Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients - Wikidata - awgldk - TriplyDB

Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients

Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients

http://www.wikidata.org/entity/Q28209134

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Tissue-specific reduction in splicing efficiency of IKBKAP due to the major mutation associated with familial dysautonomia.Indoprofen upregulates the survival motor neuron protein through a cyclooxygenase-independent mechanism Molecular Mechanisms of Neurodegeneration in Spinal Muscular Atrophy Spinal muscular atrophy: from gene discovery to clinical trials Alternative splicing for diseases, cancers, drugs, and databases Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy Manipulation of alternative splicing by a newly developed inhibitor of Clks Identification of small molecule and genetic modulators of AON-induced dystrophin exon skipping by high-throughput screening Utility of survival motor neuron ELISA for spinal muscular atrophy clinical and preclinical analyses Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy.Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy.The DcpS inhibitor RG3039 improves survival, function and motor unit pathologies in two SMA mouse models.Genetically modified pig models for neurodegenerative disorders.A novel cell immunoassay to measure survival of motor neurons protein in blood cells A high-throughput screening strategy identifies cardiotonic steroids as alternative splicing modulators Pharmacologic correction of dominant-negative GH1 deficiency causing mutations.Phosphatidylserine increases IKBKAP levels in familial dysautonomia cells Selective modification of alternative splicing by indole derivatives that target serine-arginine-rich protein splicing factors.A screen for regulators of survival of motor neuron protein levels Correction of aberrant FGFR1 alternative RNA splicing through targeting of intronic regulatory elements.Splicing of the Survival Motor Neuron genes and implications for treatment of SMA Assays for the identification and prioritization of drug candidates for spinal muscular atrophy Alternative splicing and retinal degeneration Identification 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.Temporal requirement for high SMN expression in SMA mice Therapeutic strategies for the treatment of spinal muscular atrophy.SMN-inducing compounds for the treatment of spinal muscular atrophy.In vivo selection reveals combinatorial controls that define a critical exon in the spinal muscular atrophy genes.Spinal Muscular Atrophy Therapeutics: Where do we Stand?Tetracyclines that promote SMN2 exon 7 splicing as therapeutics for spinal muscular atrophy.Small molecule screen reveals regulation of survival motor neuron protein abundance by Ras proteins.Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds Repair of pre-mRNA splicing: prospects for a therapy for spinal muscular atrophy.Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts.Natural history of denervation in SMA: relation to age, SMN2 copy number, and function Discovery, synthesis, and biological evaluation of novel SMN protein modulators.Molecular and cellular basis of spinal muscular atrophy.

P2860

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P2860

Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients

http://www.wikidata.org/entity/Q28209134

description

2001 nî lūn-bûn

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2001 թուականի Նոյեմբերին հրատարակուած գիտական յօդուած

@hyw

2001 թվականի նոյեմբերին հրատարակված գիտական հոդված

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2001年の論文

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Aclarubicin treatment restores ...... inal muscular atrophy patients

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Aclarubicin treatment restores ...... inal muscular atrophy patients

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Aclarubicin treatment restores ...... inal muscular atrophy patients

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Aclarubicin treatment restores ...... inal muscular atrophy patients

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Aclarubicin treatment restores ...... inal muscular atrophy patients

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Aclarubicin treatment restores ...... inal muscular atrophy patients

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Aclarubicin treatment restores ...... inal muscular atrophy patients

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Human Molecular Genetics

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Aclarubicin treatment restores ...... inal muscular atrophy patients

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A H Burghes

http://www.w3.org/2001/XMLSchema#string

B Pollok

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C Andreassi

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D D Coovert

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E Androphy

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J Jarecki

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J Zhou

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M Whitney

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M Zhang

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U R Monani

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2841-9

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scholarly article

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5081579

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10.1093/HMG/10.24.2841

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279610267

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11734549

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spinal muscular atrophy