Published in Hum Genet on October 26, 2007
CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy. J Clin Invest (2010) 2.62
Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy. Hum Mol Genet (2009) 1.53
Effects of 2,4-diaminoquinazoline derivatives on SMN expression and phenotype in a mouse model for spinal muscular atrophy. Hum Mol Genet (2009) 1.46
Inhibition of apoptosis blocks human motor neuron cell death in a stem cell model of spinal muscular atrophy. PLoS One (2012) 1.40
Epigallocatechin-3-gallate inhibits IL-6 synthesis and suppresses transsignaling by enhancing soluble gp130 production. Proc Natl Acad Sci U S A (2008) 1.15
A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo. Hum Gene Ther (2008) 1.13
Green tea polyphenol epigallocatechin 3-gallate in arthritis: progress and promise. Arthritis Res Ther (2010) 1.07
Splicing regulation of the survival motor neuron genes and implications for treatment of spinal muscular atrophy. Front Biosci (Landmark Ed) (2010) 0.96
Spinal muscular atrophy disease: a literature review for therapeutic strategies. J Med Life (2010) 0.92
Splicing therapeutics in SMN2 and APOB. Curr Opin Mol Ther (2009) 0.89
Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy. J Neurosci Methods (2008) 0.87
Activation of the stress proteome as a mechanism for small molecule therapeutics. Hum Mol Genet (2012) 0.85
Carboxylic acid derivatives of histone deacetylase inhibitors induce full length SMN2 transcripts: a promising target for spinal muscular atrophy therapeutics. Arch Med Sci (2011) 0.84
Histone deacetylase inhibitors as potential treatment for spinal muscular atrophy. Genet Mol Biol (2013) 0.84
Developing therapies for spinal muscular atrophy. Ann N Y Acad Sci (2015) 0.80
Resveratrol, by modulating RNA processing factor levels, can influence the alternative splicing of pre-mRNAs. PLoS One (2011) 0.79
Protein kinase C δ (PKCδ) splice variants modulate apoptosis pathway in 3T3L1 cells during adipogenesis: identification of PKCδII inhibitor. J Biol Chem (2013) 0.79
Investigations of curcumin and resveratrol on neurite outgrowth: perspectives on spinal muscular atrophy. Biomed Res Int (2014) 0.75
Multiple effects of curcumin on promoting expression of the exon 7-containing SMN2 transcript. Genes Nutr (2015) 0.75
Identification and characterization of a spinal muscular atrophy-determining gene. Cell (1995) 17.41
A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci U S A (1999) 7.60
A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol Genet (1999) 5.09
The survival motor neuron protein in spinal muscular atrophy. Hum Mol Genet (1997) 4.86
A novel nuclear structure containing the survival of motor neurons protein. EMBO J (1996) 4.72
Dietary intake of antioxidants and risk of Alzheimer disease. JAMA (2002) 4.01
A negative element in SMN2 exon 7 inhibits splicing in spinal muscular atrophy. Nat Genet (2003) 3.84
Green tea composition, consumption, and polyphenol chemistry. Prev Med (1992) 3.46
Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy. J Clin Invest (2007) 3.11
Assembly of specific SR protein complexes on distinct regulatory elements of the Drosophila doublesex splicing enhancer. Genes Dev (1996) 2.90
Treatment of spinal muscular atrophy by sodium butyrate. Proc Natl Acad Sci U S A (2001) 2.56
Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy. Hum Mol Genet (2003) 2.51
Valproic acid increases SMN levels in spinal muscular atrophy patient cells. Ann Neurol (2003) 2.28
Apolipoprotein E polymorphism and Alzheimer disease: The Indo-US Cross-National Dementia Study. Arch Neurol (2000) 2.04
Resveratrol protects against global cerebral ischemic injury in gerbils. Brain Res (2002) 1.95
The relationship between SMN, the spinal muscular atrophy protein, and nuclear coiled bodies in differentiated tissues and cultured cells. Exp Cell Res (2000) 1.89
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy. Eur J Hum Genet (2004) 1.89
Emerging role of polyphenolic compounds in the treatment of neurodegenerative diseases: a review of their intracellular targets. Eur J Pharmacol (2006) 1.85
Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients. Hum Mol Genet (2001) 1.80
Wine consumption and dementia in the elderly: a prospective community study in the Bordeaux area. Rev Neurol (Paris) (1997) 1.77
A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels. Hum Mol Genet (2005) 1.72
The role of histone acetylation in SMN gene expression. Hum Mol Genet (2005) 1.56
Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3' splice site pairing. J Biol Chem (2001) 1.56
In vitro and ex vivo evaluation of second-generation histone deacetylase inhibitors for the treatment of spinal muscular atrophy. J Neurochem (2006) 1.48
Indoprofen upregulates the survival motor neuron protein through a cyclooxygenase-independent mechanism. Chem Biol (2004) 1.45
Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients. Eur J Hum Genet (2005) 1.38
Neuroprotective mechanisms of curcumin against cerebral ischemia-induced neuronal apoptosis and behavioral deficits. J Neurosci Res (2005) 1.38
An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA. Gene Ther (2001) 1.37
Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. Mol Ther (2006) 1.37
Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts. Hum Genet (2006) 1.31
Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells. Ann Neurol (2005) 1.30
Expression of the SMN gene, the spinal muscular atrophy determining gene, in the mammalian central nervous system. Hum Mol Genet (1997) 1.28
EGCG corrects aberrant splicing of IKAP mRNA in cells from patients with familial dysautonomia. Biochem Biophys Res Commun (2003) 1.28
The benzamide M344, a novel histone deacetylase inhibitor, significantly increases SMN2 RNA/protein levels in spinal muscular atrophy cells. Hum Genet (2006) 1.27
The role of the SMN gene in proximal spinal muscular atrophy. Hum Mol Genet (1998) 1.24
Interaction of dietary antioxidants in vivo: how fruit and vegetables prevent disease? QJM (1999) 1.20
Dietary antioxidants and Parkinson disease. The Rotterdam Study. Arch Neurol (1997) 1.05
Phytoestrogens in common herbs regulate prostate cancer cell growth in vitro. Nutr Cancer (2004) 1.03
Neuroprotective effects of (-)-epigallocatechin-3-gallate in a transgenic mouse model of amyotrophic lateral sclerosis. Neurochem Res (2006) 0.94
Grape Extract, Resveratrol, and Its Analogs: A Review. J Med Food (2001) 0.93
Expression study of survival motor neuron gene in human fetal tissues. Biochem Mol Med (1997) 0.91
Curcumin induces stress response, neurite outgrowth and prevent NF-kappaB activation by inhibiting the proteasome function. Neurotox Res (2006) 0.91
Specific plasma membrane binding sites for polyphenols, including resveratrol, in the rat brain. J Pharmacol Exp Ther (2006) 0.90
Cell-specific survival motor neuron gene expression during human development of the central nervous system: implications for the pathogenesis of spinal muscular atrophy. Am J Pathol (1998) 0.84
Gene-expression profiling during curcumin-induced apoptosis reveals downregulation of CXCR4. Exp Hematol (2007) 0.83
Survival motor neuron (SMN) protein in rat is expressed as different molecular forms and is developmentally regulated. Eur J Neurosci (1998) 0.83
Expression and subcellular localization of two isoforms of the survival motor neuron protein in different cell types. J Neurosci Res (2000) 0.81
Differential subcellular localization of the survival motor neuron protein in spinal cord and skeletal muscle. Biochem Biophys Res Commun (1999) 0.79
Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature (2008) 12.21
Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy. Hum Gene Ther (2010) 1.93
Cardiac defects contribute to the pathology of spinal muscular atrophy models. Hum Mol Genet (2010) 1.76
A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels. Hum Mol Genet (2005) 1.72
Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy. Hum Mol Genet (2009) 1.53
SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2 beta 1. Hum Mol Genet (2002) 1.53
Development of a single vector system that enhances trans-splicing of SMN2 transcripts. PLoS One (2008) 1.43
Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing. Mol Ther (2007) 1.39
Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. Mol Ther (2006) 1.37
Protein phosphatase 1 binds to the RNA recognition motif of several splicing factors and regulates alternative pre-mRNA processing. Hum Mol Genet (2007) 1.33
Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy. Hum Mol Genet (2008) 1.32
Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy. J Neurosci (2010) 1.32
Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts. Hum Genet (2006) 1.31
Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease? J Anat (2013) 1.23
A direct interaction between the survival motor neuron protein and p53 and its relationship to spinal muscular atrophy. J Biol Chem (2001) 1.20
Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model. Hum Mol Genet (2009) 1.19
Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy. Biochem Biophys Res Commun (2009) 1.14
A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo. Hum Gene Ther (2008) 1.13
The COPI vesicle complex binds and moves with survival motor neuron within axons. Hum Mol Genet (2011) 1.08
Minute virus of mice NS1 interacts with the SMN protein, and they colocalize in novel nuclear bodies induced by parvovirus infection. J Virol (2002) 1.08
Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy. Mol Ther (2011) 1.07
Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice. J Vis Exp (2011) 1.03
Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy. Biochem Biophys Res Commun (2011) 1.01
A SMNDelta7 read-through product confers functionality to the SMNDelta7 protein. Neurosci Lett (2008) 0.97
RNA-targeting approaches for neuromuscular diseases. Trends Mol Med (2009) 0.97
SMN in spinal muscular atrophy and snRNP biogenesis. Wiley Interdiscip Rev RNA (2011) 0.93
Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery. Hum Gene Ther (2012) 0.92
The Ewing's sarcoma protein interacts with the Tudor domain of the survival motor neuron protein. Brain Res Mol Brain Res (2003) 0.92
The Wallerian degeneration slow (Wld(s)) gene does not attenuate disease in a mouse model of spinal muscular atrophy. Biochem Biophys Res Commun (2008) 0.92
Partial restoration of cardio-vascular defects in a rescued severe model of spinal muscular atrophy. J Mol Cell Cardiol (2012) 0.91
SMN-inducing compounds for the treatment of spinal muscular atrophy. Future Med Chem (2012) 0.90
Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA. Transgenic Res (2011) 0.87
Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy. Hum Mol Genet (2013) 0.87
Transgenic inactivation of murine myostatin does not decrease the severity of disease in a model of Spinal Muscular Atrophy. Neuromuscul Disord (2011) 0.87
Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy. J Neurosci Methods (2008) 0.87
Minute virus of mice small nonstructural protein NS2 interacts and colocalizes with the Smn protein. J Virol (2002) 0.86
Therapeutics that directly increase SMN expression to treat spinal muscular atrophy. Drug News Perspect (2010) 0.86
The spinal muscular atrophy mouse model, SMAΔ7, displays altered axonal transport without global neurofilament alterations. Acta Neuropathol (2011) 0.86
Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA. BMC Neurosci (2009) 0.85
Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds. EMBO Mol Med (2013) 0.84
Identification of a tripartite import signal in the Ewing Sarcoma protein (EWS). Biochem Biophys Res Commun (2009) 0.83
Minute virus of mice small non-structural protein NS2 localizes within, but is not required for the formation of, Smn-associated autonomous parvovirus-associated replication bodies. J Gen Virol (2005) 0.83
Survival motor neuron protein in the nucleolus of mammalian neurons. Brain Res (2002) 0.81
Optimization of SMN trans-splicing through the analysis of SMN introns. J Mol Neurosci (2011) 0.81
Replacement of huntingtin exon 1 by trans-splicing. Cell Mol Life Sci (2012) 0.80
Identification of a self-association domain in the Ewing's sarcoma protein: a novel function for arginine-glycine-glycine rich motifs? J Biochem (2010) 0.80
A survival motor neuron:tetanus toxin fragment C fusion protein for the targeted delivery of SMN protein to neurons. Brain Res (2004) 0.80
Identification and characterization of the porcine (Sus scrofa) survival motor neuron (SMN1) gene: an animal model for therapeutic studies. Dev Dyn (2008) 0.79
Analysis of a read-through promoting compound in a severe mouse model of spinal muscular atrophy. Neurosci Lett (2012) 0.79
Monitoring of recombinant survival motor neuron protein using fiber-optic surface plasmon resonance. Analyst (2004) 0.78
Restoration of SMN to Emx-1 expressing cortical neurons is not sufficient to provide benefit to a severe mouse model of Spinal Muscular Atrophy. Transgenic Res (2013) 0.76
Identification and characterisation of a nuclear localisation signal in the SMN associated protein, Gemin4. Biochem Biophys Res Commun (2008) 0.75