{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,22]],"date-time":"2026-10-22T14:30:35Z","timestamp":1792679435163,"version":"4.5.0"},"reference-count":54,"publisher":"Springer Science and Business Media LLC","issue":"7","license":[{"start":{"date-parts":[[2015,6,1]],"date-time":"2015-06-01T00:00:00Z","timestamp":1433116800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2015,6,1]],"date-time":"2015-06-01T00:00:00Z","timestamp":1433116800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Nat Chem Biol"],"published-print":{"date-parts":[[2015,7]]},"DOI":"10.1038\/nchembio.1837","type":"journal-article","created":{"date-parts":[[2015,6,1]],"date-time":"2015-06-01T15:25:16Z","timestamp":1433172316000},"page":"511-517","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":433,"title":["SMN2 splice modulators enhance U1\u2013pre-mRNA association and rescue SMA mice"],"prefix":"10.1038","volume":"11","author":[{"given":"James","family":"Palacino","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susanne E","family":"Swalley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cheng","family":"Song","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Atwood K","family":"Cheung","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lei","family":"Shu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaolu","family":"Zhang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mailin","family":"Van Hoosear","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Youngah","family":"Shin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Donovan N","family":"Chin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline Gubser","family":"Keller","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Martin","family":"Beibel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicole A","family":"Renaud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas M","family":"Smith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Salcius","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoying","family":"Shi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc","family":"Hild","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca","family":"Servais","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monish","family":"Jain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lin","family":"Deng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caroline","family":"Bullock","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"McLellan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sven","family":"Schuierer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leo","family":"Murphy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcel J J","family":"Blommers","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cecile","family":"Blaustein","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frada","family":"Berenshteyn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arnaud","family":"Lacoste","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jason R","family":"Thomas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guglielmo","family":"Roma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregory A","family":"Michaud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian S","family":"Tseng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffery A","family":"Porter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vic E","family":"Myer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John A","family":"Tallarico","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lawrence G","family":"Hamann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Curtis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark C","family":"Fishman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William F","family":"Dietrich","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natalie A","family":"Dales","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rajeev","family":"Sivasankaran","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2015,6,1]]},"reference":[{"key":"BFnchembio1837_CR1","doi-asserted-by":"publisher","first-page":"27","DOI":"10.1038\/ejhg.2011.134","volume":"20","author":"EA Sugarman","year":"2012","unstructured":"Sugarman, E.A. et al. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of &gt;72,400 specimens. Eur. J. Hum. Genet. 20, 27\u201332 (2012).","journal-title":"Eur. J. Hum. Genet."},{"key":"BFnchembio1837_CR2","doi-asserted-by":"publisher","first-page":"2120","DOI":"10.1016\/S0140-6736(08)60921-6","volume":"371","author":"MR Lunn","year":"2008","unstructured":"Lunn, M.R. & Wang, C.H. Spinal muscular atrophy. Lancet 371, 2120\u20132133 (2008).","journal-title":"Lancet"},{"key":"BFnchembio1837_CR3","doi-asserted-by":"publisher","first-page":"223","DOI":"10.1007\/s10059-012-0005-6","volume":"33","author":"J Zhou","year":"2012","unstructured":"Zhou, J., Zheng, X. & Shen, H. Targeting RNA-splicing for SMA treatment. Mol. Cells 33, 223\u2013228 (2012).","journal-title":"Mol. Cells"},{"key":"BFnchembio1837_CR4","doi-asserted-by":"publisher","first-page":"979","DOI":"10.1001\/archneurol.2011.74","volume":"68","author":"SJ Kolb","year":"2011","unstructured":"Kolb, S.J. & Kissel, J.T. Spinal muscular atrophy: a timely review. Arch. Neurol. 68, 979\u2013984 (2011).","journal-title":"Arch. Neurol."},{"key":"BFnchembio1837_CR5","doi-asserted-by":"publisher","first-page":"155","DOI":"10.1016\/0092-8674(95)90460-3","volume":"80","author":"S Lefebvre","year":"1995","unstructured":"Lefebvre, S. et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell 80, 155\u2013165 (1995).","journal-title":"Cell"},{"key":"BFnchembio1837_CR6","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. A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum. Mol. Genet. 8, 1177\u20131183 (1999).","journal-title":"Hum. Mol. Genet."},{"key":"BFnchembio1837_CR7","doi-asserted-by":"publisher","first-page":"230","DOI":"10.1038\/3030","volume":"20","author":"DK Gavrilov","year":"1998","unstructured":"Gavrilov, D.K., Shi, X., Das, K., Gilliam, T.C. & Wang, C.H. Differential SMN2 expression associated with SMA severity. Nat. Genet. 20, 230\u2013231 (1998).","journal-title":"Nat. Genet."},{"key":"BFnchembio1837_CR8","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 (1999).","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"BFnchembio1837_CR9","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. SMN\u03947, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN. Hum. Mol. Genet. 14, 845\u2013857 (2005).","journal-title":"Hum. Mol. Genet."},{"key":"BFnchembio1837_CR10","doi-asserted-by":"publisher","first-page":"2525","DOI":"10.1093\/hmg\/8.13.2525","volume":"8","author":"T Vitali","year":"1999","unstructured":"Vitali, T. et al. Detection of the survival motor neuron (SMN) genes by FISH: further evidence for a role for SMN2 in the modulation of disease severity in SMA patients. Hum. Mol. Genet. 8, 2525\u20132532 (1999).","journal-title":"Hum. Mol. Genet."},{"key":"BFnchembio1837_CR11","doi-asserted-by":"publisher","first-page":"885","DOI":"10.1016\/j.neuron.2005.12.001","volume":"48","author":"UR Monani","year":"2005","unstructured":"Monani, U.R. Spinal muscular atrophy: a deficiency in a ubiquitous protein; a motor neuron-specific disease. Neuron 48, 885\u2013896 (2005).","journal-title":"Neuron"},{"key":"BFnchembio1837_CR12","doi-asserted-by":"publisher","first-page":"979","DOI":"10.1177\/0883073807305787","volume":"22","author":"CJ Sumner","year":"2007","unstructured":"Sumner, C.J. Molecular mechanisms of spinal muscular atrophy. J. Child Neurol. 22, 979\u2013989 (2007).","journal-title":"J. Child Neurol."},{"key":"BFnchembio1837_CR13","doi-asserted-by":"publisher","first-page":"1411","DOI":"10.1086\/515465","volume":"60","author":"PE McAndrew","year":"1997","unstructured":"McAndrew, P.E. et al. Identification of proximal spinal muscular atrophy carriers and patients by analysis of SMNT and SMNC gene copy number. Am. J. Hum. Genet. 60, 1411\u20131422 (1997).","journal-title":"Am. J. Hum. Genet."},{"key":"BFnchembio1837_CR14","doi-asserted-by":"publisher","first-page":"108","DOI":"10.1038\/nrm3742","volume":"15","author":"AG Matera","year":"2014","unstructured":"Matera, A.G. & Wang, Z. A day in the life of the spliceosome. Nat. Rev. Mol. Cell Biol. 15, 108\u2013121 (2014).","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"BFnchembio1837_CR15","doi-asserted-by":"publisher","first-page":"470","DOI":"10.1038\/nature07509","volume":"456","author":"ET Wang","year":"2008","unstructured":"Wang, E.T. et al. Alternative isoform regulation in human tissue transcriptomes. Nature 456, 470\u2013476 (2008).","journal-title":"Nature"},{"key":"BFnchembio1837_CR16","doi-asserted-by":"publisher","first-page":"802","DOI":"10.1261\/rna.876308","volume":"14","author":"Z Wang","year":"2008","unstructured":"Wang, Z. & Burge, C.B. Splicing regulation: from a parts list of regulatory elements to an integrated splicing code. RNA 14, 802\u2013813 (2008).","journal-title":"RNA"},{"key":"BFnchembio1837_CR17","doi-asserted-by":"publisher","first-page":"1254806","DOI":"10.1126\/science.1254806","volume":"347","author":"HY Xiong","year":"2014","unstructured":"Xiong, H.Y. et al. The human splicing code reveals new insights into the genetic determinants of disease. Science 347, 1254806 (2014).","journal-title":"Science"},{"key":"BFnchembio1837_CR18","doi-asserted-by":"publisher","first-page":"e73","DOI":"10.1371\/journal.pbio.0050073","volume":"5","author":"Y Hua","year":"2007","unstructured":"Hua, Y., Vickers, T.A., Baker, B.F., Bennett, C.F. & Krainer, A.R. Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon. PLoS Biol. 5, e73 (2007).","journal-title":"PLoS Biol."},{"key":"BFnchembio1837_CR19","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 (2008).","journal-title":"Am. J. Hum. Genet."},{"key":"BFnchembio1837_CR20","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. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science 345, 688\u2013693 (2014).","journal-title":"Science"},{"key":"BFnchembio1837_CR21","doi-asserted-by":"publisher","first-page":"129","DOI":"10.1101\/gad.209759.112","volume":"27","author":"X Roca","year":"2013","unstructured":"Roca, X., Krainer, A.R. & Eperon, I.C. Pick one, but be quick: 5\u2032 splice sites and the problems of too many choices. Genes Dev. 27, 129\u2013144 (2013).","journal-title":"Genes Dev."},{"key":"BFnchembio1837_CR22","first-page":"2675","volume":"6","author":"MA Lewandowska","year":"2013","unstructured":"Lewandowska, M.A. The missing puzzle piece: splicing mutations. Int. J. Clin. Exp. Pathol. 6, 2675\u20132682 (2013).","journal-title":"Int. J. Clin. Exp. Pathol."},{"key":"BFnchembio1837_CR23","doi-asserted-by":"publisher","first-page":"945","DOI":"10.1007\/s12265-013-9482-z","volume":"6","author":"E Lara-Pezzi","year":"2013","unstructured":"Lara-Pezzi, E., G\u00f3mez-Salinero, J., Gatto, A. & Garc\u00eda-Pav\u00eda, P. The alternative heart: impact of alternative splicing in heart disease. J. Cardiovasc. Transl. Res. 6, 945\u2013955 (2013).","journal-title":"J. Cardiovasc. Transl. Res."},{"key":"BFnchembio1837_CR24","doi-asserted-by":"publisher","first-page":"D1055","DOI":"10.1093\/nar\/gkr1171","volume":"40","author":"J Wang","year":"2012","unstructured":"Wang, J., Zhang, J., Li, K., Zhao, W. & Cui, Q. SpliceDisease database: linking RNA splicing and disease. Nucleic Acids Res. 40, D1055\u2013D1059 (2012).","journal-title":"Nucleic Acids Res."},{"key":"BFnchembio1837_CR25","doi-asserted-by":"publisher","first-page":"4431","DOI":"10.1093\/hmg\/dds285","volume":"21","author":"M Osborne","year":"2012","unstructured":"Osborne, M. et al. Characterization of behavioral and neuromuscular junction phenotypes in a novel allelic series of SMA mouse models. Hum. Mol. Genet. 21, 4431\u20134447 (2012).","journal-title":"Hum. Mol. Genet."},{"key":"BFnchembio1837_CR26","doi-asserted-by":"publisher","first-page":"1107","DOI":"10.1128\/MCB.01262-08","volume":"29","author":"BG Burnett","year":"2009","unstructured":"Burnett, B.G. et al. Regulation of SMN protein stability. Mol. Cell. Biol. 29, 1107\u20131115 (2009).","journal-title":"Mol. Cell. Biol."},{"key":"BFnchembio1837_CR27","doi-asserted-by":"publisher","first-page":"29","DOI":"10.1016\/j.expneurol.2008.02.025","volume":"212","author":"BF El-Khodor","year":"2008","unstructured":"El-Khodor, B.F. et al. Identification of a battery of tests for drug candidate evaluation in the SMN\u03947 neonate model of spinal muscular atrophy. Exp. Neurol. 212, 29\u201343 (2008).","journal-title":"Exp. Neurol."},{"key":"BFnchembio1837_CR28","doi-asserted-by":"publisher","first-page":"3578","DOI":"10.1093\/hmg\/ddr275","volume":"20","author":"TT Le","year":"2011","unstructured":"Le, T.T. et al. Temporal requirement for high SMN expression in SMA mice. Hum. Mol. Genet. 20, 3578\u20133591 (2011).","journal-title":"Hum. Mol. Genet."},{"key":"BFnchembio1837_CR29","doi-asserted-by":"publisher","first-page":"785","DOI":"10.1172\/JCI72017","volume":"124","author":"S Kariya","year":"2014","unstructured":"Kariya, S. et al. Requirement of enhanced Survival Motoneuron protein imposed during neuromuscular junction maturation. J. Clin. Invest. 124, 785\u2013800 (2014).","journal-title":"J. Clin. Invest."},{"key":"BFnchembio1837_CR30","doi-asserted-by":"publisher","first-page":"e112","DOI":"10.1093\/nar\/gkq041","volume":"38","author":"H Richard","year":"2010","unstructured":"Richard, H. et al. Prediction of alternative isoforms from exon expression levels in RNA-Seq experiments. Nucleic Acids Res. 38, e112 (2010).","journal-title":"Nucleic Acids Res."},{"key":"BFnchembio1837_CR31","doi-asserted-by":"publisher","first-page":"3850","DOI":"10.1128\/MCB.02253-07","volume":"28","author":"E Goina","year":"2008","unstructured":"Goina, E., Skoko, N. & Pagani, F. Binding of DAZAP1 and hnRNPA1\/A2 to an exonic splicing silencer in a natural BRCA1 exon 18 mutant. Mol. Cell. Biol. 28, 3850\u20133860 (2008).","journal-title":"Mol. Cell. Biol."},{"key":"BFnchembio1837_CR32","doi-asserted-by":"publisher","first-page":"371","DOI":"10.1093\/nar\/gkl1050","volume":"35","author":"NN Singh","year":"2007","unstructured":"Singh, N.N., Singh, R.N. & Androphy, E.J. Modulating role of RNA structure in alternative splicing of a critical exon in the spinal muscular atrophy genes. Nucleic Acids Res. 35, 371\u2013389 (2007).","journal-title":"Nucleic Acids Res."},{"key":"BFnchembio1837_CR33","doi-asserted-by":"publisher","first-page":"1188","DOI":"10.1101\/gr.849004","volume":"14","author":"GE Crooks","year":"2004","unstructured":"Crooks, G.E., Hon, G., Chandonia, J.-M. & Brenner, S.E. WebLogo: a sequence logo generator. Genome Res. 14, 1188\u20131190 (2004).","journal-title":"Genome Res."},{"key":"BFnchembio1837_CR34","doi-asserted-by":"publisher","first-page":"W202","DOI":"10.1093\/nar\/gkp335","volume":"37","author":"TL Bailey","year":"2009","unstructured":"Bailey, T.L. et al. MEME SUITE: tools for motif discovery and searching. Nucleic Acids Res. 37, W202\u2013W208 (2009).","journal-title":"Nucleic Acids Res."},{"key":"BFnchembio1837_CR35","doi-asserted-by":"publisher","first-page":"R30","DOI":"10.1186\/gb-2009-10-3-r30","volume":"10","author":"M Akerman","year":"2009","unstructured":"Akerman, M., David-Eden, H., Pinter, R.Y. & Mandel-Gutfreund, Y. A computational approach for genome-wide mapping of splicing factor binding sites. Genome Biol. 10, R30 (2009).","journal-title":"Genome Biol."},{"key":"BFnchembio1837_CR36","doi-asserted-by":"publisher","first-page":"377","DOI":"10.1089\/1066527041410418","volume":"11","author":"G Yeo","year":"2004","unstructured":"Yeo, G. & Burge, C.B. Maximum entropy modeling of short sequence motifs with applications to RNA splicing signals. J. Comput. Biol. 11, 377\u2013394 (2004).","journal-title":"J. Comput. Biol."},{"key":"BFnchembio1837_CR37","doi-asserted-by":"publisher","first-page":"917","DOI":"10.1177\/1087057114522691","volume":"19","author":"M Salcius","year":"2014","unstructured":"Salcius, M. et al. SEC-TID: a label-free method for small-molecule target identification. J. Biomol. Screen. 19, 917\u2013927 (2014).","journal-title":"J. Biomol. Screen."},{"key":"BFnchembio1837_CR38","doi-asserted-by":"publisher","first-page":"e04986","DOI":"10.7554\/eLife.04986","volume":"4","author":"Y Kondo","year":"2015","unstructured":"Kondo, Y., Oubridge, C., van Roon, A.-M.M. & Nagai, K. Crystal structure of human U1 snRNP, a small nuclear ribonucleoprotein particle, reveals the mechanism of 5\u2032 splice site recognition. Elife 4, e04986 (2015).","journal-title":"Elife"},{"key":"BFnchembio1837_CR39","doi-asserted-by":"publisher","first-page":"46","DOI":"10.1124\/jpet.113.212407","volume":"350","author":"F Rigo","year":"2014","unstructured":"Rigo, F. et al. Pharmacology of a central nervous system delivered 2\u2032-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates. J. Pharmacol. Exp. Ther. 350, 46\u201355 (2014).","journal-title":"J. Pharmacol. Exp. Ther."},{"key":"BFnchembio1837_CR40","doi-asserted-by":"publisher","first-page":"570","DOI":"10.1038\/nchembio.2007.16","volume":"3","author":"Y Kotake","year":"2007","unstructured":"Kotake, Y. et al. Splicing factor SF3b as a target of the antitumor natural product pladienolide. Nat. Chem. Biol. 3, 570\u2013575 (2007).","journal-title":"Nat. Chem. Biol."},{"key":"BFnchembio1837_CR41","doi-asserted-by":"publisher","first-page":"1532","DOI":"10.1038\/sj.gt.3301550","volume":"8","author":"ML Zhang","year":"2001","unstructured":"Zhang, M.L., Lorson, C.L., Androphy, E.J. & Zhou, J. An in vivo reporter system for measuring increased inclusion of exon 7 in SMN2 mRNA: potential therapy of SMA. Gene Ther. 8, 1532\u20131538 (2001).","journal-title":"Gene Ther."},{"key":"BFnchembio1837_CR42","unstructured":"Cheung, A.K. et al. 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency\u2013related conditions. United States Patent 8,729,263 (2014)."},{"key":"BFnchembio1837_CR43","doi-asserted-by":"publisher","first-page":"861","DOI":"10.1016\/j.cell.2007.11.019","volume":"131","author":"K Takahashi","year":"2007","unstructured":"Takahashi, K. et al. Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131, 861\u2013872 (2007).","journal-title":"Cell"},{"key":"BFnchembio1837_CR44","doi-asserted-by":"publisher","first-page":"332","DOI":"10.1016\/j.stem.2009.07.011","volume":"5","author":"A Lacoste","year":"2009","unstructured":"Lacoste, A., Berenshteyn, F. & Brivanlou, A.H. An efficient and reversible transposable system for gene delivery and lineage-specific differentiation in human embryonic stem cells. Cell Stem Cell 5, 332\u2013342 (2009).","journal-title":"Cell Stem Cell"},{"key":"BFnchembio1837_CR45","doi-asserted-by":"publisher","first-page":"275","DOI":"10.1038\/nbt.1529","volume":"27","author":"SM Chambers","year":"2009","unstructured":"Chambers, S.M. et al. Highly efficient neural conversion of human ES and iPS cells by dual inhibition of SMAD signaling. Nat. Biotechnol. 27, 275\u2013280 (2009).","journal-title":"Nat. Biotechnol."},{"key":"BFnchembio1837_CR46","doi-asserted-by":"publisher","first-page":"D61","DOI":"10.1093\/nar\/gkl842","volume":"35","author":"KD Pruitt","year":"2007","unstructured":"Pruitt, K.D., Tatusova, T. & Maglott, D.R. NCBI reference sequences (RefSeq): a curated non-redundant sequence database of genomes, transcripts and proteins. Nucleic Acids Res. 35, D61\u2013D65 (2007).","journal-title":"Nucleic Acids Res."},{"key":"BFnchembio1837_CR47","doi-asserted-by":"publisher","first-page":"357","DOI":"10.1038\/nmeth.1923","volume":"9","author":"B Langmead","year":"2012","unstructured":"Langmead, B. & Salzberg, S.L. Fast gapped-read alignment with Bowtie 2. Nat. Methods 9, 357\u2013359 (2012).","journal-title":"Nat. Methods"},{"key":"BFnchembio1837_CR48","doi-asserted-by":"publisher","first-page":"993","DOI":"10.1016\/0092-8674(84)90049-7","volume":"36","author":"AR Krainer","year":"1984","unstructured":"Krainer, A.R., Maniatis, T., Ruskin, B. & Green, M.R. Normal and mutant human \u03b2-globin pre-mRNAs are faithfully and efficiently spliced in vitro. Cell 36, 993\u20131005 (1984).","journal-title":"Cell"},{"key":"BFnchembio1837_CR49","doi-asserted-by":"publisher","first-page":"9415","DOI":"10.1093\/nar\/16.20.9415","volume":"16","author":"AR Krainer","year":"1988","unstructured":"Krainer, A.R. Pre-mRNA splicing by complementation with purified human U1, U2, U4\/U6 and U5 snRNPs. Nucleic Acids Res. 16, 9415\u20139429 (1988).","journal-title":"Nucleic Acids Res."},{"key":"BFnchembio1837_CR50","first-page":"289","volume":"118","author":"B Kastner","year":"1999","unstructured":"Kastner, B. & L\u00fchrmann, R. Purification of U small nuclear ribonucleoprotein particles. Methods Mol. Biol. 118, 289\u2013298 (1999).","journal-title":"Methods Mol. Biol."},{"key":"BFnchembio1837_CR51","doi-asserted-by":"publisher","first-page":"1986","DOI":"10.1021\/jm991174y","volume":"43","author":"A Frostell-Karlsson","year":"2000","unstructured":"Frostell-Karlsson, A. et al. Biosensor analysis of the interaction between immobilized human serum albumin and drug compounds for prediction of human serum albumin binding levels. J. Med. Chem. 43, 1986\u20131992 (2000).","journal-title":"J. Med. Chem."},{"key":"BFnchembio1837_CR52","doi-asserted-by":"publisher","first-page":"51","DOI":"10.1038\/nature06684","volume":"452","author":"M Parisien","year":"2008","unstructured":"Parisien, M. & Major, F. The MC-Fold and MC-Sym pipeline infers RNA structure from sequence data. Nature 452, 51\u201355 (2008).","journal-title":"Nature"},{"key":"BFnchembio1837_CR53","doi-asserted-by":"publisher","first-page":"2705","DOI":"10.1021\/ci3001088","volume":"52","author":"M Rueda","year":"2012","unstructured":"Rueda, M., Totrov, M. & Abagyan, R. ALiBERO: evolving a team of complementary pocket conformations rather than a single leader. J. Chem. Inf. Model. 52, 2705\u20132714 (2012).","journal-title":"J. Chem. Inf. Model."},{"key":"BFnchembio1837_CR54","doi-asserted-by":"publisher","first-page":"983","DOI":"10.1006\/jmbi.1994.1052","volume":"235","author":"R Abagyan","year":"1994","unstructured":"Abagyan, R. & Totrov, M. Biased probability Monte Carlo conformational searches and electrostatic calculations for peptides and proteins. J. Mol. Biol. 235, 983\u20131002 (1994).","journal-title":"J. Mol. Biol."}],"updated-by":[{"DOI":"10.1038\/nchembio0915-741a","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2015,8,18]],"date-time":"2015-08-18T00:00:00Z","timestamp":1439856000000}},{"DOI":"10.1038\/nchembio0416-304c","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2016,3,18]],"date-time":"2016-03-18T00:00:00Z","timestamp":1458259200000}}],"container-title":["Nature Chemical Biology"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.nature.com\/articles\/nchembio.1837.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/www.nature.com\/articles\/nchembio.1837","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/www.nature.com\/articles\/nchembio.1837.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,5,18]],"date-time":"2023-05-18T15:39:07Z","timestamp":1684424347000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/nchembio.1837"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015,6,1]]},"references-count":54,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2015,7]]}},"alternative-id":["BFnchembio1837"],"URL":"https:\/\/doi.org\/10.1038\/nchembio.1837","relation":{},"ISSN":["1552-4450","1552-4469"],"issn-type":[{"value":"1552-4450","type":"print"},{"value":"1552-4469","type":"electronic"}],"subject":[],"published":{"date-parts":[[2015,6,1]]},"assertion":[{"value":"5 December 2014","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"6 May 2015","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"1 June 2015","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"15 July 2015","order":4,"name":"change_date","label":"Change Date","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Corrigendum","order":5,"name":"change_type","label":"Change Type","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"The authors forgot to include a description of the measurement of SMN protein levels in tissue samples in the Online Methods. This description has been included in the HTML and PDF versions of the article.","order":6,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"11 February 2016","order":5,"name":"change_date","label":"Change Date","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Corrigendum","order":5,"name":"change_type","label":"Change Type","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"In the version of this article originally published online, the schematic for the construct in\n                      Figure 4a\n                      was incorrect. A corrected figure has been provided in the HTML and PDF versions of the article.","order":6,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}}]}}