Diverse small-molecule modulators of SMN expression found by high-throughput compound screening: early leads towards a therapeutic for spinal muscular atrophy.
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Spinal muscular atrophyDcpS as a therapeutic target for spinal muscular atrophy.SMN transcript levels in leukocytes of SMA patients determined by absolute real-time PCRMolecular Mechanisms of Neurodegeneration in Spinal Muscular AtrophySpinal muscular atrophy: from gene discovery to clinical trialsTrichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy.Utility of survival motor neuron ELISA for spinal muscular atrophy clinical and preclinical analysesEffects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophyPostsymptomatic 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.A novel cell immunoassay to measure survival of motor neurons protein in blood cellsHigh throughput screening for neurodegeneration and complex disease phenotypes.The effect of diet on the protective action of D156844 observed in spinal muscular atrophy mice.Spinal muscular atrophy: mechanisms and therapeutic strategies.A screen for regulators of survival of motor neuron protein levelsSplicing 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.Drug discovery and development for spinal muscular atrophy: lessons from screening approaches and future challenges for clinical development.Therapeutic strategies for the treatment of spinal muscular atrophy.SMN-inducing compounds for the treatment of spinal muscular atrophy.Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 and SMN2.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 compoundsA cell system for phenotypic screening of modifiers of SMN2 gene expression and function.Advances in therapeutic development for spinal muscular atrophy.Discovery, synthesis, and biological evaluation of novel SMN protein modulators.Disease mechanisms and therapeutic approaches in spinal muscular atrophy.DcpS is a transcript-specific modulator of RNA in mammalian cells.The effect of the DcpS inhibitor D156844 on the protective action of follistatin in mice with spinal muscular atrophy.Small Molecules in Development for the Treatment of Spinal Muscular Atrophy.Therapeutics development for spinal muscular atrophy.Clinical and molecular cross-sectional study of a cohort of adult type III spinal muscular atrophy patients: clues from a biomarker studyPerspectives on clinical trials in spinal muscular atrophyThe E3 ubiquitin ligase mind bomb 1 ubiquitinates and promotes the degradation of survival of motor neuron protein.Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy.Neurobiological applications of small molecule screening.
P2860
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P2860
Diverse small-molecule modulators of SMN expression found by high-throughput compound screening: early leads towards a therapeutic for spinal muscular atrophy.
description
2005 nî lūn-bûn
@nan
2005年の論文
@ja
2005年学术文章
@wuu
2005年学术文章
@zh-cn
2005年学术文章
@zh-hans
2005年学术文章
@zh-my
2005年学术文章
@zh-sg
2005年學術文章
@yue
2005年學術文章
@zh
2005年學術文章
@zh-hant
name
Diverse small-molecule modulat ...... c for spinal muscular atrophy.
@en
type
label
Diverse small-molecule modulat ...... c for spinal muscular atrophy.
@en
prefLabel
Diverse small-molecule modulat ...... c for spinal muscular atrophy.
@en
P2093
P356
P1476
Diverse small-molecule modulat ...... ic for spinal muscular atrophy
@en
P2093
Alexandra Bernardino
Brian A Pollok
Daniel D Coovert
Jeffrey Stack
Jill Jarecki
Michael Whitney
Xiaocun Chen
P304
P356
10.1093/HMG/DDI205
P50
P577
2005-06-08T00:00:00Z