{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,2,21]],"date-time":"2025-02-21T11:19:38Z","timestamp":1740136778471,"version":"3.37.3"},"reference-count":87,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2022,8,1]],"date-time":"2022-08-01T00:00:00Z","timestamp":1659312000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2022,8,1]],"date-time":"2022-08-01T00:00:00Z","timestamp":1659312000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"content-domain":{"domain":["molpharm.aspetjournals.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Pharmacology"],"published-print":{"date-parts":[[2022,8]]},"DOI":"10.1124\/molpharm.122.000529","type":"journal-article","created":{"date-parts":[[2022,6,6]],"date-time":"2022-06-06T12:35:11Z","timestamp":1654518911000},"page":"92-105","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Effects of Inhibitors of SLC9A-Type Sodium-Proton Exchangers on Survival Motor Neuron 2 (SMN2) mRNA Splicing and Expression"],"prefix":"10.1016","volume":"102","author":[{"given":"Sambee","family":"Kanda","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0930-7354","authenticated-orcid":false,"given":"Emily","family":"Moulton","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2912-6400","authenticated-orcid":false,"given":"Matthew E.R.","family":"Butchbach","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1124\/molpharm.122.000529_bib1","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"},{"key":"10.1124\/molpharm.122.000529_bib2","doi-asserted-by":"crossref","first-page":"915","DOI":"10.1056\/NEJMoa2009965","article-title":"Risdiplam in type 1 spinal muscular atrophy","volume":"384","author":"Baranello","year":"2021","journal-title":"N Engl J Med"},{"key":"10.1124\/molpharm.122.000529_bib3","doi-asserted-by":"crossref","first-page":"D991","DOI":"10.1093\/nar\/gks1193","article-title":"NCBI GEO: archive for functional genomics data sets--update","volume":"41","author":"Barrett","year":"2013","journal-title":"Nucleic Acids Res"},{"key":"10.1124\/molpharm.122.000529_bib4","doi-asserted-by":"crossref","first-page":"6243","DOI":"10.1074\/jbc.270.11.6243","article-title":"The alternative splicing pattern of the tenascin-C pre-mRNA is controlled by the extracellular pH","volume":"270","author":"Borsi","year":"1995","journal-title":"J Biol Chem"},{"key":"10.1124\/molpharm.122.000529_bib5","doi-asserted-by":"crossref","first-page":"1107","DOI":"10.1128\/MCB.01262-08","article-title":"Regulation of SMN protein stability","volume":"29","author":"Burnett","year":"2009","journal-title":"Mol Cell Biol"},{"key":"10.1124\/molpharm.122.000529_bib6","doi-asserted-by":"crossref","first-page":"7","DOI":"10.3389\/fmolb.2016.00007","article-title":"Survival Motor Neuron genes: implications for spinal muscular atrophy and other neurodegenerative diseases","volume":"3","author":"Butchbach","year":"2016","journal-title":"Front Mol Biosci"},{"key":"10.1124\/molpharm.122.000529_bib7","doi-asserted-by":"crossref","first-page":"10067","DOI":"10.1021\/acs.jmedchem.6b00670","article-title":"Small molecules in development for the treatment of spinal muscular atrophy","volume":"59","author":"Calder","year":"2016","journal-title":"J Med Chem"},{"key":"10.1124\/molpharm.122.000529_bib8","first-page":"e00299","article-title":"A heterologous cell model for studying the role of T-cell intracellular antigen 1 in Welander distal myopathy","volume":"39","author":"Carrascoso","year":"2018","journal-title":"Mol Cell Biol"},{"key":"10.1124\/molpharm.122.000529_bib9","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1086\/498853","article-title":"Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 SMN2","volume":"78","author":"Cartegni","year":"2006","journal-title":"Am J Hum Genet"},{"key":"10.1124\/molpharm.122.000529_bib10","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1038\/ng854","article-title":"Disruption of an SF2\/ASF-dependent exonic splicing enhancer in SMN2 SMN1","volume":"30","author":"Cartegni","year":"2002","journal-title":"Nat Genet"},{"key":"10.1124\/molpharm.122.000529_bib11","doi-asserted-by":"crossref","first-page":"828","DOI":"10.1002\/ajmg.b.32600","article-title":"Nhe5 deficiency enhances learning and memory via upregulating Bdnf\/TrkB signaling in mice","volume":"174","author":"Chen","year":"2017","journal-title":"Am J Med Genet B Neuropsychiatr Genet"},{"key":"10.1124\/molpharm.122.000529_bib12","article-title":"High expression level of Tra2-\u03b21 is responsible for increased SMN2 exon 7 inclusion in the testis of SMA mice","volume":"10","author":"Chen","year":"2015","journal-title":"PLoS One"},{"key":"10.1124\/molpharm.122.000529_bib13","doi-asserted-by":"crossref","first-page":"268","DOI":"10.1016\/j.mcn.2008.07.002","article-title":"Extracellular pH change modulates the exon 7 splicing in SMN2 mRNA","volume":"39","author":"Chen","year":"2008","journal-title":"Mol Cell Neurosci"},{"key":"10.1124\/molpharm.122.000529_bib14","doi-asserted-by":"crossref","first-page":"6430","DOI":"10.1038\/s41598-019-42872-w","article-title":"The Na+\/H+-exchanger NHE1 regulates extra- and intracellular pH and nimodipine-sensitive [Ca2+]i in the suprachiasmatic nucleus","volume":"9","author":"Cheng","year":"2019","journal-title":"Sci Rep"},{"key":"10.1124\/molpharm.122.000529_bib15","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1089\/adt.2014.587","article-title":"Assays for the identification and prioritization of drug candidates for spinal muscular atrophy","volume":"12","author":"Cherry","year":"2014","journal-title":"Assay Drug Dev Technol"},{"key":"10.1124\/molpharm.122.000529_bib16","doi-asserted-by":"crossref","first-page":"438","DOI":"10.1101\/gad.1884910","article-title":"SMN2 exon 7 skipping is a principal contributor to spinal muscular atrophy severity","volume":"24","author":"Cho","year":"2010","journal-title":"Genes Dev"},{"key":"10.1124\/molpharm.122.000529_bib17","doi-asserted-by":"crossref","first-page":"R64","DOI":"10.1186\/gb-2007-8-4-r64","article-title":"Discovery of tissue-specific exons using comprehensive human exon microarrays","volume":"8","author":"Clark","year":"2007","journal-title":"Genome Biol"},{"key":"10.1124\/molpharm.122.000529_bib18","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1006\/nbdi.1996.0010","article-title":"The neurobiology of childhood spinal muscular atrophy","volume":"3","author":"Crawford","year":"1996","journal-title":"Neurobiol Dis"},{"key":"10.1124\/molpharm.122.000529_bib19","doi-asserted-by":"crossref","first-page":"1244","DOI":"10.1046\/j.1471-0528.2002.02083.x","article-title":"Prenatal diagnosis for risk of spinal muscular atrophy","volume":"109","author":"Cusc\u00f3","year":"2002","journal-title":"BJOG"},{"key":"10.1124\/molpharm.122.000529_bib20","doi-asserted-by":"crossref","first-page":"1576","DOI":"10.1124\/mol.107.037150","article-title":"Inhibition of TRPP3 channel by amiloride and analogs","volume":"72","author":"Dai","year":"2007","journal-title":"Mol Pharmacol"},{"key":"10.1124\/molpharm.122.000529_bib21","doi-asserted-by":"crossref","first-page":"2246","DOI":"10.1091\/mbc.e11-01-0066","article-title":"Regulation of dendritic spine growth through activity-dependent recruitment of the brain-enriched Na+\/H+ exchanger NHE5","volume":"22","author":"Diering","year":"2011","journal-title":"Mol Biol Cell"},{"key":"10.1124\/molpharm.122.000529_bib22","doi-asserted-by":"crossref","first-page":"220","DOI":"10.1002\/humu.21419","article-title":"SMN2 exon 7 splicing is inhibited by binding of hnRNP A1 to a common ESS motif that spans the 3\u2032 splice site","volume":"32","author":"Doktor","year":"2011","journal-title":"Hum Mutat"},{"key":"10.1124\/molpharm.122.000529_bib23","doi-asserted-by":"crossref","first-page":"236","DOI":"10.1016\/j.mam.2012.05.001","article-title":"SLC9\/NHE gene family, a plasma membrane and organellar family of Na+\/H+ exchangers","volume":"34","author":"Donowitz","year":"2013","journal-title":"Mol Aspects Med"},{"key":"10.1124\/molpharm.122.000529_bib24","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1074\/mcp.M113.035600","article-title":"Analysis of the human tissue-specific expression by genome-wide integration of transcriptomics and antibody-based proteomics","volume":"13","author":"Fagerberg","year":"2014","journal-title":"Mol Cell Proteomics"},{"key":"10.1124\/molpharm.122.000529_bib25","doi-asserted-by":"crossref","first-page":"162","DOI":"10.3389\/fncel.2019.00162","article-title":"Fatty acid signaling mechanisms in neural cells: fatty acid receptors","volume":"13","author":"Falomir-Lockhart","year":"2019","journal-title":"Front Cell Neurosci"},{"key":"10.1124\/molpharm.122.000529_bib26","doi-asserted-by":"crossref","first-page":"702","DOI":"10.1091\/mbc.E15-04-0257","article-title":"Endosomal Na+\/H+ exchanger NHE5 influences MET recycling and cell migration","volume":"27","author":"Fan","year":"2016","journal-title":"Mol Biol Cell"},{"key":"10.1124\/molpharm.122.000529_bib27","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1093\/bib\/bbr004","article-title":"Design and validation issues in RNA-seq experiments","volume":"12","author":"Fang","year":"2011","journal-title":"Brief Bioinform"},{"key":"10.1124\/molpharm.122.000529_bib28","doi-asserted-by":"crossref","first-page":"964","DOI":"10.1093\/hmg\/ddv629","article-title":"Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset","volume":"25","author":"Feng","year":"2016","journal-title":"Hum Mol Genet"},{"key":"10.1124\/molpharm.122.000529_bib29","doi-asserted-by":"crossref","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","article-title":"Nusinersen versus sham control in infantile-onset spinal muscular atrophy","volume":"377","author":"Finkel","year":"2017","journal-title":"N Engl J Med"},{"key":"10.1124\/molpharm.122.000529_bib30","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0180657","article-title":"The effects of C5-substituted 2,4-diaminoquinazolines on selected transcript expression in spinal muscular atrophy cells","volume":"12","author":"Gentillon","year":"2017","journal-title":"PLoS One"},{"key":"10.1124\/molpharm.122.000529_bib31","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.braindev.2011.04.010","article-title":"SMN2 expression and modulates SF2\/ASF and hnRNPA1 expression in SMA fibroblast cell lines","volume":"34","author":"Harahap","year":"2012","journal-title":"Brain Dev"},{"key":"10.1124\/molpharm.122.000529_bib32","doi-asserted-by":"crossref","first-page":"1013","DOI":"10.1177\/0883073807305669","article-title":"SMN transcript stability: could modulation of messenger RNA degradation provide a novel therapy for spinal muscular atrophy?","volume":"22","author":"Heier","year":"2007","journal-title":"J Child Neurol"},{"key":"10.1124\/molpharm.122.000529_bib33","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"},{"key":"10.1124\/molpharm.122.000529_bib34","doi-asserted-by":"crossref","first-page":"554","DOI":"10.1007\/s004390000409","article-title":"Exclusion of Htra2-\u03b21, an up-regulator of full-length SMN2 transcript, as a modifying gene for spinal muscular atrophy","volume":"107","author":"Helmken","year":"2000","journal-title":"Hum Genet"},{"key":"10.1124\/molpharm.122.000529_bib35","doi-asserted-by":"crossref","first-page":"9618","DOI":"10.1073\/pnas.160181697","article-title":"Htra2-\u03b2 1 stimulates an exonic splicing enhancer and can restore full-length SMN survival motor neuron 2 SMN2)","volume":"97","author":"Hofmann","year":"2000","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1124\/molpharm.122.000529_bib36","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"},{"key":"10.1124\/molpharm.122.000529_bib37","doi-asserted-by":"crossref","first-page":"7183","DOI":"10.1038\/s41598-017-07468-2","article-title":"TIA1 is a gender-specific disease modifier of a mild mouse model of spinal muscular atrophy","volume":"7","author":"Howell","year":"2017","journal-title":"Sci Rep"},{"key":"10.1124\/molpharm.122.000529_bib38","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":"10.1124\/molpharm.122.000529_bib39","doi-asserted-by":"crossref","first-page":"20879","DOI":"10.1074\/jbc.M114.555284","article-title":"Activation of AMP-activated protein kinase regulates hippocampal neuronal pH by recruiting Na(+)\/H(+) exchanger NHE5 to the cell surface","volume":"289","author":"Jinadasa","year":"2014","journal-title":"J Biol Chem"},{"key":"10.1124\/molpharm.122.000529_bib40","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"},{"key":"10.1124\/molpharm.122.000529_bib41","doi-asserted-by":"crossref","first-page":"3426","DOI":"10.1073\/pnas.0700343104","article-title":"An intronic element contributes to splicing repression in spinal muscular atrophy","volume":"104","author":"Kashima","year":"2007","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1124\/molpharm.122.000529_bib42","first-page":"572","article-title":"TIA1 associated with perturbed splicing","volume":"34","author":"Klar","year":"2013","journal-title":"Hum Mutat"},{"key":"10.1124\/molpharm.122.000529_bib43","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/BF01871102","article-title":"Amiloride and its analogs as tools in the study of ion transport","volume":"105","author":"Kleyman","year":"1988","journal-title":"J Membr Biol"},{"key":"10.1124\/molpharm.122.000529_bib44","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1093\/bioinformatics\/btt703","article-title":"Causal analysis approaches in Ingenuity Pathway Analysis","volume":"30","author":"Kr\u00e4mer","year":"2014","journal-title":"Bioinformatics"},{"key":"10.1124\/molpharm.122.000529_bib45","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1007\/s10585-019-10001-6","article-title":"NHE5 regulates growth factor signaling, integrin trafficking, and degradation in glioma cells","volume":"36","author":"Kurata","year":"2019","journal-title":"Clin Exp Metastasis"},{"key":"10.1124\/molpharm.122.000529_bib46","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":"10.1124\/molpharm.122.000529_bib47","doi-asserted-by":"crossref","first-page":"468","DOI":"10.1111\/cns.12524","article-title":"Amiloride analogs as ASIC1a inhibitors","volume":"22","author":"Leng","year":"2016","journal-title":"CNS Neurosci Ther"},{"key":"10.1124\/molpharm.122.000529_bib48","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1186\/s12964-018-0266-6","article-title":"Regulation of the master regulator FOXM1 in cancer","volume":"16","author":"Liao","year":"2018","journal-title":"Cell Commun Signal"},{"key":"10.1124\/molpharm.122.000529_bib49","doi-asserted-by":"crossref","first-page":"1229","DOI":"10.1387\/ijdb.092976rl","article-title":"Fatty acid binding proteins in brain development and disease","volume":"54","author":"Liu","year":"2010","journal-title":"Int J Dev Biol"},{"key":"10.1124\/molpharm.122.000529_bib50","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1093\/hmg\/9.2.259","article-title":"An exonic enhancer is required for inclusion of an essential exon in the SMA-determining gene SMN","volume":"9","author":"Lorson","year":"2000","journal-title":"Hum Mol Genet"},{"key":"10.1124\/molpharm.122.000529_bib51","doi-asserted-by":"crossref","first-page":"6307","DOI":"10.1073\/pnas.96.11.6307","article-title":"SMN gene regulates splicing and is responsible for spinal muscular atrophy","volume":"96","author":"Lorson","year":"1999","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1124\/molpharm.122.000529_bib52","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0106818","article-title":"Transcriptome profiling of spinal muscular atrophy motor neurons derived from mouse embryonic stem cells","volume":"9","author":"Maeda","year":"2014","journal-title":"PLoS One"},{"key":"10.1124\/molpharm.122.000529_bib53","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1016\/S0223-5234(03)00100-4","article-title":"An overview of inhibitors of Na(+)\/H(+) exchanger","volume":"38","author":"Masereel","year":"2003","journal-title":"Eur J Med Chem"},{"key":"10.1124\/molpharm.122.000529_bib54","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-dose gene-replacement therapy for spinal muscular atrophy","volume":"377","author":"Mendell","year":"2017","journal-title":"N Engl J Med"},{"key":"10.1124\/molpharm.122.000529_bib55","doi-asserted-by":"crossref","first-page":"625","DOI":"10.1056\/NEJMoa1710504","article-title":"Nusinersen versus sham control in later-onset spinal muscular atrophy","volume":"378","author":"Mercuri","year":"2018","journal-title":"N Engl J Med"},{"key":"10.1124\/molpharm.122.000529_bib56","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1016\/j.nbd.2010.01.006","article-title":"Neuromuscular defects and breathing disorders in a new mouse model of spinal muscular atrophy","volume":"38","author":"Michaud","year":"2010","journal-title":"Neurobiol Dis"},{"key":"10.1124\/molpharm.122.000529_bib57","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1093\/hmg\/8.7.1177","article-title":"SMN1 SMN2","volume":"8","author":"Monani","year":"1999","journal-title":"Hum Mol Genet"},{"key":"10.1124\/molpharm.122.000529_bib58","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1093\/hmg\/9.3.333","article-title":"SMN2 Smn(-\/-) mice and results in a mouse with spinal muscular atrophy [published correction appears in Hum Mol Genet (2007) 16:2648]","volume":"9","author":"Monani","year":"2000","journal-title":"Hum Mol Genet"},{"key":"10.1124\/molpharm.122.000529_bib59","doi-asserted-by":"crossref","first-page":"688","DOI":"10.1126\/science.1250127","article-title":"Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy","volume":"345","author":"Naryshkin","year":"2014","journal-title":"Science"},{"key":"10.1124\/molpharm.122.000529_bib60","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1083\/jcb.201502059","article-title":"SAM68 is a physiological regulator of SMN2 splicing in spinal muscular atrophy","volume":"211","author":"Pagliarini","year":"2015","journal-title":"J Cell Biol"},{"key":"10.1124\/molpharm.122.000529_bib61","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1038\/nchembio.1837","article-title":"SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice","volume":"11","author":"Palacino","year":"2015","journal-title":"Nat Chem Biol"},{"key":"10.1124\/molpharm.122.000529_bib62","doi-asserted-by":"crossref","first-page":"409","DOI":"10.1136\/jmg.15.6.409","article-title":"Incidence, prevalence, and gene frequency studies of chronic childhood spinal muscular atrophy","volume":"15","author":"Pearn","year":"1978","journal-title":"J Med Genet"},{"key":"10.1124\/molpharm.122.000529_bib63","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"},{"key":"10.1124\/molpharm.122.000529_bib64","doi-asserted-by":"crossref","first-page":"e45","DOI":"10.1093\/nar\/29.9.e45","article-title":"A new mathematical model for relative quantification in real-time RT-PCR","volume":"29","author":"Pfaffl","year":"2001","journal-title":"Nucleic Acids Res"},{"key":"10.1124\/molpharm.122.000529_bib65","doi-asserted-by":"crossref","first-page":"527","DOI":"10.1146\/annurev.pharmtox.42.092001.143801","article-title":"The changing face of the Na+\/H+ exchanger, NHE1: structure, regulation, and cellular actions","volume":"42","author":"Putney","year":"2002","journal-title":"Annu Rev Pharmacol Toxicol"},{"key":"10.1124\/molpharm.122.000529_bib66","doi-asserted-by":"crossref","first-page":"6501","DOI":"10.1021\/acs.jmedchem.8b00741","article-title":"Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 ( SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)","volume":"61","author":"Ratni","year":"2018","journal-title":"J Med Chem"},{"key":"10.1124\/molpharm.122.000529_bib67","doi-asserted-by":"crossref","first-page":"6086","DOI":"10.1021\/acs.jmedchem.6b00459","article-title":"Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy","volume":"59","author":"Ratni","year":"2016","journal-title":"J Med Chem"},{"key":"10.1124\/molpharm.122.000529_bib68","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1007\/s00210-008-0364-x","article-title":"The Na+\/H+ exchanger modulates long-term potentiation in rat hippocampal slices","volume":"379","author":"R\u00f6nicke","year":"2009","journal-title":"Naunyn Schmiedebergs Arch Pharmacol"},{"key":"10.1124\/molpharm.122.000529_bib69","doi-asserted-by":"crossref","first-page":"1101","DOI":"10.1038\/nprot.2008.73","article-title":"Analyzing real-time PCR data by the comparative C(T) method","volume":"3","author":"Schmittgen","year":"2008","journal-title":"Nat Protoc"},{"key":"10.1124\/molpharm.122.000529_bib70","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1016\/0027-5107(86)90121-1","article-title":"Alzheimer disease fibroblasts are hypersensitive to the lethal effects of a DNA-damaging chemical","volume":"159","author":"Scudiero","year":"1986","journal-title":"Mutat Res"},{"key":"10.1124\/molpharm.122.000529_bib71","doi-asserted-by":"crossref","first-page":"18957","DOI":"10.1074\/jbc.M113.527341","article-title":"FABP3 protein promotes \u03b1-synuclein oligomerization associated with 1-methyl-1,2,3,6-tetrahydropiridine-induced neurotoxicity","volume":"289","author":"Shioda","year":"2014","journal-title":"J Biol Chem"},{"key":"10.1124\/molpharm.122.000529_bib72","doi-asserted-by":"crossref","first-page":"935","DOI":"10.1128\/MCB.00945-10","article-title":"TIA1 prevents skipping of a critical exon associated with spinal muscular atrophy","volume":"31","author":"Singh","year":"2011","journal-title":"Mol Cell Biol"},{"key":"10.1124\/molpharm.122.000529_bib73","doi-asserted-by":"crossref","first-page":"1476","DOI":"10.1038\/s41467-017-01559-4","article-title":"Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers","volume":"8","author":"Sivaramakrishnan","year":"2017","journal-title":"Nat Commun"},{"key":"10.1124\/molpharm.122.000529_bib74","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1002\/mgg3.141","article-title":"SMN1 SMN2 copy numbers in cell lines derived from patients with spinal muscular atrophy as measured by array digital PCR","volume":"3","author":"Stabley","year":"2015","journal-title":"Mol Genet Genomic Med"},{"key":"10.1124\/molpharm.122.000529_bib75","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1016\/j.nmd.2017.02.002","article-title":"Establishing a reference dataset for the authentication of spinal muscular atrophy cell lines using STR profiling and digital PCR","volume":"27","author":"Stabley","year":"2017","journal-title":"Neuromuscul Disord"},{"key":"10.1124\/molpharm.122.000529_bib76","doi-asserted-by":"crossref","first-page":"3219","DOI":"10.1172\/JCI121658","article-title":"Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain","volume":"128","author":"Sumner","year":"2018","journal-title":"J Clin Invest"},{"key":"10.1124\/molpharm.122.000529_bib77","doi-asserted-by":"crossref","first-page":"6302","DOI":"10.1074\/jbc.275.9.6302","article-title":"Kinetic and pharmacological properties of human brain Na(+)\/H(+) exchanger isoform 5 stably expressed in Chinese hamster ovary cells","volume":"275","author":"Szab\u00f3","year":"2000","journal-title":"J Biol Chem"},{"key":"10.1124\/molpharm.122.000529_bib78","doi-asserted-by":"crossref","first-page":"42623","DOI":"10.1074\/jbc.M206629200","article-title":"Clathrin-mediated endocytosis and recycling of the neuron-specific Na+\/H+ exchanger NHE5 isoform. Regulation by phosphatidylinositol 3\u2032-kinase and the actin cytoskeleton","volume":"277","author":"Sz\u00e1szi","year":"2002","journal-title":"J Biol Chem"},{"key":"10.1124\/molpharm.122.000529_bib79","doi-asserted-by":"crossref","first-page":"8691","DOI":"10.1523\/JNEUROSCI.0417-15.2015","article-title":"Disease mechanisms and therapeutic approaches in spinal muscular atrophy","volume":"35","author":"Tisdale","year":"2015","journal-title":"J Neurosci"},{"key":"10.1124\/molpharm.122.000529_bib80","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0081313","article-title":"Na+\/H+ exchangers induce autophagy in neurons and inhibit polyglutamine-induced aggregate formation","volume":"8","author":"Togashi","year":"2013","journal-title":"PLoS One"},{"key":"10.1124\/molpharm.122.000529_bib81","first-page":"E4604","article-title":"Mechanistic studies of a small-molecule modulator of SMN2 splicing","volume":"115","author":"Wang","year":"2018","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1124\/molpharm.122.000529_bib82","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0115205","article-title":"Targeting SR proteins improves SMN expression in spinal muscular atrophy cells","volume":"9","author":"Wee","year":"2014","journal-title":"PLoS One"},{"key":"10.1124\/molpharm.122.000529_bib83","doi-asserted-by":"crossref","first-page":"6070","DOI":"10.1021\/acs.jmedchem.6b00460","article-title":"Discovery and optimization of small molecule splicing modifiers of survival motor neuron 2 as a treatment for spinal muscular atrophy","volume":"59","author":"Woll","year":"2016","journal-title":"J Med Chem"},{"key":"10.1124\/molpharm.122.000529_bib84","doi-asserted-by":"crossref","first-page":"10411","DOI":"10.1523\/JNEUROSCI.1285-18.2018","article-title":"FABP3 in the anterior cingulate cortex modulates the methylation status of the glutamic acid decarboxylase67 promoter region","volume":"38","author":"Yamamoto","year":"2018","journal-title":"J Neurosci"},{"key":"10.1124\/molpharm.122.000529_bib85","doi-asserted-by":"crossref","first-page":"112","DOI":"10.1002\/biot.200700169","article-title":"Statistical methods for efficiency adjusted real-time PCR quantification","volume":"3","author":"Yuan","year":"2008","journal-title":"Biotechnol J"},{"key":"10.1124\/molpharm.122.000529_bib86","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1038\/nature13992","article-title":"A comparative encyclopedia of DNA elements in the mouse genome","volume":"515","author":"Yue","year":"2014","journal-title":"Nature"},{"key":"10.1124\/molpharm.122.000529_bib87","doi-asserted-by":"crossref","first-page":"26","DOI":"10.1002\/ana.21241","article-title":"5-(N-ethyl-N-isopropyl)-amiloride enhances SMN2 exon 7 inclusion and protein expression in spinal muscular atrophy cells","volume":"63","author":"Yuo","year":"2008","journal-title":"Ann Neurol"}],"container-title":["Molecular Pharmacology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0026895X2401246X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0026895X2401246X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/syndication.highwire.org\/content\/doi\/10.1124\/molpharm.122.000529","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,2,19]],"date-time":"2025-02-19T09:29:16Z","timestamp":1739957356000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0026895X2401246X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,8]]},"references-count":87,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2022,8]]}},"alternative-id":["S0026895X2401246X"],"URL":"https:\/\/doi.org\/10.1124\/molpharm.122.000529","relation":{},"ISSN":["0026-895X"],"issn-type":[{"type":"print","value":"0026-895X"}],"subject":[],"published":{"date-parts":[[2022,8]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Effects of Inhibitors of SLC9A-Type Sodium-Proton Exchangers on Survival Motor Neuron 2 (SMN2) mRNA Splicing and Expression","name":"articletitle","label":"Article Title"},{"value":"Molecular Pharmacology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1124\/molpharm.122.000529","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 by The American Society for Pharmacology and Experimental Therapeutics","name":"copyright","label":"Copyright"}]}}