{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,17]],"date-time":"2025-12-17T12:55:04Z","timestamp":1765976104194},"reference-count":69,"publisher":"The Endocrine Society","issue":"10","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2016,10,1]]},"DOI":"10.1210\/en.2015-2038","type":"journal-article","created":{"date-parts":[[2016,6,2]],"date-time":"2016-06-02T17:19:44Z","timestamp":1464887984000},"page":"3972-3982","source":"Crossref","is-referenced-by-count":8,"title":["Rescue of Isolated GH Deficiency Type II (IGHD II) via Pharmacologic Modulation of GH-1 Splicing"],"prefix":"10.1210","volume":"157","author":[{"given":"Maria Consolata","family":"Miletta","sequence":"first","affiliation":[{"name":"Division of Pediatric Endocrinology, Diabetology and Metabolism, Department of Pediatrics and Department of Clinical Research, Inselspital, Bern University Hospital, University of Bern, Bern, CH-3010 Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vibor","family":"Petkovic","sequence":"additional","affiliation":[{"name":"Division of Pediatric Endocrinology, Diabetology and Metabolism, Department of Pediatrics and Department of Clinical Research, Inselspital, Bern University Hospital, University of Bern, Bern, CH-3010 Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andr\u00e9e","family":"Ebl\u00e9","sequence":"additional","affiliation":[{"name":"Division of Pediatric Endocrinology, Diabetology and Metabolism, Department of Pediatrics and Department of Clinical Research, Inselspital, Bern University Hospital, University of Bern, Bern, CH-3010 Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christa E.","family":"Fl\u00fcck","sequence":"additional","affiliation":[{"name":"Division of Pediatric Endocrinology, Diabetology and Metabolism, Department of Pediatrics and Department of Clinical Research, Inselspital, Bern University Hospital, University of Bern, Bern, CH-3010 Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Primus-E.","family":"Mullis","sequence":"additional","affiliation":[{"name":"Division of Pediatric Endocrinology, Diabetology and Metabolism, Department of Pediatrics and Department of Clinical Research, Inselspital, Bern University Hospital, University of Bern, Bern, CH-3010 Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"80","published-online":{"date-parts":[[2016,6,2]]},"reference":[{"issue":"4","key":"2019041121351321100_B1","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1038\/nrg775","article-title":"Listening to silence and understanding nonsense: exonic mutations that affect splicing","volume":"3","author":"Cartegni","year":"2002","journal-title":"Nat Rev Genet"},{"key":"2019041121351321100_B2","doi-asserted-by":"crossref","first-page":"291","DOI":"10.1146\/annurev.biochem.72.121801.161720","article-title":"Mechanisms of alternative pre-messenger RNA splicing","volume":"72","author":"Black","year":"2003","journal-title":"Annu Rev Biochem"},{"issue":"8","key":"2019041121351321100_B3","doi-asserted-by":"crossref","first-page":"381","DOI":"10.1016\/S0968-0004(00)01604-2","article-title":"Alternative pre-mRNA splicing: the logic of combinatorial control","volume":"25","author":"Smith","year":"2000","journal-title":"Trends Biochem Sci"},{"issue":"7","key":"2019041121351321100_B4","first-page":"663","article-title":"The superfamily of arginine\/serine-rich splicing factors","volume":"1","author":"Fu","year":"1995","journal-title":"RNA"},{"issue":"3","key":"2019041121351321100_B5","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1016\/S0955-0674(00)00212-X","article-title":"Pre-mRNA splicing in the new millennium","volume":"13","author":"Hastings","year":"2001","journal-title":"Curr Opin Cell Biol"},{"issue":"3","key":"2019041121351321100_B6","first-page":"335","article-title":"Overexpression of the SR proteins ASF\/SF2 and SC35 influences alternative splicing in vivo in diverse ways","volume":"1","author":"Wang","year":"1995","journal-title":"RNA"},{"issue":"3","key":"2019041121351321100_B7","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1016\/S0955-0674(99)80050-7","article-title":"Determinants of SR protein specificity","volume":"11","author":"Tacke","year":"1999","journal-title":"Curr Opin Cell Biol"},{"issue":"5179","key":"2019041121351321100_B8","doi-asserted-by":"crossref","first-page":"1706","DOI":"10.1126\/science.8085156","article-title":"Regulation of alternative splicing in vivo by overexpression of antagonistic splicing factors","volume":"265","author":"C\u00e1ceres","year":"1994","journal-title":"Science"},{"issue":"15","key":"2019041121351321100_B9","doi-asserted-by":"crossref","first-page":"3719","DOI":"10.1093\/nar\/9.15.3719","article-title":"Human growth hormone DNA sequence and mRNA structure: possible alternative splicing","volume":"9","author":"DeNoto","year":"1981","journal-title":"Nucleic Acids Res"},{"issue":"1\u20132","key":"2019041121351321100_B10","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1016\/S0303-7207(00)00202-1","article-title":"GH induced lipolysis stimulation in 3T3-L1 adipocytes stably expressing hGHR: analysis on signaling pathway and activity of 20K hGH","volume":"162","author":"Asada","year":"2000","journal-title":"Mol Cell Endocrinol"},{"issue":"1","key":"2019041121351321100_B11","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1016\/0167-4781(88)90062-0","article-title":"Molecular cloning of cDNA encoding 20 kDa variant human growth hormone and the alternative splicing mechanism","volume":"949","author":"Masuda","year":"1988","journal-title":"Biochim Biophys Acta"},{"issue":"16","key":"2019041121351321100_B12","doi-asserted-by":"crossref","first-page":"6331","DOI":"10.1093\/nar\/15.16.6331","article-title":"A new natural hGH variant\u201317.5 kd\u2013produced by alternative splicing. An additional consensus sequence which might play a role in branchpoint selection","volume":"15","author":"Lecomte","year":"1987","journal-title":"Nucleic Acids Res"},{"issue":"1","key":"2019041121351321100_B13","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1006\/jmbi.1994.1135","article-title":"The crystal structure of affinity-matured human growth hormone at 2 A resolution","volume":"236","author":"Ultsch","year":"1994","journal-title":"J Mol Biol"},{"issue":"pt 20","key":"2019041121351321100_B14","doi-asserted-by":"crossref","first-page":"3685","DOI":"10.1242\/jcs.114.20.3685","article-title":"Misfolded growth hormone causes fragmentation of the Golgi apparatus and disrupts endoplasmic reticulum-to-Golgi traffic","volume":"114","author":"Graves","year":"2001","journal-title":"J Cell Sci"},{"issue":"6","key":"2019041121351321100_B15","first-page":"2134","article-title":"Inhibition of growth hormone (GH) secretion by a mutant GH-I gene product in neuroendocrine cells containing secretory granules: an implication for isolated GH deficiency inherited in an autosomal dominant manner","volume":"84","author":"Hayashi","year":"1999","journal-title":"J Clin Endocrinol Metab"},{"issue":"3","key":"2019041121351321100_B16","doi-asserted-by":"crossref","first-page":"883","DOI":"10.1210\/endo.141.3.7380","article-title":"Autosomal dominant growth hormone (GH) deficiency type II: the Del32\u201371-GH deletion mutant suppresses secretion of wild-type GH","volume":"141","author":"Lee","year":"2000","journal-title":"Endocrinology"},{"issue":"2","key":"2019041121351321100_B17","doi-asserted-by":"crossref","first-page":"720","DOI":"10.1210\/en.2002-220847","article-title":"Autosomal dominant growth hormone deficiency disrupts secretory vesicles in vitro and in vivo in transgenic mice","volume":"144","author":"McGuinness","year":"2003","journal-title":"Endocrinology"},{"issue":"12","key":"2019041121351321100_B18","doi-asserted-by":"crossref","first-page":"4728","DOI":"10.1210\/jc.2009-0746","article-title":"A molecular basis for variation in clinical severity of isolated growth hormone deficiency type II","volume":"94","author":"Hamid","year":"2009","journal-title":"J Clin Endocrinol Metab"},{"issue":"4","key":"2019041121351321100_B19","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1177\/0009922806293924","article-title":"Twenty years of recombinant human growth hormone in children: relevance to pediatric care providers","volume":"46","author":"Hardin","year":"2007","journal-title":"Clin Pediatr (Phila)"},{"issue":"2","key":"2019041121351321100_B20","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1530\/eje.0.1490091","article-title":"Intrinsic imperfections of endocrine replacement therapy","volume":"149","author":"Romijn","year":"2003","journal-title":"Eur J Endocrinol"},{"issue":"4","key":"2019041121351321100_B21","doi-asserted-by":"crossref","first-page":"2089","DOI":"10.1210\/jc.2004-1280","article-title":"Isolated autosomal dominant growth hormone deficiency: an evolving pituitary deficit? A multicenter follow-up study","volume":"90","author":"Mullis","year":"2005","journal-title":"J Clin Endocrinol Metab"},{"issue":"6","key":"2019041121351321100_B22","doi-asserted-by":"crossref","first-page":"2988","DOI":"10.1210\/en.2003-1724","article-title":"GH1 splicing is regulated by multiple enhancers whose mutation produces a dominant-negative GH isoform that can be degraded by allele-specific small interfering RNA (siRNA)","volume":"145","author":"Ryther","year":"2004","journal-title":"Endocrinology"},{"issue":"9","key":"2019041121351321100_B23","doi-asserted-by":"crossref","first-page":"4400","DOI":"10.1210\/en.2010-0196","article-title":"Isolated GH deficiency type II: knockdown of the harmful \u03943GH recovers wt-GH secretion in rat tumor pituitary cells","volume":"151","author":"Lochmatter","year":"2010","journal-title":"Endocrinology"},{"issue":"2","key":"2019041121351321100_B24","doi-asserted-by":"crossref","first-page":"580","DOI":"10.1210\/en.2007-1360","article-title":"Rescue of pituitary function in a mouse model of isolated growth hormone deficiency type II by RNA interference","volume":"149","author":"Shariat","year":"2008","journal-title":"Endocrinology"},{"issue":"pt 3","key":"2019041121351321100_B25","doi-asserted-by":"crossref","first-page":"483","DOI":"10.1042\/BST0360483","article-title":"Substances that can change alternative splice-site selection","volume":"36","author":"Sumanasekera","year":"2008","journal-title":"Biochem Soc Trans"},{"issue":"2","key":"2019041121351321100_B26","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1177\/1087057112459901","article-title":"A high-throughput assay to identify small-molecule modulators of alternative pre-mRNA splicing","volume":"18","author":"Arslan","year":"2013","journal-title":"J Biomol Screen"},{"issue":"19","key":"2019041121351321100_B27","doi-asserted-by":"crossref","first-page":"2481","DOI":"10.1093\/hmg\/ddg256","article-title":"Valproic acid increases the SMN2 protein level: a well-known drug as a potential therapy for spinal muscular atrophy","volume":"12","author":"Brichta","year":"2003","journal-title":"Hum Mol Genet"},{"issue":"17","key":"2019041121351321100_B28","doi-asserted-by":"crossref","first-page":"9808","DOI":"10.1073\/pnas.171105098","article-title":"Treatment of spinal muscular atrophy by sodium butyrate","volume":"98","author":"Chang","year":"2001","journal-title":"Proc Natl Acad Sci USA"},{"issue":"24","key":"2019041121351321100_B29","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"},{"issue":"1","key":"2019041121351321100_B30","doi-asserted-by":"crossref","first-page":"101","DOI":"10.1007\/s00439-006-0186-1","article-title":"The benzamide M344, a novel histone deacetylase inhibitor, significantly increases SMN2 RNA\/protein levels in spinal muscular atrophy cells","volume":"120","author":"Riessland","year":"2006","journal-title":"Hum Genet"},{"issue":"3","key":"2019041121351321100_B31","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1111\/j.1752-8062.2011.00290.x","article-title":"Pharmacologic correction of dominant-negative GH1 deficiency causing mutations","volume":"4","author":"Poling","year":"2011","journal-title":"Clin Transl Sci"},{"issue":"2","key":"2019041121351321100_B32","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1210\/jc.2002-020269","article-title":"A novel IVS2\u20132A>T splicing mutation in the GH-1 gene in familial isolated growth hormone deficiency type II in the spectrum of other splicing mutations in the Russian population","volume":"88","author":"Fofanova","year":"2003","journal-title":"J Clin Endocrinol Metab"},{"issue":"5","key":"2019041121351321100_B33","first-page":"1261","article-title":"Familial growth hormone deficiency: a model of dominant and recessive mutations affecting a monomeric protein","volume":"79","author":"Cogan","year":"1994","journal-title":"J Clin Endocrinol Metab"},{"issue":"6","key":"2019041121351321100_B34","doi-asserted-by":"crossref","first-page":"909","DOI":"10.1093\/hmg\/6.6.909","article-title":"A novel mechanism of aberrant pre-mRNA splicing in humans","volume":"6","author":"Cogan","year":"1997","journal-title":"Hum Mol Genet"},{"issue":"9","key":"2019041121351321100_B35","doi-asserted-by":"crossref","first-page":"1491","DOI":"10.1093\/hmg\/7.9.1491","article-title":"Characterization of an intron splice enhancer that regulates alternative splicing of human GH pre-mRNA","volume":"7","author":"McCarthy","year":"1998","journal-title":"Hum Mol Genet"},{"issue":"2","key":"2019041121351321100_B36","doi-asserted-by":"crossref","first-page":"275","DOI":"10.1016\/0014-4827(73)90445-X","article-title":"Intracellular location of newly synthesized growth hormone","volume":"79","author":"Bancroft","year":"1973","journal-title":"Exp Cell Res"},{"issue":"2","key":"2019041121351321100_B37","doi-asserted-by":"crossref","first-page":"847","DOI":"10.1210\/jcem.87.2.8236","article-title":"An exon splice enhancer mutation causes autosomal dominant GH deficiency","volume":"87","author":"Moseley","year":"2002","journal-title":"J Clin Endocrinol Metab"},{"issue":"1","key":"2019041121351321100_B38","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1210\/en.2006-0772","article-title":"Isolated autosomal dominant growth hormone deficiency: stimulating mutant GH-1 gene expression drives GH-1 splice-site selection, cell proliferation, and apoptosis","volume":"148","author":"Salemi","year":"2007","journal-title":"Endocrinology"},{"issue":"6","key":"2019041121351321100_B39","doi-asserted-by":"crossref","first-page":"2650","DOI":"10.1210\/en.2009-1280","article-title":"Growth hormone (GH)-releasing hormone increases the expression of the dominant-negative GH isoform in cases of isolated GH deficiency due to GH splice-site mutations","volume":"151","author":"Petkovic","year":"2010","journal-title":"Endocrinology"},{"issue":"3","key":"2019041121351321100_B40","doi-asserted-by":"crossref","first-page":"1005","DOI":"10.1210\/jc.2007-1918","article-title":"A functional common polymorphism in the vitamin D-responsive element of the GH1 promoter contributes to isolated growth hormone deficiency","volume":"93","author":"Giordano","year":"2008","journal-title":"J Clin Endocrinol Metab"},{"issue":"5","key":"2019041121351321100_B41","doi-asserted-by":"crossref","first-page":"896","DOI":"10.1111\/j.1474-9726.2011.00727.x","article-title":"Alternative splicing factor or splicing factor-2 plays a key role in intron retention of the endoglin gene during endothelial senescence","volume":"10","author":"Blanco","year":"2011","journal-title":"Aging Cell"},{"issue":"2","key":"2019041121351321100_B42","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1007\/s00439-003-0949-x","article-title":"Disruption of exon definition produces a dominant-negative growth hormone isoform that causes somatotroph death and IGHD II","volume":"113","author":"Ryther","year":"2003","journal-title":"Hum Genet"},{"issue":"4","key":"2019041121351321100_B43","doi-asserted-by":"crossref","first-page":"402","DOI":"10.1006\/meth.2001.1262","article-title":"Analysis of relative gene expression data using real-time quantitative PCR and the 2(\u2212\u0394\u0394C(T)) method","volume":"25","author":"Livak","year":"2001","journal-title":"Methods"},{"issue":"17","key":"2019041121351321100_B44","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"},{"issue":"17","key":"2019041121351321100_B45","doi-asserted-by":"crossref","first-page":"9004","DOI":"10.1073\/pnas.93.17.9004","article-title":"A human homologue of the Drosophila sex determination factor transformer-2 has conserved splicing regulatory functions","volume":"93","author":"Dauwalder","year":"1996","journal-title":"Proc Natl Acad Sci USA"},{"issue":"35","key":"2019041121351321100_B46","doi-asserted-by":"crossref","first-page":"23619","DOI":"10.1074\/jbc.M710175200","article-title":"Growth hormone deficiency and splicing fidelity: two serine\/arginine-rich proteins, ASF\/SF2 and SC35, act antagonistically","volume":"283","author":"Solis","year":"2008","journal-title":"J Biol Chem"},{"issue":"11","key":"2019041121351321100_B47","doi-asserted-by":"crossref","first-page":"1071","DOI":"10.1038\/sj.embor.7400273","article-title":"Restoration of the cystic fibrosis transmembrane conductance regulator function by splicing modulation","volume":"5","author":"Nissim-Rafinia","year":"2004","journal-title":"EMBO Rep"},{"issue":"7","key":"2019041121351321100_B48","doi-asserted-by":"crossref","first-page":"1158","DOI":"10.1101\/gad.4.7.1158","article-title":"Purification and characterization of pre-mRNA splicing factor SF2 from HeLa cells","volume":"4","author":"Krainer","year":"1990","journal-title":"Genes Dev"},{"issue":"4","key":"2019041121351321100_B49","first-page":"1247","article-title":"Screening for growth hormone (GH) gene splice-site mutations in sporadic cases with severe isolated GH deficiency using ectopic transcript analysis","volume":"80","author":"Binder","year":"1995","journal-title":"J Clin Endocrinol Metab"},{"issue":"12","key":"2019041121351321100_B50","first-page":"3591","article-title":"A recurring dominant negative mutation causes autosomal dominant growth hormone deficiency\u2013a clinical research center study","volume":"80","author":"Cogan","year":"1995","journal-title":"J Clin Endocrinol Metab"},{"key":"2019041121351321100_B51","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1002\/humu.10168","article-title":"Novel mutations of the growth hormone 1 (GH1) gene disclosed by modulation of the clinical selection criteria for individuals with short stature","volume":"21","author":"Millar","year":"2003","journal-title":"Hum Mutat"},{"issue":"6","key":"2019041121351321100_B52","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1530\/eje.0.1470755","article-title":"Isolated GH deficiency (IGHD) type II: imaging of the pituitary gland by magnetic resonance reveals characteristic differences in comparison with severe IGHD of unknown origin","volume":"147","author":"Binder","year":"2002","journal-title":"Eur J Endocrinol"},{"issue":"11","key":"2019041121351321100_B53","doi-asserted-by":"crossref","first-page":"4387","DOI":"10.1210\/jc.2007-0684","article-title":"Variable phenotypes in familial isolated growth hormone deficiency caused by a G6664A mutation in the GH-1 gene","volume":"92","author":"Hess","year":"2007","journal-title":"J Clin Endocrinol Metab"},{"issue":"9","key":"2019041121351321100_B54","doi-asserted-by":"crossref","first-page":"3191","DOI":"10.1210\/jc.2008-2783","article-title":"Expanding the spectrum of mutations in GH1 and GHRHR: genetic screening in a large cohort of patients with congenital isolated growth hormone deficiency","volume":"94","author":"Alatzoglou","year":"2009","journal-title":"J Clin Endocrinol Metab"},{"issue":"13","key":"2019041121351321100_B55","doi-asserted-by":"crossref","first-page":"1569","DOI":"10.1101\/gad.10.13.1569","article-title":"SR proteins and splicing control","volume":"10","author":"Manley","year":"1996","journal-title":"Genes Dev"},{"issue":"2","key":"2019041121351321100_B56","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1210\/jc.2009-1247","article-title":"Growth hormone (GH) deficiency type II: a novel GH-1 gene mutation (GH-R178H) affecting secretion and action","volume":"95","author":"Petkovic","year":"2010","journal-title":"J Clin Endocrinol Metab"},{"issue":"9","key":"2019041121351321100_B57","doi-asserted-by":"crossref","first-page":"1283","DOI":"10.1016\/0277-5379(87)90109-X","article-title":"Clinical pharmacology of sodium butyrate in patients with acute leukemia","volume":"23","author":"Miller","year":"1987","journal-title":"Eur J Cancer Clin Oncol"},{"issue":"1","key":"2019041121351321100_B58","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1002\/wrna.1140","article-title":"Exon and intron definition in pre-mRNA splicing","volume":"4","author":"De Conti","year":"2013","journal-title":"Wiley Interdiscip Rev RNA"},{"issue":"3","key":"2019041121351321100_B59","doi-asserted-by":"crossref","first-page":"e59916","DOI":"10.1371\/journal.pone.0059916","article-title":"Interplay between DMD point mutations and splicing signals in dystrophinopathy phenotypes","volume":"8","author":"Juan-Mateu","year":"2013","journal-title":"PLoS One"},{"issue":"24","key":"2019041121351321100_B60","doi-asserted-by":"crossref","first-page":"13797","DOI":"10.1073\/pnas.93.24.13797","article-title":"Proteasome-dependent endoplasmic reticulum-associated protein degradation: an unconventional route to a familiar fate","volume":"93","author":"Werner","year":"1996","journal-title":"Proc Natl Acad Sci USA"},{"key":"2019041121351321100_B61","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1146\/annurev.med.50.1.57","article-title":"The ubiquitin-proteasome pathway and pathogenesis of human diseases","volume":"50","author":"Schwartz","year":"1999","journal-title":"Annu Rev Med"},{"issue":"7","key":"2019041121351321100_B62","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1002\/ajh.23725","article-title":"A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease","volume":"89","author":"Reid","year":"2014","journal-title":"Am J Hematol"},{"issue":"12","key":"2019041121351321100_B63","doi-asserted-by":"crossref","first-page":"1956","DOI":"10.1182\/blood-2013-11-538470","article-title":"A phase 2 trial of HQK-1001 in HbE-\u03b2 thalassemia demonstrates HbF induction and reduced anemia","volume":"123","author":"Patthamalai","year":"2014","journal-title":"Blood"},{"issue":"3","key":"2019041121351321100_B64","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1038\/35106079","article-title":"Histone deacetylases and cancer: causes and therapies","volume":"1","author":"Marks","year":"2001","journal-title":"Nat Rev Cancer"},{"issue":"8","key":"2019041121351321100_B65","first-page":"2292","article-title":"A phase I dose escalation and bioavailability study of oral sodium phenylbutyrate in patients with refractory solid tumor malignancies","volume":"7","author":"Gilbert","year":"2001","journal-title":"Clin Cancer Res"},{"issue":"7","key":"2019041121351321100_B66","first-page":"1584","article-title":"Initiation of growth arrest and apoptosis of MCF-7 mammary carcinoma cells by tributyrin, a triglyceride analogue of the short-chain fatty acid butyrate, is associated with mitochondrial activity","volume":"59","author":"Heerdt","year":"1999","journal-title":"Cancer Res"},{"issue":"3","key":"2019041121351321100_B67","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1002\/wrna.1158","article-title":"Targeting RNA splicing for disease therapy","volume":"4","author":"Havens","year":"2013","journal-title":"Wiley Interdiscip Rev RNA"},{"issue":"2","key":"2019041121351321100_B68","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1038\/nsb887","article-title":"Correction of disease-associated exon skipping by synthetic exon-specific activators","volume":"10","author":"Cartegni","year":"2003","journal-title":"Nat Struct Biol"},{"issue":"7","key":"2019041121351321100_B69","doi-asserted-by":"crossref","first-page":"4114","DOI":"10.1073\/pnas.0633863100","article-title":"Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts","volume":"100","author":"Skordis","year":"2003","journal-title":"Proc Natl Acad Sci USA"}],"container-title":["Endocrinology"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/academic.oup.com\/endo\/article-pdf\/157\/10\/3972\/10841008\/endo3972.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,7,1]],"date-time":"2022-07-01T00:25:13Z","timestamp":1656635113000},"score":1,"resource":{"primary":{"URL":"https:\/\/academic.oup.com\/endo\/article\/157\/10\/3972\/2758378"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,6,2]]},"references-count":69,"journal-issue":{"issue":"10","published-online":{"date-parts":[[2016,6,2]]},"published-print":{"date-parts":[[2016,10,1]]}},"URL":"https:\/\/doi.org\/10.1210\/en.2015-2038","relation":{},"ISSN":["0013-7227","1945-7170"],"issn-type":[{"value":"0013-7227","type":"print"},{"value":"1945-7170","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,6,2]]}}}