A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy
about
The splicing regulator Sam68 binds to a novel exonic splicing silencer and functions in SMN2 alternative splicing in spinal muscular atrophyThe zinc finger protein ZPR1 is a potential modifier of spinal muscular atrophy.Molecular Mechanisms of Neurodegeneration in Spinal Muscular AtrophyMolecular, genetic and stem cell-mediated therapeutic strategies for spinal muscular atrophy (SMA)Splicing therapy for neuromuscular diseaseOxidative Stress Triggers Body-Wide Skipping of Multiple Exons of the Spinal Muscular Atrophy GeneMolecular basis of purine-rich RNA recognition by the human SR-like protein Tra2-β1Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical studySMN in spinal muscular atrophy and snRNP biogenesisTrans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblastsAn 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.A short antisense oligonucleotide ameliorates symptoms of severe mouse models of spinal muscular atrophySplicing of the Survival Motor Neuron genes and implications for treatment of SMATIA1 prevents skipping of a critical exon associated with spinal muscular atrophy.Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.A humanized Smn gene containing the SMN2 nucleotide alteration in exon 7 mimics SMN2 splicing and the SMA disease phenotype.Temporal requirement for high SMN expression in SMA miceSMN-inducing compounds for the treatment of spinal muscular atrophy.Antisense oligonucleotides for the treatment of spinal muscular atrophy.A feedback loop regulates splicing of the spinal muscular atrophy-modifying gene, SMN2.A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes.Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy.Efficient in vivo manipulation of alternative pre-mRNA splicing events using antisense morpholinos in mice.Regulation of the mutually exclusive exons 8a and 8 in the CaV1.2 calcium channel transcript by polypyrimidine tract-binding protein.Synthesis and characterization of pseudocantharidins, novel phosphatase modulators that promote the inclusion of exon 7 into the SMN (survival of motoneuron) pre-mRNARepair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy.Advances in therapeutic development for spinal muscular atrophy.Alternative splicing in spinal muscular atrophy underscores the role of an intron definition model.Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophyDisruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA.mRNA transcript diversity creates new opportunities for pharmacological intervention.Global identification of hnRNP A1 binding sites for SSO-based splicing modulation.Antisense-based therapy for the treatment of spinal muscular atrophySplicing regulation in spinal muscular atrophy by an RNA structure formed by long-distance interactions.Genetic inhibition of JNK3 ameliorates spinal muscular atrophy.Mechanistic principles of antisense targets for the treatment of spinal muscular atrophyTuning of alternative splicing--switch from proto-oncogene to tumor suppressor
P2860
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P2860
A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy
description
2009 nî lūn-bûn
@nan
2009 թուականին հրատարակուած գիտական յօդուած
@hyw
2009 թվականին հրատարակված գիտական հոդված
@hy
2009年の論文
@ja
2009年論文
@yue
2009年論文
@zh-hant
2009年論文
@zh-hk
2009年論文
@zh-mo
2009年論文
@zh-tw
2009年论文
@wuu
name
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@ast
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@en
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@nl
type
label
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@ast
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@en
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@nl
prefLabel
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@ast
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@en
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@nl
P2093
P2860
P3181
P356
P1433
P1476
A short antisense oligonucleot ...... xon in spinal muscular atrophy
@en
P2093
Laxman Gangwani
Lu Cheng Cao
Maria Shishimorova
Natalia N Singh
Ravindra N Singh
P2860
P304
P3181
P356
10.4161/RNA.6.3.8723
P407
P577
2009-01-01T00:00:00Z