{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T14:18:20Z","timestamp":1791296300720,"version":"4.1.0"},"reference-count":76,"publisher":"Oxford University Press (OUP)","issue":"7","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2013,4,1]]},"DOI":"10.1093\/hmg\/dds540","type":"journal-article","created":{"date-parts":[[2012,12,22]],"date-time":"2012-12-22T20:45:20Z","timestamp":1356209120000},"page":"1328-1347","source":"Crossref","is-referenced-by-count":121,"title":["Plastin 3 ameliorates spinal muscular atrophy via delayed axon pruning and improves neuromuscular junction functionality"],"prefix":"10.1093","volume":"22","author":[{"given":"Bastian","family":"Ackermann","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Kr\u00f6ber","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Torres-Benito","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anke","family":"Borgmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miriam","family":"Peters","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seyyed Mohsen","family":"Hosseini Barkooie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rocio","family":"Tejero","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miriam","family":"Jakubik","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julia","family":"Schreml","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janine","family":"Milbradt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas F.","family":"Wunderlich","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Markus","family":"Riessland","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucia","family":"Tabares","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brunhilde","family":"Wirth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2012,12,20]]},"reference":[{"key":"key\n\t\t\t\t20170522135657_DDS540C1","doi-asserted-by":"crossref","first-page":"539","DOI":"10.1523\/JNEUROSCI.23-02-00539.2003","article-title":"Aberrant patterning of neuromuscular synapses in choline acetyltransferase-deficient mice","volume":"23","author":"Brandon","year":"2003","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170522135657_DDS540C2","doi-asserted-by":"crossref","first-page":"909","DOI":"10.1016\/0092-8674(91)90364-5","article-title":"Agrin mediates cell contact-induced acetylcholine receptor clustering","volume":"67","author":"Campanelli","year":"1991","journal-title":"Cell"},{"key":"key\n\t\t\t\t20170522135657_DDS540C3","doi-asserted-by":"crossref","first-page":"701","DOI":"10.1113\/jphysiol.1970.sp009187","article-title":"Neuromuscular transmission in new-born rats","volume":"209","author":"Redfern","year":"1970","journal-title":"J. Physiol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C4","doi-asserted-by":"crossref","first-page":"1439","DOI":"10.1093\/hmg\/11.12.1439","article-title":"Neurofilament accumulation at the motor endplate and lack of axonal sprouting in a spinal muscular atrophy mouse model","volume":"11","author":"Cifuentes-Diaz","year":"2002","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C5","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1016\/j.nmd.2004.04.004","article-title":"Concerns about the design of clinical trials for spinal muscular atrophy","volume":"14","author":"Crawford","year":"2004","journal-title":"Neuromuscul. Disord."},{"key":"key\n\t\t\t\t20170522135657_DDS540C6","doi-asserted-by":"crossref","first-page":"704","DOI":"10.1002\/ana.20473","article-title":"Natural history of denervation in SMA: relation to age, SMN2 copy number, and function","volume":"57","author":"Swoboda","year":"2005","journal-title":"Ann. Neurol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C7","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C8","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C9","doi-asserted-by":"crossref","first-page":"801","DOI":"10.1083\/jcb.200304128","article-title":"Smn, the spinal muscular atrophy-determining gene product, modulates axon growth and localization of beta-actin mRNA in growth cones of motoneurons","volume":"163","author":"Rossoll","year":"2003","journal-title":"J. Cell Biol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C10","doi-asserted-by":"crossref","first-page":"2839","DOI":"10.1002\/jnr.21743","article-title":"Neurodevelopmental abnormalities in neurosphere-derived neural stem cells from SMN-depleted mice","volume":"86","author":"Shafey","year":"2008","journal-title":"J. Neurosci. Res."},{"key":"key\n\t\t\t\t20170522135657_DDS540C11","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C12","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1523\/JNEUROSCI.4434-08.2009","article-title":"Impaired synaptic vesicle release and immaturity of neuromuscular junctions in spinal muscular atrophy mice","volume":"29","author":"Kong","year":"2009","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170522135657_DDS540C13","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1523\/JNEUROSCI.4496-09.2010","article-title":"Altered intracellular Ca2+ homeostasis in nerve terminals of severe spinal muscular atrophy mice","volume":"30","author":"Ruiz","year":"2010","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170522135657_DDS540C14","doi-asserted-by":"crossref","first-page":"e26164","DOI":"10.1371\/journal.pone.0026164","article-title":"SMN requirement for synaptic vesicle, active zone and microtubule postnatal organization in motor nerve terminals","volume":"6","author":"Torres-Benito","year":"2011","journal-title":"PLoS One"},{"key":"key\n\t\t\t\t20170522135657_DDS540C15","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"},{"key":"key\n\t\t\t\t20170522135657_DDS540C16","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1086\/338627","article-title":"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","volume":"70","author":"Feldkotter","year":"2002","journal-title":"Am. J. Hum. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C17","doi-asserted-by":"crossref","first-page":"1023","DOI":"10.1016\/S0092-8674(00)80368-2","article-title":"The SMN-SIP1 complex has an essential role in spliceosomal snRNP biogenesis","volume":"90","author":"Fischer","year":"1997","journal-title":"Cell"},{"key":"key\n\t\t\t\t20170522135657_DDS540C18","doi-asserted-by":"crossref","first-page":"615","DOI":"10.1016\/S0092-8674(00)81632-3","article-title":"A novel function for SMN, the spinal muscular atrophy disease gene product, in pre-mRNA splicing","volume":"95","author":"Pellizzoni","year":"1998","journal-title":"Cell"},{"key":"key\n\t\t\t\t20170522135657_DDS540C19","doi-asserted-by":"crossref","first-page":"720","DOI":"10.1101\/gad.974702","article-title":"miRNPs: a novel class of ribonucleoproteins containing numerous microRNAs","volume":"16","author":"Mourelatos","year":"2002","journal-title":"Genes Dev."},{"key":"key\n\t\t\t\t20170522135657_DDS540C20","doi-asserted-by":"crossref","first-page":"244","DOI":"10.1016\/j.molcel.2008.06.004","article-title":"Inactivation of the SMN complex by oxidative stress","volume":"31","author":"Wan","year":"2008","journal-title":"Mol. Cell"},{"key":"key\n\t\t\t\t20170522135657_DDS540C21","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"},{"key":"key\n\t\t\t\t20170522135657_DDS540C22","doi-asserted-by":"crossref","first-page":"5074","DOI":"10.1523\/JNEUROSCI.5808-11.2012","article-title":"Survival motor neuron affects plastin 3 protein levels leading to motor defects","volume":"32","author":"Hao le","year":"2012","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170522135657_DDS540C23","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1242\/dmm.007245","article-title":"The contribution of mouse models to understanding the pathogenesis of spinal muscular atrophy","volume":"4","author":"Sleigh","year":"2011","journal-title":"Dis. Model. Mech."},{"key":"key\n\t\t\t\t20170522135657_DDS540C24","doi-asserted-by":"crossref","first-page":"3479","DOI":"10.1152\/jn.00789.2011","article-title":"Dual pools of actin at presynaptic terminals","volume":"107","author":"Bleckert","year":"2012","journal-title":"J. Neurophysiol"},{"key":"key\n\t\t\t\t20170522135657_DDS540C25","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":"Nolle","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C26","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1186\/1741-7015-10-24","article-title":"Fasudil improves survival and promotes skeletal muscle development in a mouse model of spinal muscular atrophy","volume":"10","author":"Bowerman","year":"2012","journal-title":"BMC Med."},{"key":"key\n\t\t\t\t20170522135657_DDS540C27","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C28","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1016\/j.cmet.2008.01.006","article-title":"PDK1 deficiency in POMC-expressing cells reveals FOXO1-dependent and -independent pathways in control of energy homeostasis and stress response","volume":"7","author":"Belgardt","year":"2008","journal-title":"Cell Metab."},{"key":"key\n\t\t\t\t20170522135657_DDS540C29","doi-asserted-by":"crossref","first-page":"5080","DOI":"10.1093\/nar\/23.24.5080","article-title":"A cre-transgenic mouse strain for the ubiquitous deletion of loxP-flanked gene segments including deletion in germ cells","volume":"23","author":"Schwenk","year":"1995","journal-title":"Nucleic Acids Res."},{"key":"key\n\t\t\t\t20170522135657_DDS540C30","doi-asserted-by":"crossref","first-page":"1995","DOI":"10.1083\/jcb.127.6.1995","article-title":"Functional differences between L- and T-plastin isoforms","volume":"127","author":"Arpin","year":"1994","journal-title":"J. Cell Biol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C31","doi-asserted-by":"crossref","first-page":"66","DOI":"10.1038\/71709","article-title":"A mouse model for spinal muscular atrophy","volume":"24","author":"Hsieh-Li","year":"2000","journal-title":"Nat. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C32","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1016\/j.expneurol.2008.02.025","article-title":"Identification of a battery of tests for drug candidate evaluation in the SMNDelta7 neonate model of spinal muscular atrophy","volume":"212","author":"El-Khodor","year":"2008","journal-title":"Exp. Neurol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C33","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C34","doi-asserted-by":"crossref","first-page":"389","DOI":"10.1146\/annurev.neuro.22.1.389","article-title":"Development of the vertebrate neuromuscular junction","volume":"22","author":"Sanes","year":"1999","journal-title":"Annu. Rev. Neurosci."},{"key":"key\n\t\t\t\t20170522135657_DDS540C35","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/S0896-6273(00)80893-4","article-title":"Synapse elimination and indelible memory","volume":"25","author":"Lichtman","year":"2000","journal-title":"Neuron"},{"key":"key\n\t\t\t\t20170522135657_DDS540C36","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C37","doi-asserted-by":"crossref","first-page":"3399","DOI":"10.1093\/hmg\/ddn234","article-title":"SMN complex localizes to the sarcomeric Z-disc and is a proteolytic target of calpain","volume":"17","author":"Walker","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C38","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"},{"key":"key\n\t\t\t\t20170522135657_DDS540C39","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1016\/S0896-6273(01)80026-X","article-title":"Requirement for the homeobox gene Hb9 in the consolidation of motor neuron identity","volume":"23","author":"Arber","year":"1999","journal-title":"Neuron"},{"key":"key\n\t\t\t\t20170522135657_DDS540C40","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1016\/S0896-6273(01)00287-2","article-title":"Patterning of muscle acetylcholine receptor gene expression in the absence of motor innervation","volume":"30","author":"Yang","year":"2001","journal-title":"Neuron"},{"key":"key\n\t\t\t\t20170522135657_DDS540C41","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C42","doi-asserted-by":"crossref","first-page":"946","DOI":"10.1002\/mus.880170816","article-title":"Intestinal pseudo-obstruction in adult spinal muscular atrophy","volume":"17","author":"Ionasescu","year":"1994","journal-title":"Muscle Nerve"},{"key":"key\n\t\t\t\t20170522135657_DDS540C43","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1097\/CND.0b013e318183e0fa","article-title":"Low bone mineral density in spinal muscular atrophy","volume":"10","author":"Khatri","year":"2008","journal-title":"J. Clin. Neuromuscul. Dis."},{"key":"key\n\t\t\t\t20170522135657_DDS540C44","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1136\/jmg.2008.057950","article-title":"Congenital heart disease is a feature of severe infantile spinal muscular atrophy","volume":"45","author":"Rudnik-Schoneborn","year":"2008","journal-title":"J. Med. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C45","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1016\/j.nmd.2011.10.021","article-title":"Density, calibre and ramification of muscle capillaries are altered in a mouse model of severe spinal muscular atrophy","volume":"22","author":"Somers","year":"2012","journal-title":"Neuromuscul. Disord."},{"key":"key\n\t\t\t\t20170522135657_DDS540C46","doi-asserted-by":"crossref","first-page":"1731","DOI":"10.1016\/S0002-9440(10)63428-1","article-title":"Deletion of murine Smn exon 7 directed to liver leads to severe defect of liver development associated with iron overload","volume":"165","author":"Vitte","year":"2004","journal-title":"Am. J. Pathol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C47","doi-asserted-by":"crossref","first-page":"4216","DOI":"10.1093\/hmg\/ddq340","article-title":"SMN deficiency disrupts brain development in a mouse model of severe spinal muscular atrophy","volume":"19","author":"Wishart","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C48","article-title":"Severe SMA mice show organ impairment that cannot be rescued by therapy with the HDACi JNJ-26481585","author":"Schreml","year":"2012","journal-title":"Eur. J. Hum. Genet"},{"key":"key\n\t\t\t\t20170522135657_DDS540C49","doi-asserted-by":"crossref","first-page":"3906","DOI":"10.1093\/hmg\/ddq330","article-title":"Arrhythmia and cardiac defects are a feature of spinal muscular atrophy model mice","volume":"19","author":"Heier","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C50","doi-asserted-by":"crossref","first-page":"3895","DOI":"10.1093\/hmg\/ddq300","article-title":"Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery","volume":"19","author":"Bevan","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C51","doi-asserted-by":"crossref","first-page":"4059","DOI":"10.1093\/hmg\/ddq329","article-title":"Cardiac defects contribute to the pathology of spinal muscular atrophy models","volume":"19","author":"Shababi","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C52","doi-asserted-by":"crossref","first-page":"14250","DOI":"10.1523\/JNEUROSCI.1835-11.2011","article-title":"Piccolo regulates the dynamic assembly of presynaptic F-actin","volume":"31","author":"Waites","year":"2011","journal-title":"J. Neurosci."},{"key":"key\n\t\t\t\t20170522135657_DDS540C53","doi-asserted-by":"crossref","first-page":"780","DOI":"10.1126\/science.8430330","article-title":"Synaptic vesicle phosphoproteins and regulation of synaptic function","volume":"259","author":"Greengard","year":"1993","journal-title":"Science"},{"key":"key\n\t\t\t\t20170522135657_DDS540C54","doi-asserted-by":"crossref","first-page":"539","DOI":"10.1016\/S0896-6273(00)00064-7","article-title":"Actin-dependent regulation of neurotransmitter release at central synapses","volume":"27","author":"Morales","year":"2000","journal-title":"Neuron"},{"key":"key\n\t\t\t\t20170522135657_DDS540C55","doi-asserted-by":"crossref","first-page":"344","DOI":"10.1038\/nrn2373","article-title":"Actin in action: the interplay between the actin cytoskeleton and synaptic efficacy","volume":"9","author":"Cingolani","year":"2008","journal-title":"Nat. Rev. Neurosci."},{"key":"key\n\t\t\t\t20170522135657_DDS540C56","doi-asserted-by":"crossref","first-page":"845","DOI":"10.1093\/hmg\/ddi078","article-title":"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","volume":"14","author":"Le","year":"2005","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C57","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1002\/dneu.20912","article-title":"Synaptic defects in spinal muscular atrophy animal models","volume":"72","author":"Torres-Benito","year":"2012","journal-title":"Dev. Neurobiol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C58","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C59","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1038\/nbt.1610","article-title":"Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN","volume":"28","author":"Foust","year":"2010","journal-title":"Nat. Biotechnol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C60","doi-asserted-by":"crossref","first-page":"681","DOI":"10.1093\/hmg\/ddq514","article-title":"Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice","volume":"20","author":"Dominguez","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"key\n\t\t\t\t20170522135657_DDS540C61","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":"key\n\t\t\t\t20170522135657_DDS540C62","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C63","doi-asserted-by":"crossref","first-page":"3029","DOI":"10.1172\/JCI57291","article-title":"Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy","volume":"121","author":"Lutz","year":"2011","journal-title":"J. Clin. Invest."},{"key":"key\n\t\t\t\t20170522135657_DDS540C64","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1146\/annurev.neuro.28.061604.135757","article-title":"The actin cytoskeleton: integrating form and function at the synapse","volume":"28","author":"Dillon","year":"2005","journal-title":"Annu. Rev. Neurosci."},{"key":"key\n\t\t\t\t20170522135657_DDS540C65","doi-asserted-by":"crossref","first-page":"1321","DOI":"10.1083\/jcb.150.6.1321","article-title":"The actin-driven movement and formation of acetylcholine receptor clusters","volume":"150","author":"Dai","year":"2000","journal-title":"J. Cell Biol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C66","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1038\/377232a0","article-title":"Failure of postsynaptic specialization to develop at neuromuscular junctions of rapsyn-deficient mice","volume":"377","author":"Gautam","year":"1995","journal-title":"Nature"},{"key":"key\n\t\t\t\t20170522135657_DDS540C67","doi-asserted-by":"crossref","first-page":"e8478","DOI":"10.1371\/journal.pone.0008478","article-title":"The function of cortactin in the clustering of acetylcholine receptors at the vertebrate neuromuscular junction","volume":"4","author":"Madhavan","year":"2009","journal-title":"PLoS One"},{"key":"key\n\t\t\t\t20170522135657_DDS540C68","doi-asserted-by":"crossref","first-page":"213492","DOI":"10.1155\/2012\/213492","article-title":"Plastin family of actin-bundling proteins: its functions in leukocytes, neurons, intestines, and cancer","volume":"2012","author":"Shinomiya","year":"2012","journal-title":"Int. J. Cell Biol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C69","first-page":"175","article-title":"Cre\/loxP recombination system and gene targeting","volume":"180","author":"Kuhn","year":"2002","journal-title":"Methods Mol. Biol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C70","doi-asserted-by":"crossref","first-page":"4489","DOI":"10.1073\/pnas.032068699","article-title":"Ability of the hydrophobic FGF and basic TAT peptides to promote cellular uptake of recombinant Cre recombinase: a tool for efficient genetic engineering of mammalian genomes","volume":"99","author":"Peitz","year":"2002","journal-title":"Proc. Natl Acad. Sci. USA"},{"key":"key\n\t\t\t\t20170522135657_DDS540C71","doi-asserted-by":"crossref","first-page":"4293","DOI":"10.1093\/nar\/19.15.4293","article-title":"Simplified mammalian DNA isolation procedure","volume":"19","author":"Laird","year":"1991","journal-title":"Nucleic Acids Res."},{"key":"key\n\t\t\t\t20170522135657_DDS540C72","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1016\/S0022-2836(75)80083-0","article-title":"Detection of specific sequences among DNA fragments separated by gel electrophoresis","volume":"98","author":"Southern","year":"1975","journal-title":"J. Mol. Biol."},{"key":"key\n\t\t\t\t20170522135657_DDS540C73","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."},{"key":"key\n\t\t\t\t20170522135657_DDS540C74","doi-asserted-by":"crossref","first-page":"744","DOI":"10.1016\/j.nmd.2010.06.010","article-title":"Ultrastructural changes in diaphragm neuromuscular junctions in a severe mouse model for spinal muscular atrophy and their prevention by bifunctional U7 snRNA correcting SMN2 splicing","volume":"20","author":"Voigt","year":"2010","journal-title":"Neuromuscul. Disord."},{"key":"key\n\t\t\t\t20170522135657_DDS540C75","first-page":"63","article-title":"Beitr\u00e4ge zur Anatomie der Muskulatur und der peripheren Nerven von Carausius (Dixippus) morosus BR","volume":"66","author":"Marquardt","year":"1940","journal-title":"Zool. Jahrb\u00fccher. Abt. Anat."},{"key":"key\n\t\t\t\t20170522135657_DDS540C76","doi-asserted-by":"crossref","first-page":"114","DOI":"10.1113\/jphysiol.1955.sp005397","article-title":"A further study of the statistical composition on the end-plate potential","volume":"130","author":"Martin","year":"1955","journal-title":"J. Physiol."}],"container-title":["Human Molecular Genetics"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/academic.oup.com\/hmg\/article-pdf\/22\/7\/1328\/13928847\/dds540.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,7,7]],"date-time":"2019-07-07T10:54:27Z","timestamp":1562496867000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/hmg\/article-lookup\/doi\/10.1093\/hmg\/dds540"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2012,12,20]]},"references-count":76,"journal-issue":{"issue":"7","published-online":{"date-parts":[[2012,12,20]]},"published-print":{"date-parts":[[2013,4,1]]}},"URL":"https:\/\/doi.org\/10.1093\/hmg\/dds540","relation":{},"ISSN":["1460-2083","0964-6906"],"issn-type":[{"value":"1460-2083","type":"electronic"},{"value":"0964-6906","type":"print"}],"subject":[],"published":{"date-parts":[[2012,12,20]]}}}