An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA
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Indoprofen upregulates the survival motor neuron protein through a cyclooxygenase-independent mechanismAlternative splicing for diseases, cancers, drugs, and databasesHigh expression level of Tra2-β1 is responsible for increased SMN2 exon 7 inclusion in the testis of SMA miceDoxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettesIdentification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screeningEffects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophyCNS-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.Somatic Therapy of a Mouse SMA Model with a U7 snRNA Gene Correcting SMN2 SplicingA high-throughput screening strategy identifies cardiotonic steroids as alternative splicing modulatorsAlternative splicing of exon 10 in the tau gene as a target for treatment of tauopathies.Selective modification of alternative splicing by indole derivatives that target serine-arginine-rich protein splicing factors.Splicing of the Survival Motor Neuron genes and implications for treatment of SMAAssays for the identification and prioritization of drug candidates for spinal muscular atrophyIdentification of novel compounds that increase SMN protein levels using an improved SMN2 reporter cell assay.A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo.Restoration of the cystic fibrosis transmembrane conductance regulator function by splicing modulation.Therapeutic strategies for the treatment of spinal muscular atrophy.Inhibition of apoptosis blocks human motor neuron cell death in a stem cell model of spinal muscular atrophy.In vivo selection reveals combinatorial controls that define a critical exon in the spinal muscular atrophy genes.SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compoundsDiscovery, synthesis, and biological evaluation of novel SMN protein modulators.Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy.Identification of a novel cyclic AMP-response element (CRE-II) and the role of CREB-1 in the cAMP-induced expression of the survival motor neuron (SMN) gene.Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA.P-coumaric acid regulates exon 12 splicing of the ATP7B gene by modulating hnRNP A1 protein expressions.Splicing inhibition of U2AF65 leads to alternative exon skipping.Therapy development for spinal muscular atrophy in SMN independent targets.Altered mRNA Splicing in SMN-Depleted Motor Neuron-Like Cells.A transgene carrying an A2G missense mutation in the SMN gene modulates phenotypic severity in mice with severe (type I) spinal muscular atrophy.Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy.Spinal muscular atrophy: an update on therapeutic progressTargeting RNA-splicing for SMA treatment.Progress in therapeutic antisense applications for neuromuscular disorders.Spinal muscular atrophy disease: a literature review for therapeutic strategies.Developments in the discovery of drugs for spinal muscular atrophy: successful beginnings and future prospects.Alternative splicing of in-frame exon associated with premature termination codons: implications for readthrough therapies.A double-reporter splicing assay for determining splicing efficiency in mammalian cells.
P2860
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P2860
An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA
description
2001 nî lūn-bûn
@nan
2001 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2001 թվականի հոտեմբերին հրատարակված գիտական հոդված
@hy
2001年の論文
@ja
2001年論文
@yue
2001年論文
@zh-hant
2001年論文
@zh-hk
2001年論文
@zh-mo
2001年論文
@zh-tw
2001年论文
@wuu
name
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@ast
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@en
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@nl
type
label
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@ast
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@en
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@nl
prefLabel
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@ast
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@en
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@nl
P2093
P2860
P3181
P356
P1433
P1476
An in vivo reporter system for ...... mRNA: potential therapy of SMA
@en
P2093
P2860
P2888
P304
P3181
P356
10.1038/SJ.GT.3301550
P407
P577
2001-10-01T00:00:00Z