{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,12]],"date-time":"2026-04-12T13:09:16Z","timestamp":1775999356443,"version":"3.50.1"},"reference-count":48,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2018,3,1]],"date-time":"2018-03-01T00:00:00Z","timestamp":1519862400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2018,3,1]],"date-time":"2018-03-01T00:00:00Z","timestamp":1519862400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Sci Rep"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Histone deacetylase (HDAC) inhibitors are of significant interest as drugs. However, their use to treat neurological disorders has raised concern because HDACs are required for brain function. We have previously shown that a triple combination formulation (TCF) of the pan HDACi vorinostat (Vo), 2-hydroxypropyl-beta-cyclodextrin (HPBCD) and polyethylene glycol (PEG) 400 improves pharmacokinetic exposure and entry of Vo into the brain. TCF treatment significantly delayed both neurodegeneration and death in the\n                    <jats:italic>Npc1<\/jats:italic>\n                    <jats:sup>\n                      <jats:italic>nmf164<\/jats:italic>\n                    <\/jats:sup>\n                    murine model of Niemann-Pick Type C (NPC) disease. The TCF induces no metabolic toxicity, but its risk to normal brain functions and potential utility in treating lung disease, a major NPC clinical complication, remain unknown. Here we report that TCF administered in healthy mice for 8\u201310 months was not detrimental to the brain or neuromuscular functions based on quantitative analyses of Purkinje neurons, neuroinflammation, neurocognitive\/muscular disease symptom progression, cerebellar\/hippocampal nerve fiber-staining, and\n                    <jats:italic>Hdac<\/jats:italic>\n                    gene-expression. The TCF also improved delivery of Vo to lungs and reduced accumulation of foamy macrophages in\n                    <jats:italic>Npc1<\/jats:italic>\n                    <jats:sup>\n                      <jats:italic>nmf164<\/jats:italic>\n                    <\/jats:sup>\n                    mice, with no injury. Together, these data support feasibility of tolerable, chronic administration of an HDACi formulation that treats murine NPC neurological disease and lung pathology, a frequent cause of death in this and possibly additional disorders.\n                  <\/jats:p>","DOI":"10.1038\/s41598-018-22162-7","type":"journal-article","created":{"date-parts":[[2018,2,23]],"date-time":"2018-02-23T06:32:24Z","timestamp":1519367544000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice"],"prefix":"10.1038","volume":"8","author":[{"given":"Md. Suhail","family":"Alam","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bruce","family":"Cooper","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joseph D.","family":"Farris","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5065-158X","authenticated-orcid":false,"given":"Kasturi","family":"Haldar","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2018,3,1]]},"reference":[{"key":"22162_CR1","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/bs.pmch.2014.11.002","volume":"54","author":"N Chessum","year":"2015","unstructured":"Chessum, N., Jones, K., Pasqua, E. & Tucker, M. Recent advances in cancer therapeutics. Progress in medicinal chemistry 54, 1\u201363 (2015).","journal-title":"Progress in medicinal chemistry"},{"key":"22162_CR2","doi-asserted-by":"publisher","first-page":"1439","DOI":"10.4155\/fmc.12.80","volume":"4","author":"G Giannini","year":"2012","unstructured":"Giannini, G., Cabri, W., Fattorusso, C. & Rodriquez, M. Histone deacetylase inhibitors in the treatment of cancer: overview and perspectives. Future Medicinal Chemistry 4, 1439\u20131460 (2012).","journal-title":"Future Medicinal Chemistry"},{"key":"22162_CR3","doi-asserted-by":"publisher","first-page":"3898","DOI":"10.3390\/molecules20033898","volume":"20","author":"M Mottamal","year":"2015","unstructured":"Mottamal, M., Zheng, S. L., Huang, T. L. & Wang, G. D. Histone Deacetylase Inhibitors in Clinical Studies as Templates for New Anticancer Agents. Molecules 20, 3898\u20133941 (2015).","journal-title":"Molecules"},{"key":"22162_CR4","doi-asserted-by":"publisher","first-page":"219","DOI":"10.1038\/nrd4579","volume":"14","author":"KJ Falkenberg","year":"2014","unstructured":"Falkenberg, K. J. & Johnstone, R. W. Histone deacetylases and their inhibitors in cancer, neurological diseases and immune disorders (vol 13, pg 673, 2014). Nature Reviews Drug Discovery 14, 219\u2013219 (2014).","journal-title":"Nature Reviews Drug Discovery"},{"key":"22162_CR5","doi-asserted-by":"publisher","first-page":"981","DOI":"10.1158\/1541-7786.MCR-07-0324","volume":"5","author":"M Dokmanovic","year":"2007","unstructured":"Dokmanovic, M., Clarke, C. & Marks, P. A. Histone deacetylase inhibitors: Overview and perspectives. Molecular Cancer Research 5, 981\u2013989 (2007).","journal-title":"Molecular Cancer Research"},{"key":"22162_CR6","doi-asserted-by":"publisher","first-page":"5541","DOI":"10.1038\/sj.onc.1210620","volume":"26","author":"WS Xu","year":"2007","unstructured":"Xu, W. S., Parmigiani, R. B. & Marks, P. A. Histone deacetylase inhibitors: molecular mechanisms of action. Oncogene 26, 5541\u20135552 (2007).","journal-title":"Oncogene"},{"key":"22162_CR7","doi-asserted-by":"publisher","unstructured":"Di, X.-J., Han, D.-Y., Wang, Y.-J., Chance, M.R. & Mu, T.-W. SAHA Enhances Proteostasis of Epilepsy-Associated \u03b11(A322D)\u03b22\u03b32 GABA(a) Receptors. Chemistry & biology 20, https:\/\/doi.org\/10.1016\/j.chembiol.2013.1009.1020 (2013).","DOI":"10.1016\/j.chembiol.2013.1009.1020"},{"key":"22162_CR8","doi-asserted-by":"publisher","first-page":"966","DOI":"10.1073\/pnas.1221046110","volume":"110","author":"C Yang","year":"2013","unstructured":"Yang, C. et al. Histone deacetylase inhibitors increase glucocerebrosidase activity in Gaucher disease by modulation of molecular chaperones. Proceedings of the National Academy of Sciences of the United States of America 110, 966\u2013971 (2013).","journal-title":"Proceedings of the National Academy of Sciences of the United States of America"},{"key":"22162_CR9","doi-asserted-by":"publisher","first-page":"2623","DOI":"10.2174\/1568026611212220014","volume":"12","author":"B Calamini","year":"2012","unstructured":"Calamini, B. & Morimoto, R. I. Protein Homeostasis as a Therapeutic Target for Diseases of Protein Conformation. Current Topics in Medicinal Chemistry 12, 2623\u20132640 (2012).","journal-title":"Current Topics in Medicinal Chemistry"},{"key":"22162_CR10","doi-asserted-by":"publisher","first-page":"326ra323","DOI":"10.1126\/scitranslmed.aad9407","volume":"8","author":"MS Alam","year":"2016","unstructured":"Alam, M. S., Getz, M. & Haldar, K. Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model. Science translational medicine 8, 326ra323 (2016).","journal-title":"Science translational medicine"},{"key":"22162_CR11","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-5-16","volume":"5","author":"MT Vanier","year":"2010","unstructured":"Vanier, M. T. Niemann-Pick disease type C. Orphanet J Rare Dis 5, 16 (2010).","journal-title":"Orphanet J Rare Dis"},{"key":"22162_CR12","doi-asserted-by":"publisher","first-page":"228","DOI":"10.1126\/science.277.5323.228","volume":"277","author":"ED Carstea","year":"1997","unstructured":"Carstea, E. D. et al. Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis. Science 277, 228\u2013231 (1997).","journal-title":"Science"},{"key":"22162_CR13","doi-asserted-by":"publisher","first-page":"2298","DOI":"10.1126\/science.290.5500.2298","volume":"290","author":"S Naureckiene","year":"2000","unstructured":"Naureckiene, S. et al. Identification of HE1 as the second gene of Niemann-Pick C disease. Science 290, 2298\u20132301 (2000).","journal-title":"Science"},{"key":"22162_CR14","doi-asserted-by":"publisher","first-page":"15330","DOI":"10.1073\/pnas.1112751108","volume":"108","author":"X Xie","year":"2011","unstructured":"Xie, X., Brown, M. S., Shelton, J. M., Richardson, J. A., Goldstein, J. L. & Liang, G. Amino acid substitution in NPC1 that abolishes cholesterol binding reproduces phenotype of complete NPC1 deficiency in mice. Proc. Natl. Acad. Sci. USA 108, 15330\u201315335 (2011).","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"22162_CR15","doi-asserted-by":"publisher","first-page":"e1003462","DOI":"10.1371\/journal.pgen.1003462","volume":"9","author":"T Yu","year":"2013","unstructured":"Yu, T. & Lieberman, A. P. Npc1 acting in neurons and glia is essential for the formation and maintenance of CNS myelin. PLoS Genet 9, e1003462 (2013).","journal-title":"PLoS Genet"},{"key":"22162_CR16","doi-asserted-by":"publisher","first-page":"1582","DOI":"10.1016\/j.ajpath.2016.02.012","volume":"186","author":"BE Kennedy","year":"2016","unstructured":"Kennedy, B. E., Hundert, A. S., Goguen, D., Weaver, I. C. & Karten, B. Presymptomatic Alterations in Amino Acid Metabolism and DNA Methylation in the Cerebellum of a Murine Model of Niemann-Pick Type C Disease. The American journal of pathology 186, 1582\u20131597 (2016).","journal-title":"The American journal of pathology"},{"key":"22162_CR17","doi-asserted-by":"publisher","first-page":"816593","DOI":"10.1155\/2015\/816593","volume":"2015","author":"H Alobaidy","year":"2015","unstructured":"Alobaidy, H. Recent advances in the diagnosis and treatment of niemann-pick disease type C in children: a guide to early diagnosis for the general pediatrician. Int J Pediatr 2015, 816593 (2015).","journal-title":"Int J Pediatr"},{"key":"22162_CR18","doi-asserted-by":"publisher","first-page":"1207","DOI":"10.1002\/ppul.20725","volume":"42","author":"N Guillemot","year":"2007","unstructured":"Guillemot, N., Troadec, C., de Villemeur, T. B., Clement, A. & Fauroux, B. Lung disease in Niemann-Pick disease. Pediatric pulmonology 42, 1207\u20131214 (2007).","journal-title":"Pediatric pulmonology"},{"key":"22162_CR19","doi-asserted-by":"publisher","first-page":"119","DOI":"10.1111\/j.1399-0004.2009.01325.x","volume":"77","author":"M Griese","year":"2010","unstructured":"Griese, M. et al. Respiratory disease in Niemann-Pick type C2 is caused by pulmonary alveolar proteinosis. Clinical genetics 77, 119\u2013130 (2010).","journal-title":"Clinical genetics"},{"key":"22162_CR20","doi-asserted-by":"publisher","first-page":"61","DOI":"10.1007\/s40265-013-0164-6","volume":"74","author":"KA Lyseng-Williamson","year":"2014","unstructured":"Lyseng-Williamson, K. A. Miglustat: a review of its use in Niemann-Pick disease type C. Drugs 74, 61\u201374 (2014).","journal-title":"Drugs"},{"key":"22162_CR21","doi-asserted-by":"publisher","first-page":"37","DOI":"10.1016\/j.jceh.2014.02.005","volume":"4","author":"A Nagral","year":"2014","unstructured":"Nagral, A. Gaucher disease. Journal of clinical and experimental hepatology 4, 37\u201350 (2014).","journal-title":"Journal of clinical and experimental hepatology"},{"key":"22162_CR22","doi-asserted-by":"publisher","first-page":"351","DOI":"10.1016\/j.ymgme.2009.12.006","volume":"99","author":"JE Wraith","year":"2010","unstructured":"Wraith, J. E. et al. Miglustat in adult and juvenile patients with Niemann-Pick disease type C: Long-term data from a clinical trial. Mol. Genet. Metab. 99, 351\u2013357 (2010).","journal-title":"Mol. Genet. Metab."},{"key":"22162_CR23","doi-asserted-by":"publisher","first-page":"358","DOI":"10.1016\/j.ymgme.2009.11.007","volume":"99","author":"M Pineda","year":"2010","unstructured":"Pineda, M. et al. Clinical experience with miglustat therapy in pediatric patients with Niemann-Pick disease type C: a case series. Molecular genetics and metabolism 99, 358\u2013366 (2010).","journal-title":"Molecular genetics and metabolism"},{"key":"22162_CR24","doi-asserted-by":"publisher","first-page":"2377","DOI":"10.1073\/pnas.0810895106","volume":"106","author":"B Liu","year":"2009","unstructured":"Liu, B. et al. Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1\u2212\/\u2212 mouse. Proc Natl Acad Sci USA 106, 2377\u20132382 (2009).","journal-title":"Proc Natl Acad Sci USA"},{"key":"22162_CR25","doi-asserted-by":"crossref","unstructured":"Ory, D. S. et al. Intrathecal 2-hydroxypropyl-beta-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial. Lancet (2017).","DOI":"10.1016\/S0140-6736(17)31465-4"},{"key":"22162_CR26","doi-asserted-by":"publisher","first-page":"491","DOI":"10.1007\/s10545-012-9583-x","volume":"36","author":"CC Pontikis","year":"2013","unstructured":"Pontikis, C. C., Davidson, C. D., Walkley, S. U., Platt, F. M. & Begley, D. J. Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood\u2013brain barrier permeability. J. Inherit. Metab. Dis. 36, 491\u2013498 (2013).","journal-title":"J. Inherit. Metab. Dis."},{"key":"22162_CR27","doi-asserted-by":"publisher","first-page":"9404","DOI":"10.1523\/JNEUROSCI.1317-11.2011","volume":"31","author":"A Aqul","year":"2011","unstructured":"Aqul, A. et al. Unesterified cholesterol accumulation in late endosomes\/lysosomes causes neurodegeneration and is prevented by driving cholesterol export from this compartment. J Neurosci 31, 9404\u20139413 (2011).","journal-title":"J Neurosci"},{"key":"22162_CR28","doi-asserted-by":"publisher","first-page":"276ra226","DOI":"10.1126\/scitranslmed.3010101","volume":"7","author":"CH Vite","year":"2015","unstructured":"Vite, C. H. et al. Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease. Science translational medicine 7, 276ra226 (2015).","journal-title":"Science translational medicine"},{"key":"22162_CR29","doi-asserted-by":"publisher","first-page":"309","DOI":"10.1203\/PDR.0b013e3181ee4dd2","volume":"68","author":"CM Ramirez","year":"2010","unstructured":"Ramirez, C. M. et al. Weekly cyclodextrin administration normalizes cholesterol metabolism in nearly every organ of the Niemann-Pick type C1 mouse and markedly prolongs life. Pediatr Res 68, 309\u2013315 (2010).","journal-title":"Pediatr Res"},{"key":"22162_CR30","doi-asserted-by":"publisher","first-page":"688","DOI":"10.1194\/jlr.M013789","volume":"52","author":"CM Ramirez","year":"2011","unstructured":"Ramirez, C. M. et al. Quantitative role of LAL, NPC2, and NPC1 in lysosomal cholesterol processing defined by genetic and pharmacological manipulations. Journal of lipid research 52, 688\u2013698 (2011).","journal-title":"Journal of lipid research"},{"key":"22162_CR31","doi-asserted-by":"publisher","first-page":"355ra118","DOI":"10.1126\/scitranslmed.aad9823","volume":"8","author":"T Kirkegaard","year":"2016","unstructured":"Kirkegaard, T. et al. Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses. Science translational medicine 8, 355ra118 (2016).","journal-title":"Science translational medicine"},{"key":"22162_CR32","doi-asserted-by":"publisher","first-page":"730","DOI":"10.1093\/hmg\/ddr505","volume":"21","author":"RA Maue","year":"2012","unstructured":"Maue, R. A. et al. A novel mouse model of Niemann-Pick type C disease carrying a D1005G-Npc1 mutation comparable to commonly observed human mutations. Human Molecular Genetics 21, 730\u2013750 (2012).","journal-title":"Human Molecular Genetics"},{"key":"22162_CR33","first-page":"pdb.prot4986","volume":"2008","author":"AH Fischer","year":"2008","unstructured":"Fischer, A. H., Jacobson, K. A., Rose, J. & Zeller, R. Hematoxylin and eosin staining of tissue and cell sections. CSH protocols 2008, pdb.prot4986 (2008).","journal-title":"CSH protocols"},{"key":"22162_CR34","first-page":"132","volume":"117","author":"SS Mirra","year":"1993","unstructured":"Mirra, S. S., Hart, M. N. & Terry, R. D. Making the diagnosis of Alzheimer\u2019s disease. A primer for practicing pathologists. Archives of pathology & laboratory medicine 117, 132\u2013144 (1993).","journal-title":"Archives of pathology & laboratory medicine"},{"key":"22162_CR35","first-page":"123","volume":"315","author":"G Sellge","year":"2006","unstructured":"Sellge, G. & Bischoff, S. C. Isolation, culture, and characterization of intestinal mast cells. Methods Mol Biol 315, 123\u2013138 (2006).","journal-title":"Methods Mol Biol"},{"key":"22162_CR36","doi-asserted-by":"publisher","first-page":"34569","DOI":"10.1074\/jbc.M114.576397","volume":"289","author":"J Norwood","year":"2014","unstructured":"Norwood, J., Franklin, J. M., Sharma, D. & D\u2019Mello, S. R. Histone deacetylase 3 is necessary for proper brain development. J Biol Chem 289, 34569\u201334582 (2014).","journal-title":"J Biol Chem"},{"key":"22162_CR37","doi-asserted-by":"publisher","first-page":"7876","DOI":"10.1073\/pnas.0902750106","volume":"106","author":"RL Montgomery","year":"2009","unstructured":"Montgomery, R. L., Hsieh, J., Barbosa, A. C., Richardson, J. A. & Olson, E. N. Histone deacetylases 1 and 2 control the progression of neural precursors to neurons during brain development. Proc Natl Acad Sci USA 106, 7876\u20137881 (2009).","journal-title":"Proc Natl Acad Sci USA"},{"key":"22162_CR38","doi-asserted-by":"publisher","first-page":"20","DOI":"10.1016\/j.nepig.2014.10.002","volume":"1","author":"C-H Volmar","year":"2015","unstructured":"Volmar, C.-H. & Wahlestedt, C. Histone deacetylases (HDACs) and brain function. Neuroepigenetics 1, 20\u201327 (2015).","journal-title":"Neuroepigenetics"},{"key":"22162_CR39","doi-asserted-by":"publisher","first-page":"3733","DOI":"10.1093\/hmg\/ddu081","volume":"23","author":"A Venkatraman","year":"2014","unstructured":"Venkatraman, A. et al. The histone deacetylase HDAC3 is essential for Purkinje cell function, potentially complicating the use of HDAC inhibitors in SCA1. Human Molecular Genetics 23, 3733\u20133745 (2014).","journal-title":"Human Molecular Genetics"},{"key":"22162_CR40","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1002\/ajmg.b.30969","volume":"153b","author":"NM Yanjanin","year":"2010","unstructured":"Yanjanin, N. M. et al. Linear clinical progression, independent of age of onset, in Niemann-Pick disease, type C. American journal of medical genetics. Part B, Neuropsychiatric genetics: the official publication of the International Society of Psychiatric Genetics 153b, 132\u2013140 (2010).","journal-title":"American journal of medical genetics. Part B, Neuropsychiatric genetics: the official publication of the International Society of Psychiatric Genetics"},{"key":"22162_CR41","doi-asserted-by":"publisher","first-page":"8051","DOI":"10.1074\/jbc.M113.526392","volume":"289","author":"MS Alam","year":"2014","unstructured":"Alam, M. S. et al. Plasma signature of neurological disease in the monogenetic disorder Niemann-Pick Type C. J Biol Chem 289, 8051\u20138066 (2014).","journal-title":"J Biol Chem"},{"key":"22162_CR42","doi-asserted-by":"crossref","unstructured":"Alam, M. S. et al. Genomic Expression Analyses Reveal Lysosomal, Innate Immunity Proteins, as Disease Correlates in Murine Models of a Lysosomal Storage Disorder. Plos One 7 (2012).","DOI":"10.1371\/journal.pone.0048273"},{"key":"22162_CR43","doi-asserted-by":"publisher","first-page":"56ra81","DOI":"10.1126\/scitranslmed.3001417","volume":"2","author":"FD Porter","year":"2010","unstructured":"Porter, F. D. et al. Cholesterol oxidation products are sensitive and specific blood-based biomarkers for Niemann-Pick C1 disease. Science translational medicine 2, 56ra81 (2010).","journal-title":"Science translational medicine"},{"key":"22162_CR44","doi-asserted-by":"publisher","first-page":"337ra363","DOI":"10.1126\/scitranslmed.aaf2326","volume":"8","author":"X Jiang","year":"2016","unstructured":"Jiang, X. et al. Development of a bile acid-based newborn screen for Niemann-Pick disease type C. Science translational medicine 8, 337ra363 (2016).","journal-title":"Science translational medicine"},{"key":"22162_CR45","doi-asserted-by":"publisher","first-page":"1194","DOI":"10.1038\/nm.2828","volume":"18","author":"M Jakovcevski","year":"2012","unstructured":"Jakovcevski, M. & Akbarian, S. Epigenetic mechanisms in neurological disease. Nat Med 18, 1194\u20131204 (2012).","journal-title":"Nat Med"},{"key":"22162_CR46","doi-asserted-by":"publisher","first-page":"821","DOI":"10.1016\/j.cell.2012.09.037","volume":"151","author":"R Sando","year":"2012","unstructured":"Sando, R. et al. HDAC4 Governs a Transcriptional Program Essential for Synaptic Plasticity and Memory. Cell 151, 821\u2013834 (2012).","journal-title":"Cell"},{"key":"22162_CR47","doi-asserted-by":"publisher","first-page":"4819","DOI":"10.1074\/jbc.M110.146860","volume":"286","author":"C Ma","year":"2011","unstructured":"Ma, C. & D\u2019Mello, S. R. Neuroprotection by histone deacetylase-7 (HDAC7) occurs by inhibition of c-jun expression through a deacetylase-independent mechanism. J Biol Chem 286, 4819\u20134828 (2011).","journal-title":"J Biol Chem"},{"key":"22162_CR48","doi-asserted-by":"crossref","unstructured":"Subramanian, K., Rauniyar, N., Lavallee-Adam, M., Yates, J. R., III & Balch, W. E. Quantitative Analysis of the Proteome Response to the Histone Deacetylase Inhibitor (HDACi) Vorinostat in Niemann-Pick Type C1 disease. Molecular & cellular proteomics: MCP (2017).","DOI":"10.1074\/mcp.M116.064949"}],"container-title":["Scientific Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/s41598-018-22162-7.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41598-018-22162-7","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41598-018-22162-7.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,7,1]],"date-time":"2024-07-01T09:56:14Z","timestamp":1719827774000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/s41598-018-22162-7"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,3,1]]},"references-count":48,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2018,12]]}},"alternative-id":["22162"],"URL":"https:\/\/doi.org\/10.1038\/s41598-018-22162-7","relation":{"has-preprint":[{"id-type":"doi","id":"10.1101\/191635","asserted-by":"object"}]},"ISSN":["2045-2322"],"issn-type":[{"value":"2045-2322","type":"electronic"}],"subject":[],"published":{"date-parts":[[2018,3,1]]},"assertion":[{"value":"26 September 2017","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"15 February 2018","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"1 March 2018","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"K.H. holds equity interest in Ranedis Pharmaceuticals L.L.C. U.S. Provisional Application No. 62\/011,553, entitled, \u201cFormulation for the treatment of neurological diseases and cerebral injury\u201d was filed on June 12, 2014\u201d.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing Interests"}}],"article-number":"3875"}}