthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicingSMN in spinal muscular atrophy and snRNP biogenesisCNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.Sodium vanadate combined with L-ascorbic acid delays disease progression, enhances motor performance, and ameliorates muscle atrophy and weakness in mice with spinal muscular atrophy.Pathological impact of SMN2 mis-splicing in adult SMA miceSomatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 SplicingA method of predicting changes in human gene splicing induced by genetic variants in context of cis-acting elementsEfficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide TreatmentAntisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe DiseaseTrans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.The alternative splicing repressors hnRNP A1/A2 and PTB influence pyruvate kinase isoform expression and cell metabolism.The notorious R.N.A. in the spotlight - drug or target for the treatment of diseasePharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblastsSpinal muscular atrophy: new and emerging insights from model miceAn antisense microwalk reveals critical role of an intronic position linked to a unique long-distance interaction in pre-mRNA splicingSpinal muscular atrophy: mechanisms and therapeutic strategies.Intron 7 conserved sequence elements regulate the splicing of the SMN genes.Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse modelSplicing of the Survival Motor Neuron genes and implications for treatment of SMA
P2860
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P2860
Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.
description
2008 nî lūn-bûn
@nan
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
2008年论文
@zh
2008年论文
@zh-cn
name
Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.
@ast
Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.
@en
type
label
Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.
@ast
Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.
@en
prefLabel
Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.
@ast
Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.
@en
P2093
P2860
P1476
Antisense masking of an hnRNP ...... 2 splicing in transgenic mice.
@en
P2093
C Frank Bennett
Hazeem L Okunola
Timothy A Vickers
P2860
P304
P356
10.1016/J.AJHG.2008.01.014
P407
P577
2008-03-27T00:00:00Z