Published in J Anat on July 22, 2013
Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models. Hum Mol Genet (2014) 0.94
Disease mechanisms and therapeutic approaches in spinal muscular atrophy. J Neurosci (2015) 0.92
A large animal model of spinal muscular atrophy and correction of phenotype. Ann Neurol (2015) 0.90
Advances in therapeutic development for spinal muscular atrophy. Future Med Chem (2014) 0.89
SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA. Hum Mol Genet (2015) 0.89
Spinal muscular atrophy: journeying from bench to bedside. Neurotherapeutics (2014) 0.87
Repeated low doses of morpholino antisense oligomer: an intermediate mouse model of spinal muscular atrophy to explore the window of therapeutic response. Hum Mol Genet (2015) 0.86
Mechanistic principles of antisense targets for the treatment of spinal muscular atrophy. Future Med Chem (2015) 0.84
Spinal muscular atrophy: from tissue specificity to therapeutic strategies. F1000Prime Rep (2015) 0.82
SMA-causing missense mutations in survival motor neuron (Smn) display a wide range of phenotypes when modeled in Drosophila. PLoS Genet (2014) 0.82
Chatting with the neighbors: crosstalk between Rho-kinase (ROCK) and other signaling pathways for treatment of neurological disorders. Front Neurosci (2015) 0.81
Emerging therapies and challenges in Spinal Muscular Atrophy. Ann Neurol (2016) 0.80
Histopathological Defects in Intestine in Severe Spinal Muscular Atrophy Mice Are Improved by Systemic Antisense Oligonucleotide Treatment. PLoS One (2016) 0.80
The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophy. Neurobiol Dis (2015) 0.80
Neurocalcin Delta Suppression Protects against Spinal Muscular Atrophy in Humans and across Species by Restoring Impaired Endocytosis. Am J Hum Genet (2017) 0.79
Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy. Mol Ther Nucleic Acids (2016) 0.79
Longitudinal characterization of biomarkers for spinal muscular atrophy. Ann Clin Transl Neurol (2017) 0.78
Is spinal muscular atrophy a disease of the motor neurons only: pathogenesis and therapeutic implications? Cell Mol Life Sci (2015) 0.78
Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy. Hum Mol Genet (2016) 0.78
Survival Motor Neuron (SMN) protein is required for normal mouse liver development. Sci Rep (2016) 0.77
Restoration of SMN in Schwann cells reverses myelination defects and improves neuromuscular function in spinal muscular atrophy. Hum Mol Genet (2016) 0.77
Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides. PLoS One (2016) 0.77
Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy. Nucleic Acid Ther (2017) 0.77
SECIS-binding protein 2 interacts with the SMN complex and the methylosome for selenoprotein mRNP assembly and translation. Nucleic Acids Res (2017) 0.75
Identification of processing elements and interactors implicate SMN, coilin and the pseudogene-encoded coilp1 in telomerase and box C/D scaRNP biogenesis. RNA Biol (2016) 0.75
Cardiac pathology in spinal muscular atrophy: a systematic review. Orphanet J Rare Dis (2017) 0.75
Neuroplasticity and Repair in Rodent Neurotoxic Models of Spinal Motoneuron Disease. Neural Plast (2016) 0.75
Placental development in a mouse model of spinal muscular atrophy. Biochem Biophys Res Commun (2015) 0.75
An Integrative Transcriptomic Analysis for Identifying Novel Target Genes Corresponding to Severity Spectrum in Spinal Muscular Atrophy. PLoS One (2016) 0.75
Immune dysregulation may contribute to disease pathogenesis in spinal muscular atrophy mice. Hum Mol Genet (2017) 0.75
Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders. Gene Ther (2017) 0.75
Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy. JCI Insight (2017) 0.75
A(a)LS: Ammonia-induced amyotrophic lateral sclerosis. F1000Res (2015) 0.75
Splice-Switching Therapy for Spinal Muscular Atrophy. Genes (Basel) (2017) 0.75
Atrial Septal Defect, Neuromuscular Junction and Skeletal Abnormalities in Spinal Muscular Atrophy Type III. Chin Med J (Engl) (2017) 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
The SMN-SIP1 complex has an essential role in spliceosomal snRNP biogenesis. Cell (1997) 5.57
The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy. Hum Mol Genet (2000) 5.38
The spinal muscular atrophy disease gene product, SMN, and its associated protein SIP1 are in a complex with spliceosomal snRNP proteins. Cell (1997) 5.28
SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN. Hum Mol Genet (2005) 5.23
A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol Genet (1999) 5.09
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol (2010) 4.62
Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy. Am J Hum Genet (2001) 4.50
Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model. Nature (2011) 4.04
SMN oligomerization defect correlates with spinal muscular atrophy severity. Nat Genet (1998) 3.51
Genetic mapping of chronic childhood-onset spinal muscular atrophy to chromosome 5q11.2-13.3. Nature (1990) 3.48
An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN. Hum Mol Genet (2000) 3.41
International SMA consortium meeting. (26-28 June 1992, Bonn, Germany). Neuromuscul Disord (1992) 3.26
Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy. J Clin Invest (2007) 3.11
Selective vulnerability of motor neurons and dissociation of pre- and post-synaptic pathology at the neuromuscular junction in mouse models of spinal muscular atrophy. Hum Mol Genet (2007) 3.09
Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev (2010) 3.02
Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy. Hum Mol Genet (2008) 2.82
Impaired synaptic vesicle release and immaturity of neuromuscular junctions in spinal muscular atrophy mice. J Neurosci (2009) 2.71
CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy. J Clin Invest (2010) 2.62
Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Sci Transl Med (2011) 2.56
Energy metabolism in heart failure and remodelling. Cardiovasc Res (2008) 2.37
Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect. Hum Mol Genet (2008) 2.35
Active transport of the survival motor neuron protein and the role of exon-7 in cytoplasmic localization. J Neurosci (2003) 2.28
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice. Hum Mol Genet (2010) 2.26
Molecular analysis of spinal muscular atrophy and modification of the phenotype by SMN2. Genet Med (2002) 2.26
Reduced U snRNP assembly causes motor axon degeneration in an animal model for spinal muscular atrophy. Genes Dev (2005) 2.22
Early functional impairment of sensory-motor connectivity in a mouse model of spinal muscular atrophy. Neuron (2011) 2.19
A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse. Hum Mol Genet (2011) 2.13
Neurofilament accumulation at the motor endplate and lack of axonal sprouting in a spinal muscular atrophy mouse model. Hum Mol Genet (2002) 2.13
Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy. Sci Transl Med (2010) 2.02
An SMN-dependent U12 splicing event essential for motor circuit function. Cell (2012) 1.94
Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery. Hum Mol Genet (2010) 1.89
Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet (2011) 1.88
The changing natural history of spinal muscular atrophy type 1. Neurology (2007) 1.87
Neuromuscular defects in a Drosophila survival motor neuron gene mutant. Hum Mol Genet (2003) 1.85
SMN gene duplication and the emergence of the SMN2 gene occurred in distinct hominids: SMN2 is unique to Homo sapiens. Hum Genet (2001) 1.81
SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy. Hum Mol Genet (2010) 1.80
Embryonic motor axon development in the severe SMA mouse. Hum Mol Genet (2008) 1.79
Cardiac defects contribute to the pathology of spinal muscular atrophy models. Hum Mol Genet (2010) 1.76
Arrhythmia and cardiac defects are a feature of spinal muscular atrophy model mice. Hum Mol Genet (2010) 1.75
Spinal muscular atrophy: mechanisms and therapeutic strategies. Hum Mol Genet (2010) 1.73
Neural crest stem and progenitor cells. Annu Rev Cell Dev Biol (2006) 1.71
SMN is required for sensory-motor circuit function in Drosophila. Cell (2012) 1.68
Deletion of murine SMN exon 7 directed to skeletal muscle leads to severe muscular dystrophy. J Cell Biol (2001) 1.64
Synaptic defects in the spinal and neuromuscular circuitry in a mouse model of spinal muscular atrophy. PLoS One (2010) 1.63
Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy. J Clin Invest (2011) 1.62
Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy. Hum Mol Genet (2011) 1.61
Defective Ca2+ channel clustering in axon terminals disturbs excitability in motoneurons in spinal muscular atrophy. J Cell Biol (2007) 1.57
Congenital heart disease is a feature of severe infantile spinal muscular atrophy. J Med Genet (2008) 1.57
Intelligence and cognitive function in children and adolescents with spinal muscular atrophy. Neuromuscul Disord (2002) 1.53
Reduced survival motor neuron (Smn) gene dose in mice leads to motor neuron degeneration: an animal model for spinal muscular atrophy type III. Hum Mol Genet (2000) 1.50
Reduced survival of motor neuron (SMN) protein in motor neuronal progenitors functions cell autonomously to cause spinal muscular atrophy in model mice expressing the human centromeric (SMN2) gene. J Neurosci (2010) 1.49
A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy. RNA Biol (2009) 1.46
Vascular perfusion abnormalities in infants with spinal muscular atrophy. J Pediatr (2009) 1.45
Survival motor neuron protein in motor neurons determines synaptic integrity in spinal muscular atrophy. J Neurosci (2012) 1.44
Temporal requirement for high SMN expression in SMA mice. Hum Mol Genet (2011) 1.44
Survival motor neuron (SMN) protein: role in neurite outgrowth and neuromuscular maturation during neuronal differentiation and development. Hum Mol Genet (2002) 1.42
Motor neuron rescue in spinal muscular atrophy mice demonstrates that sensory-motor defects are a consequence, not a cause, of motor neuron dysfunction. J Neurosci (2012) 1.42
Sonographic and maternal serum screening abnormalities in fetuses affected by spinal muscular atrophy. Prenat Diagn (1997) 1.39
Altered intracellular Ca2+ homeostasis in nerve terminals of severe spinal muscular atrophy mice. J Neurosci (2010) 1.38
Subcellular localization and axonal transport of the survival motor neuron (SMN) protein in the developing rat spinal cord. Hum Mol Genet (2000) 1.35
Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy. Hum Mol Genet (2008) 1.32
Reversible molecular pathology of skeletal muscle in spinal muscular atrophy. Hum Mol Genet (2011) 1.32
SMN deficiency disrupts brain development in a mouse model of severe spinal muscular atrophy. Hum Mol Genet (2010) 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
Review: neuromuscular synaptic vulnerability in motor neurone disease: amyotrophic lateral sclerosis and spinal muscular atrophy. Neuropathol Appl Neurobiol (2010) 1.28
Rho-kinase inactivation prolongs survival of an intermediate SMA mouse model. Hum Mol Genet (2010) 1.27
Muscles in a mouse model of spinal muscular atrophy show profound defects in neuromuscular development even in the absence of failure in neuromuscular transmission or loss of motor neurons. Dev Biol (2011) 1.26
Pre-symptomatic development of lower motor neuron connectivity in a mouse model of severe spinal muscular atrophy. Hum Mol Genet (2009) 1.26
Increased IGF-1 in muscle modulates the phenotype of severe SMA mice. Hum Mol Genet (2011) 1.24
Deletion of murine Smn exon 7 directed to liver leads to severe defect of liver development associated with iron overload. Am J Pathol (2004) 1.24
A critical smn threshold in mice dictates onset of an intermediate spinal muscular atrophy phenotype associated with a distinct neuromuscular junction pathology. Neuromuscul Disord (2011) 1.20
Spinal muscular atrophy: from gene to therapy. Semin Pediatr Neurol (2006) 1.19
Glucose metabolism and pancreatic defects in spinal muscular atrophy. Ann Neurol (2012) 1.17
Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. Mol Ther (2013) 1.16
Long-term observations of patients with infantile spinal muscular atrophy with respiratory distress type 1 (SMARD1). Neuropediatrics (2004) 1.15
Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy. Biochem Biophys Res Commun (2009) 1.14
Treatment and complications in flaccid neuromuscular scoliosis (Duchenne muscular dystrophy and spinal muscular atrophy) with posterior-only pedicle screw instrumentation. Eur Spine J (2009) 1.13
Medical considerations of long-term survival of Werdnig-Hoffmann disease. Am J Phys Med Rehabil (2007) 1.13
Synaptic defects in type I spinal muscular atrophy in human development. J Pathol (2013) 1.13
Distinct and overlapping alterations in motor and sensory neurons in a mouse model of spinal muscular atrophy. Hum Mol Genet (2006) 1.09
Digital necroses and vascular thrombosis in severe spinal muscular atrophy. Muscle Nerve (2010) 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
Classical infantile spinal muscular atrophy with SMN deficiency causes sensory neuronopathy. Neurology (2003) 1.07
SMN gene deletion in variant of infantile spinal muscular atrophy. Lancet (1995) 1.05
Fasudil improves survival and promotes skeletal muscle development in a mouse model of spinal muscular atrophy. BMC Med (2012) 1.05
Subcellular distribution of survival motor neuron (SMN) protein: possible involvement in nucleocytoplasmic and dendritic transport. Eur J Neurosci (1999) 1.04
Reduced expression of nicotinic AChRs in myotubes from spinal muscular atrophy I patients. Lab Invest (2004) 1.02
Autonomic dysfunction in cases of spinal muscular atrophy type 1 with long survival. Brain Dev (2005) 1.02
Abnormal fatty acid metabolism in childhood spinal muscular atrophy. Ann Neurol (1999) 1.01
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
Defects in neuromuscular junction remodelling in the Smn(2B/-) mouse model of spinal muscular atrophy. Neurobiol Dis (2012) 1.01
A cell-autonomous defect in skeletal muscle satellite cells expressing low levels of survival of motor neuron protein. Dev Biol (2012) 1.00
Spinal muscular atrophy--clinical and genetic correlations. Neuromuscul Disord (1997) 1.00
Density, calibre and ramification of muscle capillaries are altered in a mouse model of severe spinal muscular atrophy. Neuromuscul Disord (2011) 0.98
SMN requirement for synaptic vesicle, active zone and microtubule postnatal organization in motor nerve terminals. PLoS One (2011) 0.97
Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585. Eur J Hum Genet (2012) 0.96
Temporal requirement for SMN in motoneuron development. Hum Mol Genet (2013) 0.96
The distribution of SMN protein complex in human fetal tissues and its alteration in spinal muscular atrophy. Hum Mol Genet (1998) 0.95
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
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
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
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
Replacement of huntingtin exon 1 by trans-splicing. Cell Mol Life Sci (2012) 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
Analysis of a read-through promoting compound in a severe mouse model of spinal muscular atrophy. Neurosci Lett (2012) 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
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