Published in Drug News Perspect on October 01, 2010
Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease? J Anat (2013) 1.23
Fasudil improves survival and promotes skeletal muscle development in a mouse model of spinal muscular atrophy. BMC Med (2012) 1.05
Alternative splicing in spinal muscular atrophy underscores the role of an intron definition model. RNA Biol (2011) 0.93
Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery. Hum Gene Ther (2012) 0.92
A multi-exon-skipping detection assay reveals surprising diversity of splice isoforms of spinal muscular atrophy genes. PLoS One (2012) 0.92
New therapeutic approaches to spinal muscular atrophy. Curr Neurol Neurosci Rep (2012) 0.91
Coilin phosphomutants disrupt Cajal body formation, reduce cell proliferation and produce a distinct coilin degradation product. PLoS One (2011) 0.90
AMPA GluA1-flip targeted oligonucleotide therapy reduces neonatal seizures and hyperexcitability. PLoS One (2017) 0.75
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
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
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
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
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
The COPI vesicle complex binds and moves with survival motor neuron within axons. Hum Mol Genet (2011) 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
Induction of full-length survival motor neuron by polyphenol botanical compounds. Hum Genet (2007) 1.06
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
Stimulation of GABA-induced Ca2+ influx enhances maturation of human induced pluripotent stem cell-derived neurons. PLoS One (2013) 0.90
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
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
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
Identification of a tripartite import signal in the Ewing Sarcoma protein (EWS). Biochem Biophys Res Commun (2009) 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
A survival motor neuron:tetanus toxin fragment C fusion protein for the targeted delivery of SMN protein to neurons. Brain Res (2004) 0.80
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
In Vivo Tracking of Human Neural Progenitor Cells in the Rat Brain Using Magnetic Resonance Imaging Is Not Enhanced by Ferritin Expression. Cell Transplant (2015) 0.79
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