{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,24]],"date-time":"2026-09-24T18:49:02Z","timestamp":1790275742179,"version":"4.1.0"},"reference-count":63,"publisher":"Springer Science and Business Media LLC","issue":"2","license":[{"start":{"date-parts":[[2009,2,8]],"date-time":"2009-02-08T00:00:00Z","timestamp":1234051200000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Curr Treat Options Neurol"],"published-print":{"date-parts":[[2009,3]]},"DOI":"10.1007\/s11940-009-0012-x","type":"journal-article","created":{"date-parts":[[2009,2,7]],"date-time":"2009-02-07T10:32:01Z","timestamp":1234002721000},"page":"90-101","source":"Crossref","is-referenced-by-count":22,"title":["Emerging treatment options for spinal muscular atrophy"],"prefix":"10.1007","volume":"11","author":[{"given":"Barrington G.","family":"Burnett","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas O.","family":"Crawford","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charlotte J.","family":"Sumner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2009,2,8]]},"reference":[{"key":"12_CR1","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1016\/0092-8674(95)90460-3","volume":"80","author":"S. Lefebvre","year":"1995","unstructured":"Lefebvre S, Burglen L, Reboullet S, et al.: Identification and characterization of a spinal muscular atrophy-determining gene. Cell 1995, 80:155\u2013165.","journal-title":"Cell"},{"key":"12_CR2","doi-asserted-by":"crossref","first-page":"6307","DOI":"10.1073\/pnas.96.11.6307","volume":"96","author":"C.L. Lorson","year":"1999","unstructured":"Lorson CL, Hahnen E, Androphy EJ, Wirth B: A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci U S A 1999, 96:6307\u20136311.","journal-title":"Proc Natl Acad Sci U S A"},{"key":"12_CR3","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1093\/hmg\/8.7.1177","volume":"8","author":"U.R. Monani","year":"1999","unstructured":"Monani UR, Lorson CL, Parsons DW, et al.: A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN2. Hum Mol Genet 1999, 8:1177\u20131183.","journal-title":"Hum Mol Genet"},{"key":"12_CR4","doi-asserted-by":"crossref","first-page":"845","DOI":"10.1093\/hmg\/ddi078","volume":"14","author":"T.T. Le","year":"2005","unstructured":"Le TT, Pham LT, Butchbach ME, 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 Genet 2005, 14:845\u2013857.","journal-title":"Hum Mol Genet"},{"key":"12_CR5","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1038\/ng0598-63","volume":"19","author":"C.L. Lorson","year":"1998","unstructured":"Lorson CL, Strasswimmer J, Yao JM, et al.: SMN oligomerization defect correlates with spinal muscular atrophy severity. Nat Genet 1998, 19:63\u201366.","journal-title":"Nat Genet"},{"key":"12_CR6","doi-asserted-by":"crossref","first-page":"615","DOI":"10.1016\/S0092-8674(00)81632-3","volume":"95","author":"L. Pellizzoni","year":"1998","unstructured":"Pellizzoni L, Kataoka N, Charroux B, et al.: A novel function for SMN, the spinal muscular atrophy disease gene product, in pre-mRNA splicing. Cell 1998, 95:615\u2013624.","journal-title":"Cell"},{"key":"12_CR7","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1002\/humu.20111","volume":"25","author":"Y. Sun","year":"2005","unstructured":"Sun Y, Grimmler M, Schwarzer V, et al.: Molecular and functional analysis of intragenic SMN1 mutations in patients with spinal muscular atrophy. Hum Mutat 2005, 25:64\u201371.","journal-title":"Hum Mutat"},{"key":"12_CR8","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1086\/338627","volume":"70","author":"M. Feldkotter","year":"2002","unstructured":"Feldkotter M, Schwarzer V, Wirth R, et al.: 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. Am J Hum Genet 2002, 70:358\u2013368.","journal-title":"Am J Hum Genet"},{"key":"12_CR9","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1126\/science.1155085","volume":"320","author":"G.E. Oprea","year":"2008","unstructured":"Oprea GE, Krober S, McWhorter ML, et al.: Plastin 3 is a protective modifier of autosomal recessive spinal muscular atrophy. Science 2008, 320:524\u2013527.","journal-title":"Science"},{"key":"12_CR10","doi-asserted-by":"crossref","first-page":"2133","DOI":"10.1093\/hmg\/8.12.2133","volume":"8","author":"I. Miguel-Aliaga","year":"1999","unstructured":"Miguel-Aliaga I, Culetto E, Walker DS, et al.: The Caenorhabditis elegans orthologue of the human gene responsible for spinal muscular atrophy is a maternal product critical for germline maturation and embryonic viability. Hum Mol Genet 1999, 8:2133\u20132143.","journal-title":"Hum Mol Genet"},{"key":"12_CR11","doi-asserted-by":"crossref","first-page":"23841","DOI":"10.1074\/jbc.M001441200","volume":"275","author":"S. Paushkin","year":"2000","unstructured":"Paushkin S, Charroux B, Abel L, et al.: The survival motor neuron protein of Schizosacharomyces pombe. Conservation of survival motor neuron interaction domains in divergent organisms. J Biol Chem 2000, 275:23841\u201323846.","journal-title":"J Biol Chem"},{"key":"12_CR12","doi-asserted-by":"crossref","first-page":"9920","DOI":"10.1073\/pnas.94.18.9920","volume":"94","author":"B. Schrank","year":"1997","unstructured":"Schrank B, Gotz R, Gunnersen JM, et al.: Inactivation of the survival motor neuron gene, a candidate gene for human spinal muscular atrophy, leads to massive cell death in early mouse embryos. Proc Natl Acad Sci U S A 1997, 94:9920\u20139925.","journal-title":"Proc Natl Acad Sci U S A"},{"key":"12_CR13","doi-asserted-by":"crossref","first-page":"3555","DOI":"10.1002\/j.1460-2075.1996.tb00725.x","volume":"15","author":"Q. Liu","year":"1996","unstructured":"Liu Q, Dreyfuss G: A novel nuclear structure containing the survival of motor neurons protein. Embo J 1996, 15:3555\u20133565.","journal-title":"Embo J"},{"key":"12_CR14","doi-asserted-by":"crossref","first-page":"885","DOI":"10.1016\/j.neuron.2005.12.001","volume":"48","author":"U.R. Monani","year":"2005","unstructured":"Monani UR: Spinal muscular atrophy: a deficiency in a ubiquitous protein, a motor neuron-specific disease. Neuron 2005, 48:885\u2013896.","journal-title":"Neuron"},{"key":"12_CR15","doi-asserted-by":"crossref","first-page":"305","DOI":"10.1016\/S0955-0674(02)00332-0","volume":"14","author":"S. Paushkin","year":"2002","unstructured":"Paushkin S, Gubitz AK, Massenet S, Dreyfuss G: The SMN complex, an assemblyosome of ribonucleoproteins. Curr Opin Cell Biol 2002, 14:305\u2013312.","journal-title":"Curr Opin Cell Biol"},{"key":"12_CR16","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1016\/j.cell.2008.03.031","volume":"133","author":"Z. Zhang","year":"2008","unstructured":"Zhang Z, Lotti F, Dittmar K, et al.: SMN deficiency causes tissue-specific perturbations in the repertoire of snRNAs and widespread defects in splicing. Cell 2008, 133:585\u2013600.","journal-title":"Cell"},{"key":"12_CR17","doi-asserted-by":"crossref","first-page":"e921","DOI":"10.1371\/journal.pone.0000921","volume":"2","author":"F. Gabanella","year":"2007","unstructured":"Gabanella F, Butchbach ME, Saieva L, et al.: Ribonucleoprotein assembly defects correlate with spinal muscular atrophy severity and preferentially affect a subset of spliceosomal snRNPs. PLoS ONE 2007, 2:e921.","journal-title":"PLoS ONE"},{"key":"12_CR18","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1016\/S0960-8966(06)80015-5","volume":"2","author":"T.L. Munsat","year":"1992","unstructured":"Munsat TL, Davies KE: International SMA consortium meeting. (26\u201328 June 1992, Bonn, Germany). Neuromuscul Disord 1992, 2:423\u2013428.","journal-title":"Neuromuscul Disord"},{"key":"12_CR19","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1177\/0884217503261125","volume":"33","author":"J.A. Markowitz","year":"2004","unstructured":"Markowitz JA, Tinkle MB, Fischbeck KH: Spinal muscular atrophy in the neonate. J Obstet Gynecol Neonatal Nurs 2004, 33:12\u201320.","journal-title":"J Obstet Gynecol Neonatal Nurs"},{"key":"12_CR20","doi-asserted-by":"crossref","first-page":"1027","DOI":"10.1177\/0883073807305788","volume":"22","author":"C.H. Wang","year":"2007","unstructured":"Wang CH, Finkel RS, Bertini ES, et al.: Consensus statement for standard of care in spinal muscular atrophy. J Child Neurol 2007, 22:1027\u20131049.","journal-title":"J Child Neurol"},{"key":"12_CR21","doi-asserted-by":"crossref","first-page":"1931","DOI":"10.1212\/01.wnl.0000290830.40544.b9","volume":"69","author":"M. Oskoui","year":"2007","unstructured":"Oskoui M, Levy G, Garland CJ, et al.: The changing natural history of spinal muscular atrophy type 1. Neurology 2007, 69:1931\u20131936.","journal-title":"Neurology"},{"key":"12_CR22","doi-asserted-by":"crossref","first-page":"244","DOI":"10.1016\/j.molcel.2008.06.004","volume":"31","author":"L. Wan","year":"2008","unstructured":"Wan L, Ottinger E, Cho S, et al.: Inactivation of the SMN complex by oxidative stress. Mol Cell 2008, 31:244\u2013254.","journal-title":"Mol Cell"},{"key":"12_CR23","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1016\/j.nmd.2004.04.004","volume":"14","author":"T.O. Crawford","year":"2004","unstructured":"Crawford TO: Concerns about the design of clinical trials for spinal muscular atrophy. Neuromuscul Disord 2004, 14:456\u2013460.","journal-title":"Neuromuscul Disord"},{"key":"12_CR24","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1038\/nrc1779","volume":"6","author":"S. Minucci","year":"2006","unstructured":"Minucci S, Pelicci PG: Histone deacetylase inhibitors and the promise of epigenetic (and more) treatments for cancer. Nat Rev Cancer 2006, 6:38\u201351.","journal-title":"Nat Rev Cancer"},{"key":"12_CR25","doi-asserted-by":"crossref","first-page":"1171","DOI":"10.1093\/hmg\/ddi130","volume":"14","author":"L.E. Kernochan","year":"2005","unstructured":"Kernochan LE, Russo ML, Woodling NS, et al.: The role of histone acetylation in SMN gene expression. Hum Mol Genet 2005, 14:1171\u20131182.","journal-title":"Hum Mol Genet"},{"key":"12_CR26","doi-asserted-by":"crossref","first-page":"2481","DOI":"10.1093\/hmg\/ddg256","volume":"12","author":"L. Brichta","year":"2003","unstructured":"Brichta L, Hofmann Y, Hahnen E, et al.: Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy. Hum Mol Genet 2003, 12:2481\u20132489.","journal-title":"Hum Mol Genet"},{"key":"12_CR27","doi-asserted-by":"crossref","first-page":"9808","DOI":"10.1073\/pnas.171105098","volume":"98","author":"J.G. Chang","year":"2001","unstructured":"Chang JG, Hsieh-Li HM, Jong YJ, et al.: Treatment of spinal muscular atrophy by sodium butyrate. Proc Natl Acad Sci U S A 2001, 98:9808\u20139813.","journal-title":"Proc Natl Acad Sci U S A"},{"key":"12_CR28","doi-asserted-by":"crossref","first-page":"647","DOI":"10.1002\/ana.10743","volume":"54","author":"C.J. Sumner","year":"2003","unstructured":"Sumner CJ, Huynh TN, Markowitz JA, et al.: Valproic acid increases SMN levels in spinal muscular atrophy patient cells. Ann Neurol 2003, 54:647\u2013654.","journal-title":"Ann Neurol"},{"key":"12_CR29","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1038\/sj.ejhg.5201102","volume":"12","author":"C. Andreassi","year":"2004","unstructured":"Andreassi C, Angelozzi C, Tiziano FD, et al.: Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy. Eur J Hum Genet 2004, 12:59\u201365.","journal-title":"Eur J Hum Genet"},{"key":"12_CR30","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1002\/ana.21449","volume":"64","author":"H.L. Narver","year":"2008","unstructured":"Narver HL, Kong L, Burnett BG, et al.: Sustained improvement of spinal muscular atrophy mice treated with trichostatin A plus nutrition. Ann Neurol 2008, 64:465\u2013470.","journal-title":"Ann Neurol"},{"key":"12_CR31","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1172\/JCI29562","volume":"117","author":"A.M. Avila","year":"2007","unstructured":"Avila AM, Burnett BG, Taye AA, et al.: Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy. J Clin Invest 2007, 117:659\u2013671.","journal-title":"J Clin Invest"},{"key":"12_CR32","unstructured":"Swoboda K: SMA CARNI-VAL TRIAL: Randomized double-blind placebo-controlled trial of L-carnitine and valproic acid in children with SMA type II. Presented at the 12th Annual International SMA Research Group Meeting, Boston, MA, June 19\u201321, 2008."},{"key":"12_CR33","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1212\/01.wnl.0000249142.82285.d6","volume":"68","author":"E. Mercuri","year":"2007","unstructured":"Mercuri E, Bertini E, Messina S, et al.: Randomized, double-blind, placebo-controlled trial of phenylbutyrate in spinal muscular atrophy. Neurology 2007, 68:51\u201355.","journal-title":"Neurology"},{"key":"12_CR34","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1002\/ana.20548","volume":"58","author":"S.M. Grzeschik","year":"2005","unstructured":"Grzeschik SM, Ganta M, Prior TW, et al.: Hydroxyurea enhances SMN2 gene expression in spinal muscular atrophy cells. Ann Neurol 2005, 58:194\u2013202.","journal-title":"Ann Neurol"},{"key":"12_CR35","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.jns.2007.11.012","volume":"268","author":"W.C. Liang","year":"2008","unstructured":"Liang WC, Yuo CY, Chang JG, et al.: The effect of hydroxyurea in spinal muscular atrophy cells and patients. J Neurol Sci 2008, 268:87\u201394.","journal-title":"J Neurol Sci"},{"key":"12_CR36","first-page":"103","volume":"73","author":"F. Wahl","year":"1999","unstructured":"Wahl F, Stutzmann JM: Neuroprotective effects of riluzole in neurotrauma models: a review. Acta Neurochir Suppl 1999, 73:103\u2013110.","journal-title":"Acta Neurochir Suppl"},{"key":"12_CR37","doi-asserted-by":"crossref","first-page":"432","DOI":"10.1002\/mus.10455","volume":"28","author":"H. Haddad","year":"2003","unstructured":"Haddad H, Cifuentes-Diaz C, Miroglio A, et al.: Riluzole attenuates spinal muscular atrophy disease progression in a mouse model. Muscle Nerve 2003, 28:432\u2013437.","journal-title":"Muscle Nerve"},{"key":"12_CR38","doi-asserted-by":"crossref","first-page":"1601","DOI":"10.1001\/archneur.60.11.1601","volume":"60","author":"B.S. Russman","year":"2003","unstructured":"Russman BS, Iannaccone ST, Samaha FJ: A phase 1 trial of riluzole in spinal muscular atrophy. Arch Neurol 2003, 60:1601\u20131603.","journal-title":"Arch Neurol"},{"key":"12_CR39","doi-asserted-by":"crossref","first-page":"1383","DOI":"10.1212\/WNL.47.6.1383","volume":"47","author":"R.G. Miller","year":"1996","unstructured":"Miller RG, Moore D, Young LA, et al.: Placebo-controlled trial of gabapentin in patients with amyotrophic lateral sclerosis. Neurology 1996, 47:1383\u20131388.","journal-title":"Neurology"},{"key":"12_CR40","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/S0920-1211(97)00084-3","volume":"29","author":"C.P. Taylor","year":"1998","unstructured":"Taylor CP, Gee NS, Su TZ, et al.: A summary of mechanistic hypotheses of gabapentin pharmacology. Epilepsy Res 1998, 29:233\u2013249.","journal-title":"Epilepsy Res"},{"key":"12_CR41","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1016\/S0022-510X(01)00632-3","volume":"191","author":"R.G. Miller","year":"2001","unstructured":"Miller RG, Moore DH, Dronsky V, et al.: A placebo-controlled trial of gabapentin in spinal muscular atrophy. J Neurol Sci 2001, 191:127\u2013131.","journal-title":"J Neurol Sci"},{"key":"12_CR42","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1177\/08830738030180080501","volume":"18","author":"L. Merlini","year":"2003","unstructured":"Merlini L, Solari A, Vita G, et al.: Role of gabapentin in spinal muscular atrophy: results of a multicenter, randomized Italian study. J Child Neurol 2003, 18:537\u2013541.","journal-title":"J Child Neurol"},{"key":"12_CR43","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1136\/jmg.2007.051177","volume":"45","author":"C. Angelozzi","year":"2008","unstructured":"Angelozzi C, Borgo F, Tiziano FD, et al.: Salbutamol increases SMN mRNA and protein levels in spinal muscular atrophy cells. J Med Genet 2008, 45:29\u201331.","journal-title":"J Med Genet"},{"key":"12_CR44","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1212\/WNL.59.4.609","volume":"59","author":"M. Kinali","year":"2002","unstructured":"Kinali M, Mercuri E, Main M, et al.: Pilot trial of albuterol in spinal muscular atrophy. Neurology 2002, 59:609\u2013610.","journal-title":"Neurology"},{"key":"12_CR45","doi-asserted-by":"crossref","first-page":"536","DOI":"10.1016\/j.nmd.2008.05.004","volume":"18","author":"M. Pane","year":"2008","unstructured":"Pane M, Staccioli S, Messina S, et al.: Daily salbutamol in young patients with SMA type II. Neuromuscul Disord 2008, 18:536\u2013540.","journal-title":"Neuromuscul Disord"},{"key":"12_CR46","doi-asserted-by":"crossref","first-page":"1489","DOI":"10.1016\/j.chembiol.2004.08.024","volume":"11","author":"M.R. Lunn","year":"2004","unstructured":"Lunn MR, Root DE, Martino AM, et al.: Indoprofen upregulates the survival motor neuron protein through a cyclooxygenase-independent mechanism. Chem Biol 2004, 11:1489\u20131493.","journal-title":"Chem Biol"},{"key":"12_CR47","doi-asserted-by":"crossref","first-page":"2003","DOI":"10.1093\/hmg\/ddi205","volume":"14","author":"J. Jarecki","year":"2005","unstructured":"Jarecki J, Chen X, Bernardino A, et al.: Diverse small-molecule modulators of SMN expression found by high-throughput compound screening: early leads towards a therapeutic for spinal muscular atrophy. Hum Mol Genet 2005, 14:2003\u20132018.","journal-title":"Hum Mol Genet"},{"key":"12_CR48","doi-asserted-by":"crossref","first-page":"449","DOI":"10.1021\/jm061475p","volume":"51","author":"J. Thurmond","year":"2008","unstructured":"Thurmond J, Butchbach ME, Palomo M, et al.: Synthesis and biological evaluation of novel 2,4-diaminoquinazoline derivatives as SMN2 promoter activators for the potential treatment of spinal muscular atrophy. J Med Chem 2008, 51:449\u2013469.","journal-title":"J Med Chem"},{"key":"12_CR49","doi-asserted-by":"crossref","first-page":"1471","DOI":"10.1038\/sj.mt.6300222","volume":"15","author":"T.H. Coady","year":"2007","unstructured":"Coady TH, Shababi M, Tullis GE, Lorson CL: Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing. Mol Ther 2007, 15:1471\u20131478.","journal-title":"Mol Ther"},{"key":"12_CR50","doi-asserted-by":"crossref","first-page":"2841","DOI":"10.1093\/hmg\/10.24.2841","volume":"10","author":"C. Andreassi","year":"2001","unstructured":"Andreassi C, Jarecki J, Zhou J, et al.: Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients. Hum Mol Genet 2001, 10:2841\u20132849.","journal-title":"Hum Mol Genet"},{"key":"12_CR51","doi-asserted-by":"crossref","first-page":"45476","DOI":"10.1074\/jbc.M107632200","volume":"276","author":"S.R. Lim","year":"2001","unstructured":"Lim SR, Hertel KJ: Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3\u2032 splice site pairing. J Biol Chem 2001, 276:45476\u201345483.","journal-title":"J Biol Chem"},{"key":"12_CR52","doi-asserted-by":"crossref","first-page":"1013","DOI":"10.1016\/j.ymthe.2005.08.022","volume":"12","author":"C. Madocsai","year":"2005","unstructured":"Madocsai C, Lim SR, Geib T, et al.: Correction of SMN2 pre-mRNA splicing by antisense U7 small nuclear RNAs. Mol Ther 2005, 12:1013\u20131022.","journal-title":"Mol Ther"},{"key":"12_CR53","doi-asserted-by":"crossref","first-page":"869","DOI":"10.1089\/hum.2005.16.869","volume":"16","author":"F. Sangiuolo","year":"2005","unstructured":"Sangiuolo F, Filareto A, Spitalieri P, et al.: In vitro restoration of functional SMN protein in human trophoblast cells affected by spinal muscular atrophy by small fragment homologous replacement. Hum Gene Ther 2005, 16:869\u2013880.","journal-title":"Hum Gene Ther"},{"key":"12_CR54","doi-asserted-by":"crossref","first-page":"23271","DOI":"10.1074\/jbc.M200851200","volume":"277","author":"H. Miyajima","year":"2002","unstructured":"Miyajima H, Miyaso H, Okumura M, et al.: Identification of a cis-acting element for the regulation of SMN exon 7 splicing. J Biol Chem 2002, 277:23271\u201323277.","journal-title":"J Biol Chem"},{"key":"12_CR55","doi-asserted-by":"crossref","first-page":"4114","DOI":"10.1073\/pnas.0633863100","volume":"100","author":"L.A. Skordis","year":"2003","unstructured":"Skordis LA, Dunckley MG, Yue B, et al.: Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts. Proc Natl Acad Sci U S A 2003, 100:4114\u20134119.","journal-title":"Proc Natl Acad Sci U S A"},{"key":"12_CR56","doi-asserted-by":"crossref","first-page":"54","DOI":"10.1016\/j.ymthe.2006.01.012","volume":"14","author":"T. Baughan","year":"2006","unstructured":"Baughan T, Shababi M, Coady TH, et al.: Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector. Mol Ther 2006, 14:54\u201362.","journal-title":"Mol Ther"},{"key":"12_CR57","unstructured":"Krainer A: SMN anti-sense. Presentation at the 12th Annual International SMA Research Group Meeting, Boston, MA, June 19\u201321, 2008."},{"key":"12_CR58","doi-asserted-by":"crossref","first-page":"1199","DOI":"10.1093\/hmg\/ddi131","volume":"14","author":"E.C. Wolstencroft","year":"2005","unstructured":"Wolstencroft EC, Mattis V, Bajer AA, et al.: A nonsequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels. Hum Mol Genet 2005, 14:1199\u20131210.","journal-title":"Hum Mol Genet"},{"key":"12_CR59","doi-asserted-by":"crossref","first-page":"1726","DOI":"10.1172\/JCI22922","volume":"114","author":"M. Azzouz","year":"2004","unstructured":"Azzouz M, Le T, Ralph GS, et al.: Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. J Clin Invest 2004, 114:1726\u20131731.","journal-title":"J Clin Invest"},{"key":"12_CR60","doi-asserted-by":"crossref","first-page":"1233","DOI":"10.1093\/hmg\/ddg143","volume":"12","author":"J.C. Lesbordes","year":"2003","unstructured":"Lesbordes JC, Cifuentes-Diaz C, Miroglio A, et al.: Therapeutic benefits of cardiotrophin-1 gene transfer in a mouse model of spinal muscular atrophy. Hum Mol Genet 2003, 12:1233\u20131239.","journal-title":"Hum Mol Genet"},{"key":"12_CR61","doi-asserted-by":"crossref","first-page":"5131","DOI":"10.1523\/JNEUROSCI.23-12-05131.2003","volume":"23","author":"D.A. Kerr","year":"2003","unstructured":"Kerr DA, Llado J, Shamblott MJ, et al.: Human embryonic germ cell derivatives facilitate motor recovery of rats with diffuse motor neuron injury. J Neurosci 2003, 23:5131\u20135140.","journal-title":"J Neurosci"},{"key":"12_CR62","doi-asserted-by":"crossref","first-page":"93","DOI":"10.1159\/000082130","volume":"26","author":"C.M. Morshead","year":"2004","unstructured":"Morshead CM: Adult neural stem cells: attempting to solve the identity crisis. Dev Neurosci 2004, 26:93\u2013100.","journal-title":"Dev Neurosci"},{"key":"12_CR63","doi-asserted-by":"crossref","first-page":"3316","DOI":"10.1172\/JCI35432","volume":"118","author":"S. Corti","year":"2008","unstructured":"Corti S, Nizzardo M, Nardini M, et al.: Neural stem cell transplantation can ameliorate the phenotype of a mouse model of spinal muscular atrophy. J Clin Invest 2008, 118:3316\u20133330.","journal-title":"J Clin Invest"}],"container-title":["Current Treatment Options in Neurology"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s11940-009-0012-x.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s11940-009-0012-x\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s11940-009-0012-x","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,6,2]],"date-time":"2019-06-02T00:56:54Z","timestamp":1559437014000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/s11940-009-0012-x"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2009,2,8]]},"references-count":63,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2009,3]]}},"alternative-id":["12"],"URL":"https:\/\/doi.org\/10.1007\/s11940-009-0012-x","relation":{},"ISSN":["1092-8480","1534-3138"],"issn-type":[{"value":"1092-8480","type":"print"},{"value":"1534-3138","type":"electronic"}],"subject":[],"published":{"date-parts":[[2009,2,8]]}}}