PubRank

Christian L Lorson

Author PubWeight™ 66.01‹?›

Top papers

Rank Title Journal Year PubWeight™‹?›
1 Induced pluripotent stem cells from a spinal muscular atrophy patient. Nature 2008 12.21
2 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
3 Cardiac defects contribute to the pathology of spinal muscular atrophy models. Hum Mol Genet 2010 1.76
4 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
5 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
6 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
7 Development of a single vector system that enhances trans-splicing of SMN2 transcripts. PLoS One 2008 1.43
8 Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing. Mol Ther 2007 1.39
9 Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. Mol Ther 2006 1.37
10 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
11 Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy. Hum Mol Genet 2008 1.32
12 Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy. J Neurosci 2010 1.32
13 Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts. Hum Genet 2006 1.31
14 Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease? J Anat 2013 1.23
15 A direct interaction between the survival motor neuron protein and p53 and its relationship to spinal muscular atrophy. J Biol Chem 2001 1.20
16 Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model. Hum Mol Genet 2009 1.19
17 Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy. Biochem Biophys Res Commun 2009 1.14
18 A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo. Hum Gene Ther 2008 1.13
19 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
20 The COPI vesicle complex binds and moves with survival motor neuron within axons. Hum Mol Genet 2011 1.08
21 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
22 Induction of full-length survival motor neuron by polyphenol botanical compounds. Hum Genet 2007 1.06
23 Delivery of therapeutic agents through intracerebroventricular (ICV) and intravenous (IV) injection in mice. J Vis Exp 2011 1.03
24 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
25 A SMNDelta7 read-through product confers functionality to the SMNDelta7 protein. Neurosci Lett 2008 0.97
26 RNA-targeting approaches for neuromuscular diseases. Trends Mol Med 2009 0.97
27 SMN in spinal muscular atrophy and snRNP biogenesis. Wiley Interdiscip Rev RNA 2011 0.93
28 Decreasing disease severity in symptomatic, Smn(-/-);SMN2(+/+), spinal muscular atrophy mice following scAAV9-SMN delivery. Hum Gene Ther 2012 0.92
29 The Ewing's sarcoma protein interacts with the Tudor domain of the survival motor neuron protein. Brain Res Mol Brain Res 2003 0.92
30 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
31 Partial restoration of cardio-vascular defects in a rescued severe model of spinal muscular atrophy. J Mol Cell Cardiol 2012 0.91
32 SMN-inducing compounds for the treatment of spinal muscular atrophy. Future Med Chem 2012 0.90
33 Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA. Transgenic Res 2011 0.87
34 Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy. Hum Mol Genet 2013 0.87
35 Transgenic inactivation of murine myostatin does not decrease the severity of disease in a model of Spinal Muscular Atrophy. Neuromuscul Disord 2011 0.87
36 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
37 Minute virus of mice small nonstructural protein NS2 interacts and colocalizes with the Smn protein. J Virol 2002 0.86
38 Therapeutics that directly increase SMN expression to treat spinal muscular atrophy. Drug News Perspect 2010 0.86
39 The spinal muscular atrophy mouse model, SMAΔ7, displays altered axonal transport without global neurofilament alterations. Acta Neuropathol 2011 0.86
40 Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA. BMC Neurosci 2009 0.85
41 Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds. EMBO Mol Med 2013 0.84
42 Identification of a tripartite import signal in the Ewing Sarcoma protein (EWS). Biochem Biophys Res Commun 2009 0.83
43 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
44 Survival motor neuron protein in the nucleolus of mammalian neurons. Brain Res 2002 0.81
45 Optimization of SMN trans-splicing through the analysis of SMN introns. J Mol Neurosci 2011 0.81
46 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
47 A survival motor neuron:tetanus toxin fragment C fusion protein for the targeted delivery of SMN protein to neurons. Brain Res 2004 0.80
48 Replacement of huntingtin exon 1 by trans-splicing. Cell Mol Life Sci 2012 0.80
49 Identification and characterization of the porcine (Sus scrofa) survival motor neuron (SMN1) gene: an animal model for therapeutic studies. Dev Dyn 2008 0.79
50 Analysis of a read-through promoting compound in a severe mouse model of spinal muscular atrophy. Neurosci Lett 2012 0.79
51 Monitoring of recombinant survival motor neuron protein using fiber-optic surface plasmon resonance. Analyst 2004 0.78
52 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
53 Identification and characterisation of a nuclear localisation signal in the SMN associated protein, Gemin4. Biochem Biophys Res Commun 2008 0.75