{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,19]],"date-time":"2026-02-19T07:29:00Z","timestamp":1771486140262,"version":"3.50.1"},"reference-count":212,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2017,6,1]],"date-time":"2017-06-01T00:00:00Z","timestamp":1496275200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":["clinicalkey.jp","clinicalkey.com","clinicalkey.es","clinicalkey.com.au","clinicalkey.fr","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Neurobiology of Disease"],"published-print":{"date-parts":[[2017,6]]},"DOI":"10.1016\/j.nbd.2017.01.011","type":"journal-article","created":{"date-parts":[[2017,2,3]],"date-time":"2017-02-03T01:02:00Z","timestamp":1486083720000},"page":"11-20","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":18,"special_numbering":"C","title":["Spinal muscular atrophy: Factors that modulate motor neurone vulnerability"],"prefix":"10.1016","volume":"102","author":[{"given":"Wen-Yo","family":"Tu","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julie E.","family":"Simpson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J. Robin","family":"Highley","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paul R.","family":"Heath","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"issue":"7","key":"10.1016\/j.nbd.2017.01.011_bb0005","doi-asserted-by":"crossref","first-page":"1328","DOI":"10.1093\/hmg\/dds540","article-title":"Plastin 3 ameliorates spinal muscular atrophy via delayed axon pruning and improves neuromuscular junction functionality","volume":"22","author":"Ackermann","year":"2013","journal-title":"Hum. Mol. Genet."},{"issue":"25","key":"10.1016\/j.nbd.2017.01.011_bb0010","doi-asserted-by":"crossref","first-page":"10337","DOI":"10.1073\/pnas.1104928108","article-title":"Interaction of survival of motor neuron (SMN) and HuD proteins with mRNA cpg15 rescues motor neuron axonal deficits","volume":"108","author":"Akten","year":"2011","journal-title":"Proc. Natl. Acad. Sci."},{"issue":"24","key":"10.1016\/j.nbd.2017.01.011_bb0015","doi-asserted-by":"crossref","first-page":"2841","DOI":"10.1093\/hmg\/10.24.2841","article-title":"Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients","volume":"10","author":"Andreassi","year":"2001","journal-title":"Hum. Mol. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0020","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1007\/s10048-001-0128-y","article-title":"Expression of the survival of motor neuron (SMN) gene in primary neurons and increase in SMN levels by activation of the N-methyl-d-aspartate glutamate receptor","volume":"4","author":"Andreassi","year":"2002","journal-title":"Neurogenetics"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0025","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1038\/sj.ejhg.5201102","article-title":"Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy","volume":"12","author":"Andreassi","year":"2003","journal-title":"Eur. J. Hum. Genet."},{"key":"10.1016\/j.nbd.2017.01.011_bb0030","article-title":"A functional motor unit in the culture dish: co-culture of spinal cord explants and muscle cells","volume":"62","author":"Anne-Sophie","year":"2012","journal-title":"J. Vis. Exp."},{"issue":"117 (3)","key":"10.1016\/j.nbd.2017.01.011_bb0035","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1172\/JCI29562","article-title":"Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy","volume":"117","author":"Avila","year":"2007","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.nbd.2017.01.011_bb0040","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.pneurobio.2014.02.005","article-title":"Insulin, IGF-1 and GLP-1 signaling in neurodegenerative disorders: targets for disease modification?","volume":"118","author":"Bassil","year":"2014","journal-title":"Prog. Neurobiol."},{"issue":"12","key":"10.1016\/j.nbd.2017.01.011_bb0045","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pgen.1000773","article-title":"Alternative splicing events are a late feature of pathology in a mouse model of spinal muscular atrophy","volume":"5","author":"B\u00e4umer","year":"2009","journal-title":"PLoS Genet."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0050","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1016\/j.nmd.2011.03.009","article-title":"Plastin 3 expression in discordant spinal muscular atrophy (SMA) siblings","volume":"21","author":"Bernal","year":"2011","journal-title":"Neuromuscul. Disord."},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0055","doi-asserted-by":"crossref","first-page":"775","DOI":"10.1093\/hmg\/8.5.775","article-title":"The RNA-binding properties of SMN: deletion analysis of the zebrafish orthologue defines domains conserved in evolution","volume":"8","author":"Bertrandy","year":"1999","journal-title":"Hum. Mol. Genet."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0060","doi-asserted-by":"crossref","first-page":"295","DOI":"10.1038\/nrm1074","article-title":"New insights into the roles of agrin","volume":"4","author":"Bezakova","year":"2003","journal-title":"Nat. Rev. Mol. Cell Biol."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0065","doi-asserted-by":"crossref","first-page":"953","DOI":"10.1523\/JNEUROSCI.3237-07.2008","article-title":"Exercise-induced activation of NMDA receptor promotes motor unit development and survival in a type 2 spinal muscular atrophy model mouse","volume":"28","author":"Biondi","year":"2008","journal-title":"J. Neurosci."},{"issue":"34","key":"10.1016\/j.nbd.2017.01.011_bb0070","doi-asserted-by":"crossref","first-page":"11288","DOI":"10.1523\/JNEUROSCI.1764-10.2010","article-title":"In vivo NMDA receptor activation accelerates motor unit maturation, protects spinal motor neurons, and enhances SMN2 gene expression in severe spinal muscular atrophy mice","volume":"30","author":"Biondi","year":"2010","journal-title":"J. Neurosci."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0075","doi-asserted-by":"crossref","first-page":"737","DOI":"10.1083\/jcb.200212140","article-title":"Colocalization of synapsin and actin during synaptic vesicle recycling","volume":"161","author":"Bloom","year":"2003","journal-title":"J. Cell Biol."},{"issue":"9","key":"10.1016\/j.nbd.2017.01.011_bb0080","doi-asserted-by":"crossref","first-page":"1844","DOI":"10.1093\/hmg\/ddr067","article-title":"Increased IGF-1 in muscle modulates the phenotype of severe SMA mice","volume":"20","author":"Bosch-Marc\u00e9","year":"2011","journal-title":"Hum. Mol. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0085","doi-asserted-by":"crossref","first-page":"66","DOI":"10.1016\/j.mcn.2009.05.009","article-title":"SMN, profilin IIa and plastin 3: a link between the deregulation of actin dynamics and SMA pathogenesis","volume":"42","author":"Bowerman","year":"2009","journal-title":"Mol. Cell. Neurosci."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0090","doi-asserted-by":"crossref","first-page":"1468","DOI":"10.1093\/hmg\/ddq021","article-title":"Rho-kinase inactivation prolongs survival of an intermediate SMA mouse model","volume":"19","author":"Bowerman","year":"2010","journal-title":"Hum. Mol. Genet."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0095","doi-asserted-by":"crossref","first-page":"256","DOI":"10.1038\/sj.ejhg.5201320","article-title":"Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients","volume":"13","author":"Brahe","year":"2004","journal-title":"Eur. J. Hum. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0100","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1113\/jphysiol.1987.sp016619","article-title":"Control of end-plate channel properties by neurotrophic effects and by muscle activity in rat","volume":"388","author":"Brenner","year":"1987","journal-title":"J. Physiol."},{"issue":"19","key":"10.1016\/j.nbd.2017.01.011_bb0105","doi-asserted-by":"crossref","first-page":"2481","DOI":"10.1093\/hmg\/ddg256","article-title":"Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy","volume":"12","author":"Brichta","year":"2003","journal-title":"Hum. Mol. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0110","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1007\/s00401-012-1058-5","article-title":"Unravelling the enigma of selective vulnerability in neurodegeneration: motor neurons resistant to degeneration in ALS show distinct gene expression characteristics and decreased susceptibility to excitotoxicity","volume":"125","author":"Brockington","year":"2013","journal-title":"Acta Neuropathol."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0115","doi-asserted-by":"crossref","first-page":"1019","DOI":"10.1261\/rna.039164.113","article-title":"Emerging complexity of the HuD\/ELAVl4 gene; implications for neuronal development, function, and dysfunction","volume":"19","author":"Bronicki","year":"2013","journal-title":"RNA"},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0120","doi-asserted-by":"crossref","first-page":"597","DOI":"10.1038\/nrn2670","article-title":"Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?","volume":"10","author":"Burghes","year":"2009","journal-title":"Nat. Rev. Neurosci."},{"issue":"43","key":"10.1016\/j.nbd.2017.01.011_bb0125","doi-asserted-by":"crossref","first-page":"11014","DOI":"10.1523\/JNEUROSCI.1637-06.2006","article-title":"Survival motor neuron function in motor axons is independent of functions required for small nuclear ribonucleoprotein biogenesis","volume":"26","author":"Carrel","year":"2006","journal-title":"J. Neurosci."},{"issue":"17","key":"10.1016\/j.nbd.2017.01.011_bb0130","doi-asserted-by":"crossref","first-page":"9808","DOI":"10.1073\/pnas.171105098","article-title":"Treatment of spinal muscular atrophy by sodium butyrate","volume":"98","author":"Chang","year":"2001","journal-title":"Proc. Natl. Acad. Sci."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0135","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0017768","article-title":"Axonal regeneration and neuronal function are preserved in motor neurons lacking ss-actin in vivo","volume":"6","author":"Cheever","year":"2011","journal-title":"PLoS One"},{"issue":"22","key":"10.1016\/j.nbd.2017.01.011_bb0140","doi-asserted-by":"crossref","first-page":"6929","DOI":"10.1128\/MCB.01332-08","article-title":"The RNA binding protein hnRNP Q modulates the utilization of exon 7 in the survival motor neuron 2 (SMN2) gene","volume":"28","author":"Chen","year":"2008","journal-title":"Mol. Cell. Biol."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0145","doi-asserted-by":"crossref","first-page":"517","DOI":"10.1016\/j.bbagrm.2014.03.003","article-title":"PSF contacts exon 7 of SMN2 pre-mRNA to promote exon 7 inclusion","volume":"1839","author":"Cho","year":"2014","journal-title":"Biochim. Biophys. Acta Gene Regul. Mech."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0150","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1016\/j.bbagrm.2014.02.006","article-title":"hnRNP M facilitates exon 7 inclusion of SMN2 pre-mRNA in spinal muscular atrophy by targeting an enhancer on exon 7","volume":"1839","author":"Cho","year":"2014","journal-title":"Biochim. Biophys. Acta Gene Regul. Mech."},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0155","doi-asserted-by":"crossref","first-page":"1107","DOI":"10.1083\/jcb.152.5.1107","article-title":"Deletion of murine SMN exon 7 directed to skeletal muscle leads to severe muscular dystrophy","volume":"152","author":"Cifuentes-Diaz","year":"2001","journal-title":"J. Cell Biol."},{"issue":"20","key":"10.1016\/j.nbd.2017.01.011_bb0160","doi-asserted-by":"crossref","first-page":"4043","DOI":"10.1093\/hmg\/ddt254","article-title":"Dilysine motifs in exon 2b of SMN protein mediate binding to the COPI vesicle protein \u03b1-COP and neurite outgrowth in a cell culture model of spinal muscular atrophy","volume":"22","author":"Custer","year":"2013","journal-title":"Hum. Mol. Genet."},{"issue":"20","key":"10.1016\/j.nbd.2017.01.011_bb0165","doi-asserted-by":"crossref","first-page":"12542","DOI":"10.1073\/pnas.192457199","article-title":"Gradients of substrate-bound laminin orient axonal specification of neurons","volume":"99","author":"Dertinger","year":"2002","journal-title":"Proc. Natl. Acad. Sci."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0170","doi-asserted-by":"crossref","first-page":"822","DOI":"10.1002\/bies.20449","article-title":"The RNA-binding protein HuD: a regulator of neuronal differentiation, maintenance and plasticity","volume":"28","author":"Desch\u00eanes-Furry","year":"2006","journal-title":"BioEssays"},{"issue":"10","key":"10.1016\/j.nbd.2017.01.011_bb0175","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0110846","article-title":"Presynaptic localization of Smn and hnRNP R in axon terminals of embryonic and postnatal mouse motoneurons","volume":"9","author":"Dombert","year":"2014","journal-title":"PLoS One"},{"key":"10.1016\/j.nbd.2017.01.011_bb0180","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1016\/j.conb.2016.04.004","article-title":"A role for the survival of motor neuron protein in mRNP assembly and transport","volume":"39","author":"Donlin-Asp","year":"2016","journal-title":"Curr. Opin. Neurobiol."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0185","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1016\/S0300-9084(00)00217-0","article-title":"The actin cytoskeleton and neurotransmitter release: an overview","volume":"82","author":"Doussau","year":"2000","journal-title":"Biochimie"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0190","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1097\/GIM.0b013e31802d8353","article-title":"X-linked infantile spinal muscular atrophy: clinical definition and molecular mapping","volume":"9","author":"Dressman","year":"2007","journal-title":"Genitourin. Med."},{"key":"10.1016\/j.nbd.2017.01.011_bb0195","first-page":"iii","article-title":"Muscle disorders in childhood","volume":"16","author":"Dubowitz","year":"1978","journal-title":"Major Probl. Clin. Pediatr."},{"issue":"12","key":"10.1016\/j.nbd.2017.01.011_bb0200","doi-asserted-by":"crossref","first-page":"2284","DOI":"10.1002\/cne.22332","article-title":"Identification of novel spinal cholinergic genetic subtypes disclose Chodl and Pitx2 as markers for fast motor neurons and partition cells","volume":"518","author":"Enjin","year":"2010","journal-title":"J. Comp. Neurol."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0205","doi-asserted-by":"crossref","first-page":"602","DOI":"10.1038\/ng1354","article-title":"Mutant small heat-shock protein 27 causes axonal Charcot-Marie-Tooth disease and distal hereditary motor neuropathy","volume":"36","author":"Evgrafov","year":"2004","journal-title":"Nat. Genet."},{"issue":"10","key":"10.1016\/j.nbd.2017.01.011_bb0210","doi-asserted-by":"crossref","first-page":"3914","DOI":"10.1523\/JNEUROSCI.3631-10.2011","article-title":"The survival of motor neuron (SMN) protein interacts with the mRNA-binding protein HuD and regulates localization of poly (A) mRNA in primary motor neuron axons","volume":"31","author":"Fallini","year":"2011","journal-title":"J. Neurosci."},{"key":"10.1016\/j.nbd.2017.01.011_bb0215","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1016\/j.brainres.2012.01.044","article-title":"Spinal muscular atrophy: the role of SMN in axonal mRNA regulation","volume":"1462","author":"Fallini","year":"2012","journal-title":"Brain Res."},{"issue":"16","key":"10.1016\/j.nbd.2017.01.011_bb0220","doi-asserted-by":"crossref","first-page":"3703","DOI":"10.1093\/hmg\/dds205","article-title":"The ALS disease protein TDP-43 is actively transported in motor neuron axons and regulates axon outgrowth","volume":"21","author":"Fallini","year":"2012","journal-title":"Hum. Mol. Genet."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0225","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1002\/dneu.22111","article-title":"Dynamics of survival of motor neuron (SMN) protein interaction with the mRNA-binding protein IMP1 facilitates its trafficking into motor neuron axons","volume":"74","author":"Fallini","year":"2014","journal-title":"Dev. Neurobiol."},{"issue":"13","key":"10.1016\/j.nbd.2017.01.011_bb0230","doi-asserted-by":"crossref","first-page":"3811","DOI":"10.1523\/JNEUROSCI.2396-15.2016","article-title":"Deficiency of the survival of motor neuron protein impairs mRNA localization and local translation in the growth cone of motor neurons","volume":"36","author":"Fallini","year":"2016","journal-title":"J. Neurosci."},{"issue":"24","key":"10.1016\/j.nbd.2017.01.011_bb0235","doi-asserted-by":"crossref","first-page":"2674","DOI":"10.1101\/gad.176172.111","article-title":"CPG15 regulates synapse stability in the developing and adult brain","volume":"25","author":"Fujino","year":"2011","journal-title":"Genes Dev."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0240","doi-asserted-by":"crossref","first-page":"2273","DOI":"10.1128\/MCB.26.6.2273-2285.2006","article-title":"CARM1 regulates proliferation of PC12 cells by methylating HuD","volume":"26","author":"Fujiwara","year":"2006","journal-title":"Mol. Cell. Biol."},{"issue":"9","key":"10.1016\/j.nbd.2017.01.011_bb0245","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0000921","article-title":"Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPs","volume":"2","author":"Gabanella","year":"2007","journal-title":"PLoS One"},{"key":"10.1016\/j.nbd.2017.01.011_bb0250","article-title":"Big win possible for Ionis\/Biogen antisense drug in muscular atrophy","author":"Garber","year":"2016","journal-title":"Nat. Res."},{"issue":"19","key":"10.1016\/j.nbd.2017.01.011_bb0255","doi-asserted-by":"crossref","first-page":"3645","DOI":"10.1093\/hmg\/ddp313","article-title":"LBH589 induces up to 10-fold SMN protein levels by several independent mechanisms and is effective even in cells from SMA patients non-responsive to valproate","volume":"18","author":"Garbes","year":"2009","journal-title":"Hum. Mol. Genet."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0260","doi-asserted-by":"crossref","first-page":"1063","DOI":"10.1093\/hmg\/ddm379","article-title":"Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect","volume":"17","author":"Gavrilina","year":"2008","journal-title":"Hum. Mol. Genet."},{"issue":"10","key":"10.1016\/j.nbd.2017.01.011_bb0265","doi-asserted-by":"crossref","first-page":"1951","DOI":"10.1093\/hmg\/ddq073","article-title":"The heterogeneous nuclear ribonucleoprotein-R is necessary for axonal \u03b2-actin mRNA translocation in spinal motor neurons","volume":"19","author":"Glinka","year":"2010","journal-title":"Hum. Mol. Genet."},{"issue":"11","key":"10.1016\/j.nbd.2017.01.011_bb0270","doi-asserted-by":"crossref","first-page":"3818","DOI":"10.1523\/JNEUROSCI.5775-11.2012","article-title":"Motor neuron rescue in spinal muscular atrophy mice demonstrates that sensory-motor defects are a consequence, not a cause, of motor neuron dysfunction","volume":"32","author":"Gogliotti","year":"2012","journal-title":"J. Neurosci."},{"issue":"10","key":"10.1016\/j.nbd.2017.01.011_bb0275","doi-asserted-by":"crossref","first-page":"1927","DOI":"10.1093\/hmg\/4.10.1927","article-title":"Molecular analysis of candidate genes on chromosome 5q13 in autosomal recessive spinal muscular atrophy: evidence of homozygous deletions of the SMN gene in unaffected individuals","volume":"4","author":"Hahnen","year":"1995","journal-title":"Hum. Mol. Genet."},{"issue":"29","key":"10.1016\/j.nbd.2017.01.011_bb0280","doi-asserted-by":"crossref","first-page":"13111","DOI":"10.1073\/pnas.1006151107","article-title":"miRNA malfunction causes spinal motor neuron disease","volume":"107","author":"Haramati","year":"2010","journal-title":"Proc. Natl. Acad. Sci."},{"issue":"22","key":"10.1016\/j.nbd.2017.01.011_bb0285","doi-asserted-by":"crossref","first-page":"1714","DOI":"10.1212\/WNL.0b013e3182556c05","article-title":"Mutations in the tail domain of DYNC1H1 cause dominant spinal muscular atrophy","volume":"78","author":"Harms","year":"2012","journal-title":"Neurology"},{"issue":"20","key":"10.1016\/j.nbd.2017.01.011_bb0290","doi-asserted-by":"crossref","first-page":"2720","DOI":"10.1101\/gad.908401","article-title":"Coilin forms the bridge between Cajal bodies and SMN, the spinal muscular atrophy protein","volume":"15","author":"Hebert","year":"2001","journal-title":"Genes Dev."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0295","doi-asserted-by":"crossref","first-page":"2313","DOI":"10.1093\/brain\/awq167","article-title":"Global gene expression profiling of somatic motor neuron populations with different vulnerability identify molecules and pathways of degeneration and protection","volume":"133","author":"Hedlund","year":"2010","journal-title":"Brain"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0300","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1007\/s00439-003-1025-2","article-title":"Evidence for a modifying pathway in SMA discordant families: reduced SMN level decreases the amount of its interacting partners and Htra2-beta1","volume":"114","author":"Helmken","year":"2003","journal-title":"Hum. Genet."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0305","doi-asserted-by":"crossref","first-page":"1361","DOI":"10.1212\/WNL.37.8.1361","article-title":"Extracts of muscle biopsies from patients with spinal muscular atrophies inhibit neurite outgrowth from spinal neurons","volume":"37","author":"Henderson","year":"1987","journal-title":"Neurology"},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0310","doi-asserted-by":"crossref","first-page":"670","DOI":"10.1111\/nan.12148","article-title":"Loss of nuclear TDP-43 in amyotrophic lateral sclerosis (ALS) causes altered expression of splicing machinery and widespread dysregulation of RNA splicing in motor neurones","volume":"40","author":"Highley","year":"2014","journal-title":"Neuropathol. Appl. Neurobiol."},{"issue":"17","key":"10.1016\/j.nbd.2017.01.011_bb0315","doi-asserted-by":"crossref","first-page":"2037","DOI":"10.1093\/hmg\/11.17.2037","article-title":"hnRNP-G promotes exon 7 inclusion of survival motor neuron (SMN) via direct interaction with Htra2-\u03b21","volume":"11","author":"Hofmann","year":"2002","journal-title":"Hum. Mol. Genet."},{"issue":"17","key":"10.1016\/j.nbd.2017.01.011_bb0320","doi-asserted-by":"crossref","first-page":"9618","DOI":"10.1073\/pnas.160181697","article-title":"Htra2-\u03b21 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2)","volume":"97","author":"Hofmann","year":"2000","journal-title":"Proc. Natl. Acad. Sci."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0325","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1016\/j.ajhg.2016.07.014","article-title":"The power of human protective modifiers: PLS3 and CORO1C unravel impaired endocytosis in spinal muscular atrophy and rescue SMA phenotype","volume":"99","author":"Hosseinibarkooie","year":"2016","journal-title":"Am. J. Hum. Genet."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0330","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pbio.0050073","article-title":"Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon","volume":"5","author":"Hua","year":"2007","journal-title":"PLoS Biol."},{"issue":"7367","key":"10.1016\/j.nbd.2017.01.011_bb0335","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1038\/nature10485","article-title":"Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model","volume":"478","author":"Hua","year":"2011","journal-title":"Nature"},{"key":"10.1016\/j.nbd.2017.01.011_bb0340","doi-asserted-by":"crossref","DOI":"10.1101\/gad.256644.114","article-title":"Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models","author":"Hua","year":"2015","journal-title":"Genes Dev."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0345","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1093\/hmg\/ddq500","article-title":"HuD interacts with survival motor neuron protein and can rescue spinal muscular atrophy-like neuronal defects","volume":"20","author":"Hubers","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.nbd.2017.01.011_bb0350","first-page":"ddt612","article-title":"SMN-dependent intrinsic defects in Schwann cells in mouse models of spinal muscular atrophy","author":"Hunter","year":"2013","journal-title":"Hum. Mol. Genet."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0355","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1016\/j.cell.2012.09.011","article-title":"SMN is required for sensory-motor circuit function in Drosophila","volume":"151","author":"Imlach","year":"2012","journal-title":"Cell"},{"issue":"Suppl. 2","key":"10.1016\/j.nbd.2017.01.011_bb0360","doi-asserted-by":"crossref","first-page":"R195","DOI":"10.1093\/hmg\/ddh226","article-title":"Molecular genetics of distal hereditary motor neuropathies","volume":"13","author":"Irobi","year":"2004","journal-title":"Hum. Mol. Genet."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0365","doi-asserted-by":"crossref","first-page":"597","DOI":"10.1038\/ng1328","article-title":"Hot-spot residue in small heat-shock protein 22 causes distal motor neuropathy","volume":"36","author":"Irobi","year":"2004","journal-title":"Nat. Genet."},{"issue":"20","key":"10.1016\/j.nbd.2017.01.011_bb0370","doi-asserted-by":"crossref","first-page":"4136","DOI":"10.1093\/hmg\/ddt262","article-title":"Decreased number of Gemini of coiled bodies and U12 snRNA level in amyotrophic lateral sclerosis","volume":"22","author":"Ishihara","year":"2013","journal-title":"Hum. Mol. Genet."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0375","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1002\/ana.410040309","article-title":"Sparing of the Onufrowicz nucleus in sacral anterior horn lesions","volume":"4","author":"Iwata","year":"1978","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.nbd.2017.01.011_bb0380","first-page":"ddv332","article-title":"Low levels of survival motor neuron protein are sufficient for normal muscle function in the SMN\u03947 mouse model of SMA","author":"Iyer","year":"2015","journal-title":"Hum. Mol. Genet."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0385","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1093\/hmg\/ddi467","article-title":"Distinct and overlapping alterations in motor and sensory neurons in a mouse model of spinal muscular atrophy","volume":"15","author":"Jablonka","year":"2006","journal-title":"Hum. Mol. Genet."},{"issue":"24","key":"10.1016\/j.nbd.2017.01.011_bb0390","doi-asserted-by":"crossref","first-page":"4906","DOI":"10.1093\/hmg\/ddq425","article-title":"A feedback loop regulates splicing of the spinal muscular atrophy-modifying gene, SMN2","volume":"19","author":"Jodelka","year":"2010","journal-title":"Hum. Mol. Genet."},{"issue":"36","key":"10.1016\/j.nbd.2017.01.011_bb0395","doi-asserted-by":"crossref","first-page":"10817","DOI":"10.1021\/bi971491b","article-title":"Stathmin: a tubulin-sequestering protein which forms a ternary T2S complex with two tubulin molecules","volume":"36","author":"Jourdain","year":"1997","journal-title":"Biochemistry"},{"issue":"17","key":"10.1016\/j.nbd.2017.01.011_bb0400","doi-asserted-by":"crossref","first-page":"7165","DOI":"10.1523\/JNEUROSCI.2040-12.2013","article-title":"Intra-axonal synthesis of eukaryotic translation initiation factors regulates local protein synthesis and axon growth in rat sympathetic neurons","volume":"33","author":"Kar","year":"2013","journal-title":"J. Neurosci."},{"issue":"16","key":"10.1016\/j.nbd.2017.01.011_bb0405","doi-asserted-by":"crossref","first-page":"2552","DOI":"10.1093\/hmg\/ddn156","article-title":"Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy","volume":"17","author":"Kariya","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.nbd.2017.01.011_bb0410","first-page":"dds174","article-title":"Mutant superoxide dismutase 1 (SOD1), a cause of amyotrophic lateral sclerosis, disrupts the recruitment of SMN, the spinal muscular atrophy protein to nuclear Cajal bodies","author":"Kariya","year":"2012","journal-title":"Hum. Mol. Genet."},{"issue":"24","key":"10.1016\/j.nbd.2017.01.011_bb0415","doi-asserted-by":"crossref","first-page":"3149","DOI":"10.1093\/hmg\/ddm276","article-title":"hnRNP A1 functions with specificity in repression of SMN2 exon 7 splicing","volume":"16","author":"Kashima","year":"2007","journal-title":"Hum. Mol. Genet."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0420","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1093\/brain\/awq345","article-title":"Phosphatase and tensin homologue\/protein kinase B pathway linked to motor neuron survival in human superoxide dismutase 1-related amyotrophic lateral sclerosis","volume":"134","author":"Kirby","year":"2011","journal-title":"Brain"},{"issue":"10","key":"10.1016\/j.nbd.2017.01.011_bb0425","doi-asserted-by":"crossref","first-page":"3131","DOI":"10.1523\/JNEUROSCI.07-10-03131.1987","article-title":"Human muscle cultured in monolayer and cocultured with fetal rat spinal cord: importance of dorsal root ganglia for achieving successful functional innervation","volume":"7","author":"Kobayashi","year":"1987","journal-title":"J. Neurosci."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0430","doi-asserted-by":"crossref","first-page":"502","DOI":"10.1212\/WNL.0b013e3181cef84a","article-title":"Mutant small heat shock protein B3 causes motor neuropathy utility of a candidate gene approach","volume":"74","author":"Kolb","year":"2010","journal-title":"Neurology"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0435","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1017\/S0012162200000116","article-title":"New ocular movement detector system as a communication tool in ventilator-assisted Werdnig-Hoffmann disease","volume":"42","author":"Kubota","year":"2000","journal-title":"Dev. Med. Child Neurol."},{"key":"10.1016\/j.nbd.2017.01.011_bb0440","first-page":"ddu350","article-title":"SMN regulates axonal local translation via miR-183\/mTOR pathway","author":"Kye","year":"2014","journal-title":"Hum. Mol. Genet."},{"issue":"9","key":"10.1016\/j.nbd.2017.01.011_bb0445","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0046353","article-title":"Limited phenotypic effects of selectively augmenting the SMN protein in the neurons of a mouse model of severe spinal muscular atrophy","volume":"7","author":"Lee","year":"2012","journal-title":"PLoS One"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0450","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1016\/0092-8674(95)90460-3","article-title":"Identification and characterization of a spinal muscular atrophy-determining gene","volume":"80","author":"Lefebvre","year":"1995","journal-title":"Cell"},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0455","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1038\/ng0797-265","article-title":"Correlation between severity and SMN protein level in spinal muscular atrophy","volume":"16","author":"Lefebvre","year":"1997","journal-title":"Nat. Genet."},{"issue":"11","key":"10.1016\/j.nbd.2017.01.011_bb0460","doi-asserted-by":"crossref","first-page":"1233","DOI":"10.1093\/hmg\/ddg143","article-title":"Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy","volume":"12","author":"Lesbordes","year":"2003","journal-title":"Hum. Mol. Genet."},{"issue":"19","key":"10.1016\/j.nbd.2017.01.011_bb0465","doi-asserted-by":"crossref","first-page":"4006","DOI":"10.1093\/nar\/29.19.4006","article-title":"A computational scan for U12-dependent introns in the human genome sequence","volume":"29","author":"Levine","year":"2001","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.nbd.2017.01.011_bb0470","series-title":"Seminars in cell & developmental biology","article-title":"SMN control of RNP assembly: from post-transcriptional gene regulation to motor neuron disease","author":"Li","year":"2014"},{"issue":"48","key":"10.1016\/j.nbd.2017.01.011_bb0475","doi-asserted-by":"crossref","first-page":"45476","DOI":"10.1074\/jbc.M107632200","article-title":"Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3\u2032 splice site pairing","volume":"276","author":"Lim","year":"2001","journal-title":"J. Biol. Chem."},{"issue":"11","key":"10.1016\/j.nbd.2017.01.011_bb0480","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0015457","article-title":"Synaptic defects in the spinal and neuromuscular circuitry in a mouse model of spinal muscular atrophy","volume":"5","author":"Ling","year":"2010","journal-title":"PLoS One"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0485","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1093\/hmg\/ddr453","article-title":"Severe neuromuscular denervation of clinically relevant muscles in a mouse model of spinal muscular atrophy","volume":"21","author":"Ling","year":"2012","journal-title":"Hum. Mol. Genet."},{"issue":"16","key":"10.1016\/j.nbd.2017.01.011_bb0490","doi-asserted-by":"crossref","first-page":"1833","DOI":"10.1007\/s00018-006-6061-3","article-title":"The neuroligin and neurexin families: from structure to function at the synapse","volume":"63","author":"Lise","year":"2006","journal-title":"Cell. Mol. Life Sci. CMLS"},{"key":"10.1016\/j.nbd.2017.01.011_bb0495","article-title":"PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy","author":"Little","year":"2014","journal-title":"Mol. Ther."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0500","doi-asserted-by":"crossref","first-page":"270","DOI":"10.1038\/mt.2014.209","article-title":"PTEN depletion decreases disease severity and modestly prolongs survival in a mouse model of spinal muscular atrophy","volume":"23","author":"Little","year":"2015","journal-title":"Mol. Ther."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0505","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1002\/jnr.22189","article-title":"Neurodevelopmental consequences of Smn depletion in a mouse model of spinal muscular atrophy","volume":"88","author":"Liu","year":"2010","journal-title":"J. Neurosci. Res."},{"issue":"45","key":"10.1016\/j.nbd.2017.01.011_bb0510","doi-asserted-by":"crossref","first-page":"16086","DOI":"10.1523\/JNEUROSCI.4105-11.2011","article-title":"Local RNA translation at the synapse and in disease","volume":"31","author":"Liu-Yesucevitz","year":"2011","journal-title":"J. Neurosci."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0515","doi-asserted-by":"crossref","first-page":"440","DOI":"10.1016\/j.cell.2012.09.012","article-title":"An SMN-dependent U12 splicing event essential for motor circuit function","volume":"151","author":"Lotti","year":"2012","journal-title":"Cell"},{"issue":"9630","key":"10.1016\/j.nbd.2017.01.011_bb0520","doi-asserted-by":"crossref","first-page":"2120","DOI":"10.1016\/S0140-6736(08)60921-6","article-title":"Spinal muscular atrophy","volume":"371","author":"Lunn","year":"2008","journal-title":"Lancet"},{"issue":"15","key":"10.1016\/j.nbd.2017.01.011_bb0525","doi-asserted-by":"crossref","first-page":"14803","DOI":"10.1074\/jbc.M308225200","article-title":"Identification of a novel cyclic AMP-response element (CRE-II) and the role of CREB-1 in the cAMP-induced expression of the survival motor neuron (SMN) gene","volume":"279","author":"Majumder","year":"2004","journal-title":"J. Biol. Chem."},{"issue":"12","key":"10.1016\/j.nbd.2017.01.011_bb0530","doi-asserted-by":"crossref","first-page":"679","DOI":"10.1038\/ncpneuro0355","article-title":"Mechanisms of disease: astrocytes in neurodegenerative disease","volume":"2","author":"Maragakis","year":"2006","journal-title":"Nat. Clin. Pract. Neurol."},{"issue":"10","key":"10.1016\/j.nbd.2017.01.011_bb0535","doi-asserted-by":"crossref","first-page":"3663","DOI":"10.1523\/JNEUROSCI.20-10-03663.2000","article-title":"From plaque to pretzel: fold formation and acetylcholine receptor loss at the developing neuromuscular junction","volume":"20","author":"Marques","year":"2000","journal-title":"J. Neurosci."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0540","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1016\/0022-510X(75)90207-5","article-title":"Sensory system involvement in infantile spinal muscular atrophy","volume":"26","author":"Marshall","year":"1975","journal-title":"J. Neurol. Sci."},{"issue":"25","key":"10.1016\/j.nbd.2017.01.011_bb0545","doi-asserted-by":"crossref","first-page":"8703","DOI":"10.1523\/JNEUROSCI.0204-12.2012","article-title":"Survival motor neuron protein in motor neurons determines synaptic integrity in spinal muscular atrophy","volume":"32","author":"Martinez","year":"2012","journal-title":"J. Neurosci."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0550","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1002\/path.4080","article-title":"Synaptic defects in type I spinal muscular atrophy in human development","volume":"229","author":"Mart\u00ednez-Hern\u00e1ndez","year":"2013","journal-title":"J. Pathol."},{"issue":"9","key":"10.1016\/j.nbd.2017.01.011_bb0555","doi-asserted-by":"crossref","first-page":"1418","DOI":"10.1002\/glia.22522","article-title":"Spinal muscular atrophy astrocytes exhibit abnormal calcium regulation and reduced growth factor production","volume":"61","author":"McGivern","year":"2013","journal-title":"Glia"},{"issue":"18","key":"10.1016\/j.nbd.2017.01.011_bb0560","doi-asserted-by":"crossref","first-page":"2900","DOI":"10.1093\/hmg\/ddn189","article-title":"Embryonic motor axon development in the severe SMA mouse","volume":"17","author":"McGovern","year":"2008","journal-title":"Hum. Mol. Genet."},{"issue":"7","key":"10.1016\/j.nbd.2017.01.011_bb0565","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0132364","article-title":"Plastin 3 expression does not modify spinal muscular atrophy severity in the \u22067 SMA mouse","volume":"10","author":"McGovern","year":"2015","journal-title":"PLoS One"},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0570","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1083\/jcb.200303168","article-title":"Knockdown of the survival motor neuron (Smn) protein in zebrafish causes defects in motor axon outgrowth and pathfinding","volume":"162","author":"McWhorter","year":"2003","journal-title":"J. Cell Biol."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0575","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1002\/dneu.20582","article-title":"The SMN binding protein Gemin2 is not involved in motor axon outgrowth","volume":"68","author":"McWhorter","year":"2008","journal-title":"Dev. Neurobiol."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0580","doi-asserted-by":"crossref","first-page":"453","DOI":"10.1016\/j.neuron.2010.12.032","article-title":"Early functional impairment of sensory-motor connectivity in a mouse model of spinal muscular atrophy","volume":"69","author":"Mentis","year":"2011","journal-title":"Neuron"},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0585","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1016\/j.nmd.2003.11.006","article-title":"Pilot trial of phenylbutyrate in spinal muscular atrophy","volume":"14","author":"Mercuri","year":"2004","journal-title":"Neuromuscul. Disord."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0590","doi-asserted-by":"crossref","first-page":"432","DOI":"10.1016\/j.ydbio.2011.05.667","article-title":"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","volume":"356","author":"Mikesh","year":"2011","journal-title":"Dev. Biol."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0595","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1038\/nrn3380","article-title":"Axonal transport deficits and neurodegenerative diseases","volume":"14","author":"Millecamps","year":"2013","journal-title":"Nat. Rev. Neurosci."},{"issue":"6943","key":"10.1016\/j.nbd.2017.01.011_bb0600","doi-asserted-by":"crossref","first-page":"939","DOI":"10.1038\/nature01755","article-title":"\u03b1-neurexins couple Ca2&plus; channels to synaptic vesicle exocytosis","volume":"423","author":"Missler","year":"2003","journal-title":"Nature"},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0605","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0062114","article-title":"Improved antisense oligonucleotide design to suppress aberrant SMN2 gene transcript processing: towards a treatment for spinal muscular atrophy","volume":"8","author":"Mitrpant","year":"2013","journal-title":"PLoS One"},{"issue":"7","key":"10.1016\/j.nbd.2017.01.011_bb0610","doi-asserted-by":"crossref","first-page":"949","DOI":"10.1093\/hmg\/ddm367","article-title":"Selective vulnerability of motor neurons and dissociation of pre-and post-synaptic pathology at the neuromuscular junction in mouse models of spinal muscular atrophy","volume":"17","author":"Murray","year":"2008","journal-title":"Hum. Mol. Genet."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0615","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1093\/hmg\/ddp506","article-title":"Pre-symptomatic development of lower motor neuron connectivity in a mouse model of severe spinal muscular atrophy","volume":"19","author":"Murray","year":"2010","journal-title":"Hum. Mol. Genet."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0620","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1111\/j.1365-2990.2010.01061.x","article-title":"Review: neuromuscular synaptic vulnerability in motor neurone disease: amyotrophic lateral sclerosis and spinal muscular atrophy","volume":"36","author":"Murray","year":"2010","journal-title":"Neuropathol. Appl. Neurobiol."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0625","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s40478-015-0231-1","article-title":"Transcriptional profiling of differentially vulnerable motor neurons at pre-symptomatic stage in the Smn 2b\/-mouse model of spinal muscular atrophy","volume":"3","author":"Murray","year":"2015","journal-title":"Acta neuropathol. Commun."},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0630","doi-asserted-by":"crossref","first-page":"615","DOI":"10.1038\/nn1876","article-title":"Astrocytes expressing ALS-linked mutated SOD1 release factors selectively toxic to motor neurons","volume":"10","author":"Nagai","year":"2007","journal-title":"Nat. Neurosci."},{"issue":"6197","key":"10.1016\/j.nbd.2017.01.011_bb0635","doi-asserted-by":"crossref","first-page":"688","DOI":"10.1126\/science.1250127","article-title":"SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy","volume":"345","author":"Naryshkin","year":"2014","journal-title":"Science"},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0640","doi-asserted-by":"crossref","first-page":"946","DOI":"10.1016\/j.ajhg.2013.04.011","article-title":"Mutations in BICD2, which encodes a golgin and important motor adaptor, cause congenital autosomal-dominant spinal muscular atrophy","volume":"92","author":"Neveling","year":"2013","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.nbd.2017.01.011_bb0645","doi-asserted-by":"crossref","DOI":"10.1016\/j.stem.2015.08.003","article-title":"Genome-wide RNA-Seq of human motor neurons implicates selective ER stress activation in spinal muscular atrophy","author":"Ng","year":"2015","journal-title":"Cell Stem Cell"},{"issue":"16","key":"10.1016\/j.nbd.2017.01.011_bb0650","doi-asserted-by":"crossref","first-page":"3159","DOI":"10.1093\/hmg\/ddq226","article-title":"PTEN depletion rescues axonal growth defect and improves survival in SMN-deficient motor neurons","volume":"19","author":"Ning","year":"2010","journal-title":"Hum. Mol. Genet."},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0655","doi-asserted-by":"crossref","first-page":"822","DOI":"10.1086\/425287","article-title":"A mutation in the vesicle-trafficking protein VAPB causes late-onset spinal muscular atrophy and amyotrophic lateral sclerosis","volume":"75","author":"Nishimura","year":"2004","journal-title":"Am. J. Hum. Genet."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0660","doi-asserted-by":"crossref","first-page":"340","DOI":"10.1016\/j.clinthera.2014.02.004","article-title":"Effect of combined systemic and local morpholino treatment on the spinal muscular atrophy \u03947 mouse model phenotype","volume":"36","author":"Nizzardo","year":"2014","journal-title":"Clin. Ther."},{"issue":"24","key":"10.1016\/j.nbd.2017.01.011_bb0665","doi-asserted-by":"crossref","first-page":"4865","DOI":"10.1093\/hmg\/ddr425","article-title":"The spinal muscular atrophy disease protein SMN is linked to the rho-kinase pathway via profilin","volume":"20","author":"N\u00f6lle","year":"2011","journal-title":"Hum. Mol. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0670","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1111\/neup.12070","article-title":"Minor splicing pathway is not minor any more: implications for the pathogenesis of motor neuron diseases","volume":"34","author":"Onodera","year":"2014","journal-title":"Neuropathology"},{"issue":"5875","key":"10.1016\/j.nbd.2017.01.011_bb0675","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1126\/science.1155085","article-title":"Plastin 3 is a protective modifier of autosomal recessive spinal muscular atrophy","volume":"320","author":"Oprea","year":"2008","journal-title":"Science"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0680","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1038\/mt.2011.232","article-title":"Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy","volume":"20","author":"Osman","year":"2011","journal-title":"Mol. Ther."},{"issue":"36","key":"10.1016\/j.nbd.2017.01.011_bb0685","doi-asserted-by":"crossref","first-page":"12005","DOI":"10.1523\/JNEUROSCI.2208-10.2010","article-title":"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","volume":"30","author":"Park","year":"2010","journal-title":"J. Neurosci."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0690","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1016\/j.brainresrev.2009.11.008","article-title":"Gliotransmission: exocytotic release from astrocytes","volume":"63","author":"Parpura","year":"2010","journal-title":"Brain Res. Rev."},{"issue":"72","key":"10.1016\/j.nbd.2017.01.011_bb0695","doi-asserted-by":"crossref","first-page":"72ra18","DOI":"10.1126\/scitranslmed.3001777","article-title":"Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy","volume":"3","author":"Passini","year":"2011","journal-title":"Sci. Transl. Med."},{"issue":"7","key":"10.1016\/j.nbd.2017.01.011_bb0700","doi-asserted-by":"crossref","first-page":"1235","DOI":"10.1038\/emboj.2010.19","article-title":"The splicing regulator Sam68 binds to a novel exonic splicing silencer and functions in SMN2 alternative splicing in spinal muscular atrophy","volume":"29","author":"Pedrotti","year":"2010","journal-title":"EMBO J."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0705","doi-asserted-by":"crossref","first-page":"955","DOI":"10.1016\/j.ajhg.2013.04.013","article-title":"Molecular defects in the motor adaptor BICD2 cause proximal spinal muscular atrophy with autosomal-dominant inheritance","volume":"92","author":"Peeters","year":"2013","journal-title":"Am. J. Hum. Genet."},{"issue":"5599","key":"10.1016\/j.nbd.2017.01.011_bb0710","doi-asserted-by":"crossref","first-page":"1775","DOI":"10.1126\/science.1074962","article-title":"Essential role for the SMN complex in the specificity of snRNP assembly","volume":"298","author":"Pellizzoni","year":"2002","journal-title":"Science"},{"issue":"9","key":"10.1016\/j.nbd.2017.01.011_bb0715","doi-asserted-by":"crossref","first-page":"1701","DOI":"10.1093\/hmg\/ddr046","article-title":"The COPI vesicle complex binds and moves with survival motor neuron within axons","volume":"20","author":"Peter","year":"2011","journal-title":"Hum. Mol. Genet."},{"issue":"7","key":"10.1016\/j.nbd.2017.01.011_bb0720","doi-asserted-by":"crossref","first-page":"1625","DOI":"10.1093\/hmg\/ddr600","article-title":"A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse","volume":"21","author":"Porensky","year":"2012","journal-title":"Hum. Mol. Genet."},{"issue":"11","key":"10.1016\/j.nbd.2017.01.011_bb0725","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.87908","article-title":"Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy","volume":"1","author":"Powis","year":"2016","journal-title":"JCI Insight"},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0730","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1038\/ng1123","article-title":"Mutant dynactin in motor neuron disease","volume":"33","author":"Puls","year":"2003","journal-title":"Nat. Genet."},{"issue":"12","key":"10.1016\/j.nbd.2017.01.011_bb0735","doi-asserted-by":"crossref","first-page":"1755","DOI":"10.1261\/rna.040204.113","article-title":"Genome-wide identification of mRNAs associated with the protein SMN whose depletion decreases their axonal localization","volume":"19","author":"Rage","year":"2013","journal-title":"RNA"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0740","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1016\/j.ajhg.2007.09.009","article-title":"Rare missense and synonymous variants in UBE1 are associated with X-linked infantile spinal muscular atrophy","volume":"82","author":"Ramser","year":"2008","journal-title":"Am. J. Hum. Genet."},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0745","doi-asserted-by":"crossref","first-page":"737","DOI":"10.1007\/s00418-012-0989-1","article-title":"Laminin induced local axonal translation of \u03b2-actin mRNA is impaired in SMN-deficient motoneurons","volume":"138","author":"Rathod","year":"2012","journal-title":"Histochem. Cell Biol."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0750","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1016\/S0370-4475(83)80042-2","article-title":"Electromyographic study of 50 cases of Werdnig-Hoffmann disease","volume":"13","author":"Renault","year":"1983","journal-title":"Rev. Electroencephalogr. Neurophysiol. Clin."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0755","doi-asserted-by":"crossref","first-page":"1492","DOI":"10.1093\/hmg\/ddq023","article-title":"SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy","volume":"19","author":"Riessland","year":"2010","journal-title":"Hum. Mol. Genet."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0760","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1006\/mcne.1997.0622","article-title":"\u03b3-AChR\/\u03f5-AChR switch at agrin-induced postsynaptic-like apparatus in skeletal muscle","volume":"9","author":"Rimer","year":"1997","journal-title":"Mol. Cell. Neurosci."},{"key":"10.1016\/j.nbd.2017.01.011_bb0765","first-page":"ddv148","article-title":"Astrocytes influence the severity of spinal muscular atrophy","author":"Rindt","year":"2015","journal-title":"Hum. Mol. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0770","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1093\/hmg\/11.1.93","article-title":"Specific interaction of Smn, the spinal muscular atrophy determining gene product, with hnRNP-R and gry-rbp\/hnRNP-Q: a role for Smn in RNA processing in motor axons?","volume":"11","author":"Rossoll","year":"2002","journal-title":"Hum. Mol. Genet."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0775","doi-asserted-by":"crossref","first-page":"801","DOI":"10.1083\/jcb.200304128","article-title":"Smn, the spinal muscular atrophy\u2013determining gene product, modulates axon growth and localization of \u03b2-actin mRNA in growth cones of motoneurons","volume":"163","author":"Rossoll","year":"2003","journal-title":"J. Cell Biol."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0780","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1136\/jnnp-2011-300952","article-title":"The distal hereditary motor neuropathies","volume":"83","author":"Rossor","year":"2012","journal-title":"J. Neurol. Neurosurg. Psychiatry"},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0785","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1093\/brain\/awu356","article-title":"Phenotypic and molecular insights into spinal muscular atrophy due to mutations in BICD2","volume":"138","author":"Rossor","year":"2015","journal-title":"Brain"},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0790","doi-asserted-by":"crossref","first-page":"983","DOI":"10.1212\/01.WNL.0000052788.39340.45","article-title":"Classical infantile spinal muscular atrophy with SMN deficiency causes sensory neuronopathy","volume":"60","author":"Rudnik-Sch\u00f6neborn","year":"2003","journal-title":"Neurology"},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0795","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1128\/MCB.06077-11","article-title":"A role for SMN exon 7 splicing in the selective vulnerability of motor neurons in spinal muscular atrophy","volume":"32","author":"Ruggiu","year":"2012","journal-title":"Mol. Cell. Biol."},{"key":"10.1016\/j.nbd.2017.01.011_bb0800","doi-asserted-by":"crossref","DOI":"10.1261\/rna.047373.114","article-title":"Subcellular transcriptome alterations in a cell culture model of spinal muscular atrophy point to widespread defects in axonal growth and presynaptic differentiation","author":"Saal","year":"2014","journal-title":"RNA"},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0805","doi-asserted-by":"crossref","first-page":"668","DOI":"10.1093\/hmg\/dds474","article-title":"A novel function for the survival motoneuron protein as a translational regulator","volume":"22","author":"Sanchez","year":"2013","journal-title":"Hum. Mol. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0810","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1016\/j.neuron.2011.06.031","article-title":"Selective neuronal vulnerability in neurodegenerative diseases: from stressor thresholds to degeneration","volume":"71","author":"Saxena","year":"2011","journal-title":"Neuron"},{"issue":"7","key":"10.1016\/j.nbd.2017.01.011_bb0815","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0103112","article-title":"Sensory neurons do not induce motor neuron loss in a human stem cell model of spinal muscular atrophy","volume":"9","author":"Schwab","year":"2014","journal-title":"PLoS One"},{"key":"10.1016\/j.nbd.2017.01.011_bb0820","doi-asserted-by":"crossref","DOI":"10.1212\/WNL.0000000000001269","article-title":"Novel mutations expand the clinical spectrum of DYNC1H1-associated spinal muscular atrophy","author":"Scoto","year":"2015","journal-title":"Neurology"},{"issue":"7","key":"10.1016\/j.nbd.2017.01.011_bb0825","doi-asserted-by":"crossref","first-page":"1754","DOI":"10.1093\/hmg\/ddt567","article-title":"SMN deficiency alters Nrxn2 expression and splicing in zebrafish and mouse models of spinal muscular atrophy","volume":"23","author":"See","year":"2014","journal-title":"Hum. Mol. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0830","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1016\/j.yexcr.2005.05.014","article-title":"A role for complexes of survival of motor neurons (SMN) protein with gemins and profilin in neurite-like cytoplasmic extensions of cultured nerve cells","volume":"309","author":"Sharma","year":"2005","journal-title":"Exp. Cell Res."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0835","doi-asserted-by":"crossref","first-page":"973","DOI":"10.1093\/hmg\/ddp562","article-title":"Ciliary neurotrophic factor-induced sprouting preserves motor function in a mouse model of mild spinal muscular atrophy","volume":"19","author":"Simon","year":"2010","journal-title":"Hum. Mol. Genet."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0840","doi-asserted-by":"crossref","first-page":"1333","DOI":"10.1128\/MCB.26.4.1333-1346.2006","article-title":"Splicing of a critical exon of human survival motor neuron is regulated by a unique silencer element located in the last intron","volume":"26","author":"Singh","year":"2006","journal-title":"Mol. Cell. Biol."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0845","doi-asserted-by":"crossref","first-page":"341","DOI":"10.4161\/rna.6.3.8723","article-title":"A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy","volume":"6","author":"Singh","year":"2009","journal-title":"RNA Biol."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0850","doi-asserted-by":"crossref","first-page":"855","DOI":"10.1093\/hmg\/ddt477","article-title":"Chondrolectin affects cell survival and neuronal outgrowth in in vitro and in vivo models of spinal muscular atrophy","volume":"23","author":"Sleigh","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.nbd.2017.01.011_bb0855","doi-asserted-by":"crossref","DOI":"10.1038\/nsmb.3167","article-title":"A U1 snRNP-specific assembly pathway reveals the SMN complex as a versatile hub for RNP exchange","author":"So","year":"2016","journal-title":"Nat. Struct. Mol. Biol."},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0860","doi-asserted-by":"crossref","first-page":"1701","DOI":"10.1016\/S0002-9440(10)63426-8","article-title":"Dysregulation of stathmin, a microtubule-destabilizing protein, and up-regulation of Hsp25, Hsp27, and the antioxidant peroxiredoxin 6 in a mouse model of familial amyotrophic lateral sclerosis","volume":"165","author":"Strey","year":"2004","journal-title":"Am. J. Pathol."},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0865","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1002\/ana.10743","article-title":"Valproic acid increases SMN levels in spinal muscular atrophy patient cells","volume":"54","author":"Sumner","year":"2003","journal-title":"Ann. Neurol."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0870","doi-asserted-by":"crossref","first-page":"441","DOI":"10.1097\/00005072-198007000-00004","article-title":"Spinal autonomic neurons in Werdnig-Hoffmann disease, mannosidosis, and Hurler's syndrome: distribution of autonomic neurons in the sacral spinal cord","volume":"39","author":"Sung","year":"1980","journal-title":"J. Neuropathol. Exp. Neurol."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0875","doi-asserted-by":"crossref","first-page":"599","DOI":"10.1038\/nmeth777","article-title":"A microfluidic culture platform for CNS axonal injury, regeneration and transport","volume":"2","author":"Taylor","year":"2005","journal-title":"Nat. Methods"},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0880","doi-asserted-by":"crossref","first-page":"702","DOI":"10.1152\/jn.00601.2012","article-title":"Preservation of VGLUT1 synapses on ventral calbindin-immunoreactive interneurons and normal locomotor function in a mouse model of spinal muscular atrophy","volume":"109","author":"Thirumalai","year":"2013","journal-title":"J. Neurophysiol."},{"issue":"5909","key":"10.1016\/j.nbd.2017.01.011_bb0885","doi-asserted-by":"crossref","first-page":"614","DOI":"10.1038\/302614a0","article-title":"Synapse elimination in neonatal rat muscle is sensitive to pattern of muscle use","volume":"302","author":"Thompson","year":"1983","journal-title":"Nature"},{"issue":"12","key":"10.1016\/j.nbd.2017.01.011_bb0890","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0052605","article-title":"Morphological characteristics of motor neurons do not determine their relative susceptibility to degeneration in a mouse model of severe spinal muscular atrophy","volume":"7","author":"Thomson","year":"2012","journal-title":"PLoS One"},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb0895","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1093\/hmg\/ddl482","article-title":"Stat5 constitutive activation rescues defects in spinal muscular atrophy","volume":"16","author":"Ting","year":"2007","journal-title":"Hum. Mol. Genet."},{"issue":"12","key":"10.1016\/j.nbd.2017.01.011_bb0900","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0051826","article-title":"The spinal muscular atrophy disease protein SMN is linked to the Golgi network","volume":"7","author":"Ting","year":"2012","journal-title":"PLoS One"},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0905","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1093\/hmg\/dds480","article-title":"COPI transport complexes bind to specific RNAs in neuronal cells","volume":"22","author":"Todd","year":"2013","journal-title":"Hum. Mol. Genet."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0910","doi-asserted-by":"crossref","first-page":"361","DOI":"10.1016\/j.nbd.2008.05.014","article-title":"Restoring Bcl-x L levels benefits a mouse model of spinal muscular atrophy","volume":"31","author":"Tsai","year":"2008","journal-title":"Neurobiol. Dis."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0915","doi-asserted-by":"crossref","first-page":"272","DOI":"10.1016\/j.nbd.2011.06.021","article-title":"IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice","volume":"45","author":"Tsai","year":"2012","journal-title":"Neurobiol. Dis."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb0920","doi-asserted-by":"crossref","first-page":"1450","DOI":"10.1038\/mt.2014.84","article-title":"Systemic administration of a recombinant AAV1 vector encoding IGF-1 improves disease manifestations in SMA mice","volume":"22","author":"Tsai","year":"2014","journal-title":"Mol. Ther."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0925","doi-asserted-by":"crossref","first-page":"293","DOI":"10.1083\/jcb.200104083","article-title":"Residual Cajal bodies in coilin knockout mice fail to recruit Sm snRNPs and SMN, the spinal muscular atrophy gene product","volume":"154","author":"Tucker","year":"2001","journal-title":"J. Cell Biol."},{"issue":"3","key":"10.1016\/j.nbd.2017.01.011_bb0930","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1016\/j.nbd.2009.03.005","article-title":"Survival motor neuron deficiency enhances progression in an amyotrophic lateral sclerosis mouse model","volume":"34","author":"Turner","year":"2009","journal-title":"Neurobiol. Dis."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb0935","doi-asserted-by":"crossref","first-page":"102","DOI":"10.1016\/j.mcn.2009.06.002","article-title":"Axonal mRNAs: characterisation and role in the growth and regeneration of dorsal root ganglion axons and growth cones","volume":"42","author":"Vogelaar","year":"2009","journal-title":"Mol. Cell. Neurosci."},{"issue":"45","key":"10.1016\/j.nbd.2017.01.011_bb0940","doi-asserted-by":"crossref","first-page":"11418","DOI":"10.1523\/JNEUROSCI.2352-16.2016","article-title":"Dysregulation of mRNA localization and translation in genetic disease","volume":"36","author":"Wang","year":"2016","journal-title":"J. Neurosci."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0945","doi-asserted-by":"crossref","first-page":"539","DOI":"10.1007\/s00401-012-0969-5","article-title":"Pleiotropic effects of miR-183~96~182 converge to regulate cell survival, proliferation and migration in medulloblastoma","volume":"123","author":"Weeraratne","year":"2012","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.nbd.2017.01.011_bb0950","first-page":"ddq058","article-title":"Stathmin, a microtubule-destabilizing protein, is dysregulated in spinal muscular atrophy","author":"Wen","year":"2010","journal-title":"Hum. Mol. Genet."},{"issue":"1","key":"10.1016\/j.nbd.2017.01.011_bb0955","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1006\/geno.2002.6806","article-title":"Molecular cloning and characterization of human chondrolectin, a novel type I transmembrane protein homologous to C-type lectins","volume":"80","author":"Weng","year":"2002","journal-title":"Genomics"},{"key":"10.1016\/j.nbd.2017.01.011_bb0960","first-page":"ddw084","article-title":"Cell type-specific miR-431 dysregulation in a motor neuron model of spinal muscular atrophy","author":"Wertz","year":"2016","journal-title":"Hum. Mol. Genet."},{"issue":"7","key":"10.1016\/j.nbd.2017.01.011_bb0965","doi-asserted-by":"crossref","first-page":"a003707","DOI":"10.1101\/cshperspect.a003707","article-title":"Spliceosome structure and function","volume":"3","author":"Will","year":"2011","journal-title":"Cold Spring Harb. Perspect. Biol."},{"issue":"24","key":"10.1016\/j.nbd.2017.01.011_bb0970","doi-asserted-by":"crossref","first-page":"7633","DOI":"10.1523\/JNEUROSCI.0950-09.2009","article-title":"Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy","volume":"29","author":"Williams","year":"2009","journal-title":"J. Neurosci."},{"issue":"6","key":"10.1016\/j.nbd.2017.01.011_bb0975","doi-asserted-by":"crossref","first-page":"965","DOI":"10.1083\/jcb.200703209","article-title":"Extracellular stimuli specifically regulate localized levels of individual neuronal mRNAs","volume":"178","author":"Willis","year":"2007","journal-title":"J. Cell Biol."},{"issue":"19","key":"10.1016\/j.nbd.2017.01.011_bb0980","doi-asserted-by":"crossref","first-page":"2320","DOI":"10.1101\/gad.342005","article-title":"Reduced U snRNP assembly causes motor axon degeneration in an animal model for spinal muscular atrophy","volume":"19","author":"Winkler","year":"2005","journal-title":"Genes Dev."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0985","doi-asserted-by":"crossref","first-page":"0","DOI":"10.1172\/JCI71318","article-title":"Dysregulation of ubiquitin homeostasis and \u03b2-catenin signaling promote spinal muscular atrophy","volume":"124","author":"Wishart","year":"2014","journal-title":"J. Clin. Invest."},{"issue":"12","key":"10.1016\/j.nbd.2017.01.011_bb0990","doi-asserted-by":"crossref","first-page":"2215","DOI":"10.1093\/hmg\/ddp157","article-title":"A SMN missense mutation complements SMN2 restoring snRNPs and rescuing SMA mice","volume":"18","author":"Workman","year":"2009","journal-title":"Hum. Mol. Genet."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb0995","doi-asserted-by":"crossref","first-page":"799","DOI":"10.1016\/j.celrep.2012.08.025","article-title":"FUS-SMN protein interactions link the motor neuron diseases ALS and SMA","volume":"2","author":"Yamazaki","year":"2012","journal-title":"Cell Rep."},{"issue":"2","key":"10.1016\/j.nbd.2017.01.011_bb1000","doi-asserted-by":"crossref","DOI":"10.1038\/cddis.2014.55","article-title":"PTEN regulates AMPA receptor-mediated cell viability in iPS-derived motor neurons","volume":"5","author":"Yang","year":"2014","journal-title":"Cell Death Dis."},{"issue":"5","key":"10.1016\/j.nbd.2017.01.011_bb1005","doi-asserted-by":"crossref","first-page":"577","DOI":"10.1093\/hmg\/11.5.577","article-title":"SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2\u03b21","volume":"11","author":"Young","year":"2002","journal-title":"Hum. Mol. Genet."},{"issue":"16","key":"10.1016\/j.nbd.2017.01.011_bb1010","doi-asserted-by":"crossref","first-page":"6627","DOI":"10.1523\/JNEUROSCI.23-16-06627.2003","article-title":"Active transport of the survival motor neuron protein and the role of exon-7 in cytoplasmic localization","volume":"23","author":"Zhang","year":"2003","journal-title":"J. Neurosci."},{"issue":"17","key":"10.1016\/j.nbd.2017.01.011_bb1015","doi-asserted-by":"crossref","first-page":"4330","DOI":"10.1523\/JNEUROSCI.0497-05.2005","article-title":"Extracellular domains of \u03b1-neurexins participate in regulating synaptic transmission by selectively affecting N-and P\/Q-type Ca2+ channels","volume":"25","author":"Zhang","year":"2005","journal-title":"J. Neurosci."},{"issue":"33","key":"10.1016\/j.nbd.2017.01.011_bb1020","doi-asserted-by":"crossref","first-page":"8622","DOI":"10.1523\/JNEUROSCI.3967-05.2006","article-title":"Multiprotein complexes of the survival of motor neuron protein SMN with Gemins traffic to neuronal processes and growth cones of motor neurons","volume":"26","author":"Zhang","year":"2006","journal-title":"J. Neurosci."},{"issue":"12","key":"10.1016\/j.nbd.2017.01.011_bb1025","doi-asserted-by":"crossref","first-page":"2657","DOI":"10.1002\/jnr.21308","article-title":"QNQKE targeting motif for the SMN-Gemin multiprotein complexin neurons","volume":"85","author":"Zhang","year":"2007","journal-title":"J. Neurosci. Res."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb1030","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1016\/j.cell.2008.03.031","article-title":"SMN deficiency causes tissue-specific perturbations in the repertoire of snRNAs and widespread defects in splicing","volume":"133","author":"Zhang","year":"2008","journal-title":"Cell"},{"issue":"48","key":"10.1016\/j.nbd.2017.01.011_bb1035","doi-asserted-by":"crossref","first-page":"19348","DOI":"10.1073\/pnas.1319280110","article-title":"Dysregulation of synaptogenesis genes antecedes motor neuron pathology in spinal muscular atrophy","volume":"110","author":"Zhang","year":"2013","journal-title":"Proc. Natl. Acad. Sci."},{"issue":"16","key":"10.1016\/j.nbd.2017.01.011_bb1040","doi-asserted-by":"crossref","first-page":"6885","DOI":"10.1523\/JNEUROSCI.5180-12.2013","article-title":"The microRNA-17\u201392 cluster enhances axonal outgrowth in embryonic cortical neurons","volume":"33","author":"Zhang","year":"2013","journal-title":"J. Neurosci."},{"issue":"13","key":"10.1016\/j.nbd.2017.01.011_bb1045","doi-asserted-by":"crossref","first-page":"4426","DOI":"10.1523\/JNEUROSCI.5179-11.2012","article-title":"Chondrolectin mediates growth cone interactions of motor axons with an intermediate target","volume":"32","author":"Zhong","year":"2012","journal-title":"J. Neurosci."},{"issue":"8","key":"10.1016\/j.nbd.2017.01.011_bb1050","doi-asserted-by":"crossref","first-page":"2543","DOI":"10.1523\/JNEUROSCI.3534-15.2016","article-title":"Defects in motoneuron\u2013astrocyte interactions in spinal muscular atrophy","volume":"36","author":"Zhou","year":"2016","journal-title":"J. Neurosci."},{"issue":"17","key":"10.1016\/j.nbd.2017.01.011_bb1055","doi-asserted-by":"crossref","first-page":"7308","DOI":"10.1523\/JNEUROSCI.0224-13.2013","article-title":"Skeletal muscle calpain acts through nitric oxide and neural miRNAs to regulate acetylcholine release in motor nerve terminals","volume":"33","author":"Zhu","year":"2013","journal-title":"J. Neurosci."},{"issue":"4","key":"10.1016\/j.nbd.2017.01.011_bb1060","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1007\/s10571-011-9647-8","article-title":"SMN deficiency reduces cellular ability to form stress granules, sensitizing cells to stress","volume":"31","author":"Zou","year":"2011","journal-title":"Cell. Mol. Neurobiol."}],"container-title":["Neurobiology of Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0969996117300293?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0969996117300293?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2018,9,4]],"date-time":"2018-09-04T17:26:36Z","timestamp":1536081996000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0969996117300293"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,6]]},"references-count":212,"alternative-id":["S0969996117300293"],"URL":"https:\/\/doi.org\/10.1016\/j.nbd.2017.01.011","relation":{},"ISSN":["0969-9961"],"issn-type":[{"value":"0969-9961","type":"print"}],"subject":[],"published":{"date-parts":[[2017,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Spinal muscular atrophy: Factors that modulate motor neurone vulnerability","name":"articletitle","label":"Article Title"},{"value":"Neurobiology of Disease","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.nbd.2017.01.011","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2017 Elsevier Inc. All rights reserved.","name":"copyright","label":"Copyright"}]}}