{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,8,7]],"date-time":"2025-08-07T20:49:52Z","timestamp":1754599792470},"reference-count":41,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2019,2,7]],"date-time":"2019-02-07T00:00:00Z","timestamp":1549497600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2019,2,7]],"date-time":"2019-02-07T00:00:00Z","timestamp":1549497600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100000065","name":"U.S. Department of Health &amp; Human Services | NIH | National Institute of Neurological Disorders and Stroke","doi-asserted-by":"publisher","award":["R01HD064850","R21NS064349","R21HD057402","R01HD064850","R21NS064349","R21HD057402"],"award-info":[{"award-number":["R01HD064850","R21NS064349","R21HD057402","R01HD064850","R21NS064349","R21HD057402"]}],"id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"publisher"}]},{"name":"CureSMA, FightSMA, Gwendolyn Strong Foundation"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Sci Rep"],"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder that causes progressive muscle weakness and is the leading genetic cause of infant mortality worldwide. SMA is caused by the loss of <jats:italic>survival motor neuron 1<\/jats:italic> (<jats:italic>SMN1<\/jats:italic>). In humans, a nearly identical copy gene is present, called <jats:italic>SMN2<\/jats:italic>. Although <jats:italic>SMN2<\/jats:italic> maintains the same coding sequence, this gene cannot compensate for the loss of <jats:italic>SMN1<\/jats:italic> because of a single silent nucleotide difference in <jats:italic>SMN2<\/jats:italic> exon 7. <jats:italic>SMN2<\/jats:italic> primarily produces an alternatively spliced isoform lacking exon 7, which is critical for protein function. <jats:italic>SMN2<\/jats:italic> is an important disease modifier that makes for an excellent target for therapeutic intervention because all SMA patients retain <jats:italic>SMN2<\/jats:italic>. Therefore, compounds and small molecules that can increase <jats:italic>SMN2<\/jats:italic> exon 7 inclusion, transcription and SMN protein stability have great potential for SMA therapeutics. Previously, we performed a high throughput screen and established a class of compounds that increase SMN protein in various cellular contexts. In this study, a novel compound was identified that increased SMN protein levels <jats:italic>in vivo<\/jats:italic> and ameliorated the disease phenotype in severe and intermediate mouse models of SMA.<\/jats:p>","DOI":"10.1038\/s41598-018-38208-9","type":"journal-article","created":{"date-parts":[[2019,2,7]],"date-time":"2019-02-07T11:05:41Z","timestamp":1549537541000},"update-policy":"http:\/\/dx.doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Intraperitoneal delivery of a novel drug\u2010like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy"],"prefix":"10.1038","volume":"9","author":[{"given":"E. Y.","family":"Osman","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A.","family":"Rietz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"R. A.","family":"Kline","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J. J.","family":"Cherry","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"K. J.","family":"Hodgetts","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"C. L.","family":"Lorson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"E. J.","family":"Androphy","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2019,2,7]]},"reference":[{"key":"38208_CR1","doi-asserted-by":"publisher","first-page":"6307","DOI":"10.1073\/pnas.96.11.6307","volume":"96","author":"CL Lorson","year":"1999","unstructured":"Lorson, C. L., Hahnen, E., Androphy, E. J. & Wirth, B. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci USA 96, 6307\u20136311, https:\/\/doi.org\/10.1073\/pnas.96.11.6307 (1999).","journal-title":"Proc Natl Acad Sci USA"},{"key":"38208_CR2","doi-asserted-by":"publisher","first-page":"1177","DOI":"10.1093\/hmg\/8.7.1177","volume":"8","author":"UR Monani","year":"1999","unstructured":"Monani, U. R. et al. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol Genet 8, 1177\u20131183, https:\/\/doi.org\/10.1093\/hmg\/8.7.1177 (1999).","journal-title":"Hum Mol Genet"},{"key":"38208_CR3","doi-asserted-by":"publisher","first-page":"377","DOI":"10.1038\/ng854","volume":"30","author":"L Cartegni","year":"2002","unstructured":"Cartegni, L. & Krainer, A. R. Disruption of an SF2\/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1. Nat. Genet. 30, 377\u2013384, https:\/\/doi.org\/10.1038\/ng854 (2002).","journal-title":"Nat. Genet."},{"key":"38208_CR4","doi-asserted-by":"publisher","first-page":"460","DOI":"10.1038\/ng1207","volume":"34","author":"T Kashima","year":"2003","unstructured":"Kashima, T. & Manley, J. L. A negative element in SMN2 exon 7 inhibits splicing in spinal muscular atrophy. Nat. Genet. 34, 460\u2013463, https:\/\/doi.org\/10.1038\/ng1207 (2003).","journal-title":"Nat. Genet."},{"key":"38208_CR5","doi-asserted-by":"publisher","first-page":"1333","DOI":"10.1128\/mcb.26.4.1333-1346.2006","volume":"26","author":"NK Singh","year":"2006","unstructured":"Singh, N. K., Singh, N. N., Androphy, E. J. & Singh, R. N. Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron. Mol. Cell. Biol. 26, 1333\u20131346, https:\/\/doi.org\/10.1128\/mcb.26.4.1333-1346.2006 (2006).","journal-title":"Mol. Cell. Biol."},{"key":"38208_CR6","doi-asserted-by":"publisher","first-page":"63","DOI":"10.1086\/498853","volume":"78","author":"L Cartegni","year":"2006","unstructured":"Cartegni, L., Hastings, M. L., Calarco, J. A., de Stanchina, E. & Krainer, A. R. Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 and SMN2. Am. J. Hum. Genet. 78, 63\u201377 (2006).","journal-title":"Am. J. Hum. Genet."},{"key":"38208_CR7","doi-asserted-by":"publisher","first-page":"834","DOI":"10.1016\/j.ajhg.2008.01.014","volume":"82","author":"Y Hua","year":"2008","unstructured":"Hua, Y., Vickers, T. A., Okunola, H. L., Bennett, C. F. & Krainer, A. R. Antisense masking of an hnRNP A1\/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice. Am. J. Hum. Genet. 82, 834\u2013848, https:\/\/doi.org\/10.1016\/j.ajhg.2008.01.014 (2008).","journal-title":"Am. J. Hum. Genet."},{"key":"38208_CR8","doi-asserted-by":"publisher","first-page":"123","DOI":"10.1038\/nature10485","volume":"478","author":"Y Hua","year":"2011","unstructured":"Hua, Y. et al. Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model. Nature 478, 123\u2013126, https:\/\/doi.org\/10.1038\/nature10485 (2011).","journal-title":"Nature"},{"key":"38208_CR9","doi-asserted-by":"publisher","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","volume":"377","author":"RS Finkel","year":"2017","unstructured":"Finkel, R. S. et al. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med 377, 1723\u20131732, https:\/\/doi.org\/10.1056\/NEJMoa1702752 (2017).","journal-title":"N Engl J Med"},{"key":"38208_CR10","doi-asserted-by":"publisher","first-page":"625","DOI":"10.1056\/NEJMoa1710504","volume":"378","author":"E Mercuri","year":"2018","unstructured":"Mercuri, E. et al. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med 378, 625\u2013635, https:\/\/doi.org\/10.1056\/NEJMoa1710504 (2018).","journal-title":"N Engl J Med"},{"key":"38208_CR11","doi-asserted-by":"publisher","first-page":"511","DOI":"10.1038\/nchembio.1837","volume":"11","author":"J Palacino","year":"2015","unstructured":"Palacino, J. et al. SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice. Nat. Chem. Biol. 11, 511\u2013517, https:\/\/doi.org\/10.1038\/nchembio.1837 (2015).","journal-title":"Nat. Chem. Biol."},{"key":"38208_CR12","doi-asserted-by":"publisher","first-page":"688","DOI":"10.1126\/science.1250127","volume":"345","author":"NA Naryshkin","year":"2014","unstructured":"Naryshkin, N. A. et al. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science 345, 688\u2013693, https:\/\/doi.org\/10.1126\/science.1250127 (2014).","journal-title":"SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science"},{"key":"38208_CR13","doi-asserted-by":"publisher","first-page":"10067","DOI":"10.1021\/acs.jmedchem.6b00670","volume":"59","author":"AN Calder","year":"2016","unstructured":"Calder, A. N., Androphy, E. J. & Hodgetts, K. J. Small molecules in development for the treatment of spinal muscular atrophy. J Med Chem 59, 10067\u201310083, https:\/\/doi.org\/10.1021\/acs.jmedchem.6b00670 (2016).","journal-title":"J Med Chem"},{"key":"38208_CR14","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-017-01559-4","volume":"8","author":"M Sivaramakrishnan","year":"2017","unstructured":"Sivaramakrishnan, M. et al. Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers. Nat. Commun. 8, 1476, https:\/\/doi.org\/10.1038\/s41467-017-01559-4 (2017).","journal-title":"Nat. Commun."},{"key":"38208_CR15","doi-asserted-by":"publisher","first-page":"E4604","DOI":"10.1073\/pnas.1800260115","volume":"115","author":"J Wang","year":"2018","unstructured":"Wang, J., Schultz, P. G. & Johnson, K. A. Mechanistic studies of a small-molecule modulator of SMN2 splicing. Proc. Natl. Acad. Sci. USA 115, E4604\u2013e4612, https:\/\/doi.org\/10.1073\/pnas.1800260115 (2018).","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"38208_CR16","doi-asserted-by":"publisher","first-page":"4594","DOI":"10.1021\/acs.jmedchem.6b01885","volume":"60","author":"A Rietz","year":"2017","unstructured":"Rietz, A. et al. Discovery of a Small Molecule Probe That Post-Translationally Stabilizes the Survival Motor Neuron Protein for the Treatment of Spinal Muscular Atrophy. J. Med. Chem. 60, 4594\u20134610, https:\/\/doi.org\/10.1021\/acs.jmedchem.6b01885 (2017).","journal-title":"J. Med. Chem."},{"key":"38208_CR17","unstructured":"Xiao, J. et al. In Probe Reports from the NIH Molecular Libraries Program (National Center for Biotechnology Information (US), 2010)."},{"key":"38208_CR18","doi-asserted-by":"publisher","first-page":"6215","DOI":"10.1021\/jm200497t","volume":"54","author":"J Xiao","year":"2011","unstructured":"Xiao, J. et al. Discovery, synthesis, and biological evaluation of novel SMN protein modulators. J. Med. Chem. 54, 6215\u20136233, https:\/\/doi.org\/10.1021\/jm200497t (2011).","journal-title":"J. Med. Chem."},{"key":"38208_CR19","doi-asserted-by":"publisher","first-page":"481","DOI":"10.1177\/1087057111431605","volume":"17","author":"JJ Cherry","year":"2012","unstructured":"Cherry, J. J. et al. Identification of novel compounds that increase SMN protein levels using an improved SMN2 reporter cell assay. J. Biomol. Screen. 17, 481\u2013495, https:\/\/doi.org\/10.1177\/1087057111431605 (2012).","journal-title":"J. Biomol. Screen."},{"key":"38208_CR20","doi-asserted-by":"publisher","first-page":"1035","DOI":"10.1002\/emmm.201202305","volume":"5","author":"JJ Cherry","year":"2013","unstructured":"Cherry, J. J. et al. Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds. EMBO Mol. Med. 5, 1035\u20131050, https:\/\/doi.org\/10.1002\/emmm.201202305 (2013).","journal-title":"EMBO Mol. Med."},{"key":"38208_CR21","doi-asserted-by":"publisher","first-page":"845","DOI":"10.1093\/hmg\/ddi078","volume":"14","author":"TT Le","year":"2005","unstructured":"Le, T. T. et al. SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN. Hum. Mol. Gen. 14, 845\u2013857, https:\/\/doi.org\/10.1093\/hmg\/ddi078 (2005).","journal-title":"Hum. Mol. Gen."},{"key":"38208_CR22","first-page":"1581","volume":"26","author":"W Bartsch","year":"1976","unstructured":"Bartsch, W., Sponer, G., Dietmann, K. & Fuchs, G. Acute toxicity of various solvents in the mouse and rat. LD50 of ethanol, diethylacetamide, dimethylformamide, dimethylsulfoxide, glycerine, N-methylpyrrolidone, polyethylene glycol 400, 1,2-propanediol and Tween 20. Arzneimittelforschung. 26, 1581\u20131583 (1976).","journal-title":"Arzneimittelforschung."},{"key":"38208_CR23","doi-asserted-by":"publisher","first-page":"4059","DOI":"10.1093\/hmg\/ddq329","volume":"19","author":"M Shababi","year":"2010","unstructured":"Shababi, M. et al. Cardiac defects contribute to the pathology of spinal muscular atrophy models. Hum. Mol. Genet. 19, 4059\u20134071, https:\/\/doi.org\/10.1093\/hmg\/ddq329 (2010).","journal-title":"Hum. Mol. Genet."},{"key":"38208_CR24","doi-asserted-by":"publisher","first-page":"3895","DOI":"10.1093\/hmg\/ddq300","volume":"19","author":"AK Bevan","year":"2010","unstructured":"Bevan, A. K. et al. Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery. Hum. Mol. Genet. 19, 3895\u20133905, https:\/\/doi.org\/10.1093\/hmg\/ddq300 (2010).","journal-title":"Hum. Mol. Genet."},{"key":"38208_CR25","doi-asserted-by":"publisher","first-page":"1403","DOI":"10.1016\/0306-3623(94)00300-C","volume":"26","author":"K Kramer","year":"1995","unstructured":"Kramer, K. et al. Effect of dimethyl sulfoxide (DMSO) on the electrocardiogram (ECG) in freely moving male Balb\/c mice. Gen. Pharmacol. 26, 1403\u20131407, https:\/\/doi.org\/10.1016\/0306-3623(94)00300-C (1995).","journal-title":"Gen. Pharmacol."},{"key":"38208_CR26","doi-asserted-by":"publisher","first-page":"1317","DOI":"10.1096\/fj.13-235440","volume":"28","author":"J Galvao","year":"2014","unstructured":"Galvao, J. et al. Unexpected low-dose toxicity of the universal solvent DMSO. FASEB J. 28, 1317\u20131330, https:\/\/doi.org\/10.1096\/fj.13-235440 (2014).","journal-title":"FASEB J."},{"key":"38208_CR27","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0613-5","volume":"12","author":"CA Wijngaarde","year":"2017","unstructured":"Wijngaarde, C. A. et al. Cardiac pathology in spinal muscular atrophy: a systematic review. Orphanet J. Rare Dis. 12, 67, https:\/\/doi.org\/10.1186\/s13023-017-0613-5 (2017).","journal-title":"Orphanet J. Rare Dis."},{"key":"38208_CR28","doi-asserted-by":"publisher","first-page":"949","DOI":"10.1093\/hmg\/ddm367","volume":"17","author":"LM Murray","year":"2008","unstructured":"Murray, L. M. et al. Selective vulnerability of motor neurons and dissociation of pre- and post-synaptic pathology at the neuromuscular junction in mouse models of spinal muscular atrophy. Hum. Mol. Genet. 17, 949\u2013962, https:\/\/doi.org\/10.1093\/hmg\/ddm367 (2008).","journal-title":"Hum. Mol. Genet."},{"key":"38208_CR29","doi-asserted-by":"publisher","first-page":"2552","DOI":"10.1093\/hmg\/ddn156","volume":"17","author":"S Kariya","year":"2008","unstructured":"Kariya, S. et al. Reduced SMN protein impairs maturation of the neuromuscular junctions in mouse models of spinal muscular atrophy. Hum. Mol. Genet. 17, 2552\u20132569, https:\/\/doi.org\/10.1093\/hmg\/ddn156 (2008).","journal-title":"Hum. Mol. Genet."},{"key":"38208_CR30","doi-asserted-by":"publisher","first-page":"842","DOI":"10.1523\/JNEUROSCI.4434-08.2009","volume":"29","author":"L Kong","year":"2009","unstructured":"Kong, L. et al. Impaired synaptic vesicle release and immaturity of neuromuscular junctions in spinal muscular atrophy mice. J. Neurosci. 29, 842\u2013851, https:\/\/doi.org\/10.1523\/JNEUROSCI.4434-08.2009 (2009).","journal-title":"J. Neurosci."},{"key":"38208_CR31","doi-asserted-by":"publisher","first-page":"3029","DOI":"10.1172\/JCI57291","volume":"121","author":"CM Lutz","year":"2011","unstructured":"Lutz, C. M. et al. Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy. J. Clin. Invest. 121, 3029\u20133041, https:\/\/doi.org\/10.1172\/JCI57291 (2011).","journal-title":"J. Clin. Invest."},{"key":"38208_CR32","doi-asserted-by":"publisher","first-page":"263","DOI":"10.1016\/j.nmd.2011.09.007","volume":"22","author":"M Bowerman","year":"2012","unstructured":"Bowerman, M., Murray, L. M., Beauvais, A., Pinheiro, B. & Kothary, R. A critical smn threshold in mice dictates onset of an intermediate spinal muscular atrophy phenotype associated with a distinct neuromuscular junction pathology. Neuromuscul. Disord. 22, 263\u2013276, https:\/\/doi.org\/10.1016\/j.nmd.2011.09.007 (2012).","journal-title":"Neuromuscul. Disord."},{"key":"38208_CR33","doi-asserted-by":"publisher","first-page":"5524","DOI":"10.1093\/hmg\/ddv283","volume":"24","author":"VL McGovern","year":"2015","unstructured":"McGovern, V. L. et al. SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNDelta7 mouse model of SMA. Hum. Mol. Genet. 24, 5524\u20135541, https:\/\/doi.org\/10.1093\/hmg\/ddv283 (2015).","journal-title":"Hum. Mol. Genet."},{"key":"38208_CR34","doi-asserted-by":"publisher","first-page":"72ra18","DOI":"10.1126\/scitranslmed.3001777","volume":"3","author":"MA Passini","year":"2011","unstructured":"Passini, M. A. et al. Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy. Sci. Transl. Med. 3, 72ra18, https:\/\/doi.org\/10.1126\/scitranslmed.3001777 (2011).","journal-title":"Sci. Transl. Med."},{"key":"38208_CR35","doi-asserted-by":"publisher","first-page":"1625","DOI":"10.1093\/hmg\/ddr600","volume":"21","author":"PN Porensky","year":"2012","unstructured":"Porensky, P. N. et al. A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse. Hum. Mol. Genet. 21, 1625\u20131638, https:\/\/doi.org\/10.1093\/hmg\/ddr600 (2012).","journal-title":"Hum. Mol. Genet."},{"key":"38208_CR36","doi-asserted-by":"publisher","first-page":"5144","DOI":"10.1016\/j.bmcl.2017.10.066","volume":"27","author":"S Choi","year":"2017","unstructured":"Choi, S. et al. Optimization of a series of heterocycles as survival motor neuron gene transcription enhancers. Bioorg. Med. Chem. Lett. 27, 5144\u20135148, https:\/\/doi.org\/10.1016\/j.bmcl.2017.10.066 (2017).","journal-title":"Bioorg. Med. Chem. Lett."},{"key":"38208_CR37","doi-asserted-by":"publisher","first-page":"3667","DOI":"10.1093\/hmg\/ddr288","volume":"20","author":"DY Kwon","year":"2011","unstructured":"Kwon, D. Y., Motley, W. W., Fischbeck, K. H. & Burnett, B. G. Increasing expression and decreasing degradation of SMN ameliorate the spinal muscular atrophy phenotype in mice. Hum. Mol. Genet. 20, 3667\u20133677, https:\/\/doi.org\/10.1093\/hmg\/ddr288 (2011).","journal-title":"Hum. Mol. Genet."},{"key":"38208_CR38","doi-asserted-by":"publisher","DOI":"10.1038\/s41419-017-0086-4","volume":"8","author":"A Piras","year":"2017","unstructured":"Piras, A. et al. Inhibition of autophagy delays motoneuron degeneration and extends lifespan in a mouse model of spinal muscular atrophy. Cell Death Dis. 8, 3223, https:\/\/doi.org\/10.1038\/s41419-017-0086-4 (2017).","journal-title":"Cell Death Dis."},{"key":"38208_CR39","doi-asserted-by":"publisher","first-page":"118","DOI":"10.1016\/j.nbd.2016.01.008","volume":"88","author":"E Foran","year":"2016","unstructured":"Foran, E. et al. CNS uptake of bortezomib is enhanced by P-glycoprotein inhibition: implications for spinal muscular atrophy. Neurobiol. Dis. 88, 118\u2013124, https:\/\/doi.org\/10.1016\/j.nbd.2016.01.008 (2016).","journal-title":"Neurobiol. Dis."},{"key":"38208_CR40","doi-asserted-by":"publisher","first-page":"e88427","DOI":"10.1172\/jci.insight.88427","volume":"1","author":"MB Abera","year":"2016","unstructured":"Abera, M. B. et al. ML372 blocks SMN ubiquitination and improves spinal muscular atrophy pathology in mice. JCI Insight 1, e88427, https:\/\/doi.org\/10.1172\/jci.insight.88427 (2016).","journal-title":"JCI Insight"},{"key":"38208_CR41","doi-asserted-by":"publisher","first-page":"126","DOI":"10.1523\/jneurosci.4489-09.2010","volume":"30","author":"TH Coady","year":"2010","unstructured":"Coady, T. H. & Lorson, C. L. Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy. J. Neurosci. 30, 126\u2013130, https:\/\/doi.org\/10.1523\/jneurosci.4489-09.2010 (2010).","journal-title":"J. Neurosci."}],"container-title":["Scientific Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/s41598-018-38208-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41598-018-38208-9","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41598-018-38208-9.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,12,17]],"date-time":"2022-12-17T05:52:52Z","timestamp":1671256372000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/s41598-018-38208-9"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,2,7]]},"references-count":41,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2019,12]]}},"alternative-id":["38208"],"URL":"https:\/\/doi.org\/10.1038\/s41598-018-38208-9","relation":{},"ISSN":["2045-2322"],"issn-type":[{"value":"2045-2322","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,2,7]]},"assertion":[{"value":"25 October 2018","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"20 December 2018","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"7 February 2019","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"CLL is the co-founder and Chief Scientific Officer of Shift Pharmaceuticals.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing Interests"}}],"article-number":"1633"}}