{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T04:03:17Z","timestamp":1773288197958,"version":"3.50.1"},"publisher-location":"New York, NY","reference-count":81,"publisher":"Springer US","isbn-type":[{"value":"9781071607701","type":"print"},{"value":"9781071607718","type":"electronic"}],"license":[{"start":{"date-parts":[[2020,1,1]],"date-time":"2020-01-01T00:00:00Z","timestamp":1577836800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2020,1,1]],"date-time":"2020-01-01T00:00:00Z","timestamp":1577836800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2020]]},"DOI":"10.1007\/978-1-0716-0771-8_5","type":"book-chapter","created":{"date-parts":[[2020,8,31]],"date-time":"2020-08-31T07:02:49Z","timestamp":1598857369000},"page":"69-85","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Development of Antisense Oligonucleotide Gapmers for the Treatment of Dyslipidemia and Lipodystrophy"],"prefix":"10.1007","author":[{"given":"Tejal","family":"Aslesh","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Toshifumi","family":"Yokota","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2020,9,1]]},"reference":[{"key":"5_CR1","doi-asserted-by":"publisher","first-page":"175628641877693","DOI":"10.1177\/1756286418776932","volume":"11","author":"CD Wurster","year":"2018","unstructured":"Wurster CD, Ludolph AC (2018) Antisense oligonucleotides in neurological disorders. Ther Adv Neurol Disord 11:1756286418776932. \nhttps:\/\/doi.org\/10.1177\/1756286418776932","journal-title":"Ther Adv Neurol Disord"},{"issue":"1","key":"5_CR2","doi-asserted-by":"publisher","first-page":"E1","DOI":"10.3390\/jpm9010001","volume":"9","author":"Y Shimizu-Motohashi","year":"2019","unstructured":"Shimizu-Motohashi Y, Komaki H, Motohashi N et al (2019) Restoring dystrophin expression in duchenne muscular dystrophy: current status of therapeutic approaches. J Pers Med 9(1):E1. \nhttps:\/\/doi.org\/10.3390\/jpm9010001","journal-title":"J Pers Med"},{"key":"5_CR3","doi-asserted-by":"publisher","first-page":"69","DOI":"10.1007\/978-1-4939-8651-4_4","volume":"1828","author":"K Goodkey","year":"2018","unstructured":"Goodkey K, Aslesh T, Maruyama R et al (2018) Nusinersen in the treatment of spinal muscular atrophy. Methods Mol Biol 1828:69\u201376. \nhttps:\/\/doi.org\/10.1007\/978-1-4939-8651-4_4","journal-title":"Methods Mol Biol"},{"issue":"6","key":"5_CR4","doi-asserted-by":"publisher","first-page":"1056","DOI":"10.1016\/j.neuron.2017.04.010","volume":"94","author":"KM Schoch","year":"2017","unstructured":"Schoch KM, Miller TM (2017) Antisense oligonucleotides: translation from mouse models to human neurodegenerative diseases. Neuron 94(6):1056\u20131070. \nhttps:\/\/doi.org\/10.1016\/j.neuron.2017.04.010","journal-title":"Neuron"},{"issue":"14","key":"5_CR5","doi-asserted-by":"publisher","first-page":"6518","DOI":"10.1093\/nar\/gkw236","volume":"44","author":"RL Juliano","year":"2016","unstructured":"Juliano RL (2016) The delivery of therapeutic oligonucleotides. Nucleic Acids Res 44(14):6518\u20136548. \nhttps:\/\/doi.org\/10.1093\/nar\/gkw236","journal-title":"Nucleic Acids Res"},{"key":"5_CR6","doi-asserted-by":"publisher","first-page":"259","DOI":"10.1146\/annurev.pharmtox.010909.105654","volume":"50","author":"CF Bennett","year":"2010","unstructured":"Bennett CF, Swayze EE (2010) RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol 50:259\u2013293. \nhttps:\/\/doi.org\/10.1146\/annurev.pharmtox.010909.105654","journal-title":"Annu Rev Pharmacol Toxicol"},{"key":"5_CR7","doi-asserted-by":"publisher","first-page":"46","DOI":"10.1016\/j.addr.2015.01.008","volume":"87","author":"RS Geary","year":"2015","unstructured":"Geary RS, Norris D, Yu R et al (2015) Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides. Adv Drug Deliv Rev 87:46\u201351. \nhttps:\/\/doi.org\/10.1016\/j.addr.2015.01.008","journal-title":"Adv Drug Deliv Rev"},{"key":"5_CR8","unstructured":"Crooke ST (2019) Antisense technology past, Present and Future Ionis Pharmaceuticals.\u00a0https:\/\/ir.ionispharma.com\/static-files\/. Accessed June 5 2019"},{"issue":"32","key":"5_CR9","doi-asserted-by":"publisher","first-page":"2146","DOI":"10.1093\/eurheartj\/ehu274","volume":"35","author":"M Cuchel","year":"2014","unstructured":"Cuchel M, Bruckert E, Ginsberg HN et al (2014) Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the consensus panel on familial Hypercholesterolaemia of the European atherosclerosis society. Eur Heart J 35(32):2146\u20132157","journal-title":"Eur Heart J"},{"issue":"2","key":"5_CR10","doi-asserted-by":"publisher","first-page":"262","DOI":"10.1016\/j.atherosclerosis.2012.02.019","volume":"223","author":"FJ Raal","year":"2012","unstructured":"Raal FJ, Santos RD (2012) Homozygous familial hypercholesterolemia: current perspectives on diagnosis and treatment. Atherosclerosis 223(2):262\u2013268","journal-title":"Atherosclerosis"},{"key":"5_CR11","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1016\/j.atherosclerosissup.2018.04.199","volume":"32","author":"AH Michael Davidson","year":"2018","unstructured":"Michael Davidson AH, Ahmad Z, van Lennep JR, Stevenson M (2018) Examining the high disease burden and impact on quality of life in familial Chylomicronemia syndrome. Atherosclerosis 32:1. \nhttps:\/\/doi.org\/10.1016\/j.atherosclerosissup.2018.04.199","journal-title":"Atherosclerosis"},{"key":"5_CR12","volume-title":"Principles and practice of endocrinology and metabolism","author":"R Mahley","year":"2001","unstructured":"Mahley R (2001) Biochemistry and physiology of lipid and lipoprotein metabolism. In: Becker KL (ed) Principles and practice of endocrinology and metabolism, 3rd edn. Lippincott Williams and Wilkins, Philadelphia","edition":"3"},{"issue":"5","key":"5_CR13","doi-asserted-by":"publisher","first-page":"1069","DOI":"10.1016\/j.ymthe.2017.03.023","volume":"25","author":"CA Stein","year":"2017","unstructured":"Stein CA, Castanotto D (2017) FDA-approved oligonucleotide therapies in 2017. Mol Ther 25(5):1069\u20131075. \nhttps:\/\/doi.org\/10.1016\/j.ymthe.2017.03.023","journal-title":"Mol Ther"},{"issue":"9719","key":"5_CR14","doi-asserted-by":"publisher","first-page":"998","DOI":"10.1016\/S0140-6736(10)60284-X","volume":"375","author":"FJ Raal","year":"2010","unstructured":"Raal FJ, Santos RD, Blom DJ et al (2010) Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial. Lancet 375(9719):998\u20131006. \nhttps:\/\/doi.org\/10.1016\/S0140-6736(10)60284-X","journal-title":"Lancet"},{"issue":"11","key":"5_CR15","doi-asserted-by":"publisher","first-page":"1445","DOI":"10.2165\/11635060-000000000-00000","volume":"72","author":"C Gelsinger","year":"2012","unstructured":"Gelsinger C, Steinhagen-Thiessen E, Kassner U (2012) Therapeutic potential of mipomersen in the management of familial hypercholesterolaemia. Drugs 72(11):1445\u20131455. \nhttps:\/\/doi.org\/10.2165\/11635060-000000000-00000","journal-title":"Drugs"},{"key":"5_CR16","doi-asserted-by":"publisher","first-page":"a2423","DOI":"10.1136\/bmj.a2423","volume":"337","author":"J Versmissen","year":"2008","unstructured":"Versmissen J, Oosterveer DM, Yazdanpanah M et al (2008) Efficacy of statins in familial hypercholesterolaemia: a long term cohort study. BMJ 337:a2423. \nhttps:\/\/doi.org\/10.1136\/bmj.a2423","journal-title":"BMJ"},{"issue":"9493","key":"5_CR17","doi-asserted-by":"publisher","first-page":"1267","DOI":"10.1016\/S0140-6736(05)67394-1","volume":"366","author":"C Baigent","year":"2005","unstructured":"Baigent C, Keech A, Kearney PM et al (2005) Efficacy and safety of cholesterol-lowering treatment: prospective meta-analysis of data from 90,056 participants in 14 randomised trials of statins. Lancet 366(9493):1267\u20131278. \nhttps:\/\/doi.org\/10.1016\/S0140-6736(05)67394-1","journal-title":"Lancet"},{"issue":"2","key":"5_CR18","doi-asserted-by":"publisher","first-page":"223","DOI":"10.1016\/j.amjcard.2011.03.027","volume":"108","author":"A Elis","year":"2011","unstructured":"Elis A, Zhou R, Stein EA (2011) Effect of lipid-lowering treatment on natural history of heterozygous familial hypercholesterolemia in past three decades. Am J Cardiol 108(2):223\u2013226. \nhttps:\/\/doi.org\/10.1016\/j.amjcard.2011.03.027","journal-title":"Am J Cardiol"},{"issue":"3","key":"5_CR19","doi-asserted-by":"publisher","first-page":"354","DOI":"10.1016\/j.amjcard.2010.03.033","volume":"106","author":"TR Pedersen","year":"2010","unstructured":"Pedersen TR, Cater NB, Faergeman O et al (2010) Comparison of atorvastatin 80 mg\/day versus simvastatin 20 to 40 mg\/day on frequency of cardiovascular events late (five years) after acute myocardial infarction (from the incremental decrease in end points through aggressive lipid lowering [IDEAL] trial). Am J Cardiol 106(3):354\u2013359. \nhttps:\/\/doi.org\/10.1016\/j.amjcard.2010.03.033","journal-title":"Am J Cardiol"},{"issue":"19","key":"5_CR20","doi-asserted-by":"publisher","first-page":"2437","DOI":"10.1001\/jama.294.19.2437","volume":"294","author":"TR Pedersen","year":"2005","unstructured":"Pedersen TR, Faergeman O, Kastelein JJ et al (2005) High-dose atorvastatin vs usual-dose simvastatin for secondary prevention after myocardial infarction: the IDEAL study: a randomized controlled trial. JAMA 294(19):2437\u20132445. \nhttps:\/\/doi.org\/10.1001\/jama.294.19.2437","journal-title":"JAMA"},{"issue":"1","key":"5_CR21","doi-asserted-by":"publisher","first-page":"3","DOI":"10.1016\/j.atherosclerosis.2011.06.028","volume":"217","author":"AL Catapano","year":"2011","unstructured":"Catapano AL, Reiner Z, De Backer G et al (2011) ESC\/EAS guidelines for the management of dyslipidaemias the task force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and the European atherosclerosis society (EAS). Atherosclerosis 217(1):3\u201346","journal-title":"Atherosclerosis"},{"issue":"17","key":"5_CR22","doi-asserted-by":"publisher","first-page":"3132","DOI":"10.2174\/13816128113199990312","volume":"19","author":"C Gebhard","year":"2013","unstructured":"Gebhard C, Huard G, Kritikou EA et al (2013) Apolipoprotein B antisense inhibition-update on mipomersen. Curr Pharm Des 19(17):3132\u20133142","journal-title":"Curr Pharm Des"},{"issue":"5","key":"5_CR23","doi-asserted-by":"publisher","first-page":"910","DOI":"10.1016\/j.bcp.2008.11.005","volume":"77","author":"RZ Yu","year":"2009","unstructured":"Yu RZ, Lemonidis KM, Graham MJ et al (2009) Cross-species comparison of in vivo PK\/PD relationships for second-generation antisense oligonucleotides targeting apolipoprotein B-100. Biochem Pharmacol 77(5):910\u2013919. \nhttps:\/\/doi.org\/10.1016\/j.bcp.2008.11.005","journal-title":"Biochem Pharmacol"},{"issue":"2","key":"5_CR24","doi-asserted-by":"publisher","first-page":"127","DOI":"10.1080\/14656566.2018.1550071","volume":"20","author":"JS Parham","year":"2019","unstructured":"Parham JS, Goldberg AC (2019) Mipomersen and its use in familial hypercholesterolemia. Expert Opin Pharmacother 20(2):127\u2013131. \nhttps:\/\/doi.org\/10.1080\/14656566.2018.1550071","journal-title":"Expert Opin Pharmacother"},{"issue":"5","key":"5_CR25","doi-asserted-by":"publisher","first-page":"872","DOI":"10.1194\/jlr.M400492-JLR200","volume":"46","author":"RM Crooke","year":"2005","unstructured":"Crooke RM, Graham MJ, Lemonidis KM et al (2005) An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis. J Lipid Res 46(5):872\u2013884. \nhttps:\/\/doi.org\/10.1194\/jlr.M400492-JLR200","journal-title":"J Lipid Res"},{"issue":"16","key":"5_CR26","doi-asserted-by":"publisher","first-page":"1729","DOI":"10.1161\/CIRCULATIONAHA.105.606442","volume":"114","author":"JJ Kastelein","year":"2006","unstructured":"Kastelein JJ, Wedel MK, Baker BF et al (2006) Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation 114(16):1729\u20131735. \nhttps:\/\/doi.org\/10.1161\/CIRCULATIONAHA.105.606442","journal-title":"Circulation"},{"issue":"21","key":"5_CR27","doi-asserted-by":"publisher","first-page":"2650","DOI":"10.1093\/eurheartj\/ehr148","volume":"32","author":"F Akdim","year":"2011","unstructured":"Akdim F, Tribble DL, Flaim JD et al (2011) Efficacy of apolipoprotein B synthesis inhibition in subjects with mild-to-moderate hyperlipidaemia. Eur Heart J 32(21):2650\u20132659. \nhttps:\/\/doi.org\/10.1093\/eurheartj\/ehr148","journal-title":"Eur Heart J"},{"key":"5_CR28","unstructured":"Genyzme Corporation. KYNAMRO\u00ae (mipomersen sodium) injection prescribing information. (2013). \nhttp:\/\/www.kynamro.com\/~\/media\/Kynamro\/Files\/KYNAMRO-PI.pdf\n\n. Accessed June 5 2019"},{"issue":"1","key":"5_CR29","doi-asserted-by":"publisher","first-page":"131","DOI":"10.1210\/er.2014-1062","volume":"36","author":"GF Lewis","year":"2015","unstructured":"Lewis GF, Xiao C, Hegele RA (2015) Hypertriglyceridemia in the genomic era: a new paradigm. Endocr Rev 36(1):131\u2013147. \nhttps:\/\/doi.org\/10.1210\/er.2014-1062","journal-title":"Endocr Rev"},{"issue":"3","key":"5_CR30","doi-asserted-by":"publisher","first-page":"981","DOI":"10.3390\/nu5030981","volume":"5","author":"A Brahm","year":"2013","unstructured":"Brahm A, Hegele RA (2013) Hypertriglyceridemia. Nutrients 5(3):981\u20131001. \nhttps:\/\/doi.org\/10.3390\/nu5030981","journal-title":"Nutrients"},{"issue":"8","key":"5_CR31","doi-asserted-by":"publisher","first-page":"655","DOI":"10.1016\/S2213-8587(13)70191-8","volume":"2","author":"RA Hegele","year":"2014","unstructured":"Hegele RA, Ginsberg HN, Chapman MJ et al (2014) The polygenic nature of hypertriglyceridaemia: implications for definition, diagnosis, and management. Lancet Diabetes Endocrinol 2(8):655\u2013666. \nhttps:\/\/doi.org\/10.1016\/S2213-8587(13)70191-8","journal-title":"Lancet Diabetes Endocrinol"},{"issue":"2","key":"5_CR32","doi-asserted-by":"publisher","first-page":"189","DOI":"10.1194\/jlr.R009720","volume":"52","author":"CT Johansen","year":"2011","unstructured":"Johansen CT, Kathiresan S, Hegele RA (2011) Genetic determinants of plasma triglycerides. J Lipid Res 52(2):189\u2013206. \nhttps:\/\/doi.org\/10.1194\/jlr.R009720","journal-title":"J Lipid Res"},{"issue":"4","key":"5_CR33","doi-asserted-by":"publisher","first-page":"247","DOI":"10.1097\/MOL.0b013e3283471972","volume":"22","author":"CT Johansen","year":"2011","unstructured":"Johansen CT, Hegele RA (2011) Genetic bases of hypertriglyceridemic phenotypes. Curr Opin Lipidol 22(4):247\u2013253. \nhttps:\/\/doi.org\/10.1097\/MOL.0b013e3283471972","journal-title":"Curr Opin Lipidol"},{"issue":"5","key":"5_CR34","doi-asserted-by":"publisher","first-page":"833","DOI":"10.1016\/j.bbalip.2011.10.007","volume":"1821","author":"CT Johansen","year":"2012","unstructured":"Johansen CT, Hegele RA (2012) Allelic and phenotypic spectrum of plasma triglycerides. Biochim Biophys Acta 1821(5):833\u2013842. \nhttps:\/\/doi.org\/10.1016\/j.bbalip.2011.10.007","journal-title":"Biochim Biophys Acta"},{"issue":"5","key":"5_CR35","doi-asserted-by":"publisher","first-page":"438","DOI":"10.1056\/NEJMoa1400283","volume":"373","author":"D Gaudet","year":"2015","unstructured":"Gaudet D, Alexander VJ, Baker BF et al (2015) Antisense inhibition of Apolipoprotein C-III in patients with hypertriglyceridemia. N Engl J Med 373(5):438\u2013447. \nhttps:\/\/doi.org\/10.1056\/NEJMoa1400283","journal-title":"N Engl J Med"},{"issue":"5","key":"5_CR36","doi-asserted-by":"publisher","first-page":"799","DOI":"10.1111\/j.1742-1241.2007.01678.x","volume":"62","author":"DC Chan","year":"2008","unstructured":"Chan DC, Chen MM, Ooi EM et al (2008) An ABC of apolipoprotein C-III: a clinically useful new cardiovascular risk factor? Int J Clin Pract 62(5):799\u2013809. \nhttps:\/\/doi.org\/10.1111\/j.1742-1241.2007.01678.x","journal-title":"Int J Clin Pract"},{"key":"5_CR37","doi-asserted-by":"publisher","first-page":"19","DOI":"10.1016\/j.atherosclerosissup.2017.05.003","volume":"30","author":"I Gouni-Berthold","year":"2017","unstructured":"Gouni-Berthold I (2017) The role of antisense oligonucleotide therapy against apolipoprotein-CIII in hypertriglyceridemia. Atheroscler Suppl 30:19\u201327. \nhttps:\/\/doi.org\/10.1016\/j.atherosclerosissup.2017.05.003","journal-title":"Atheroscler Suppl"},{"issue":"2","key":"5_CR38","doi-asserted-by":"publisher","first-page":"239","DOI":"10.1161\/ATVBAHA.109.197830","volume":"30","author":"CO Mendivil","year":"2010","unstructured":"Mendivil CO, Zheng C, Furtado J et al (2010) Metabolism of very-low-density lipoprotein and low-density lipoprotein containing apolipoprotein C-III and not other small apolipoproteins. Arterioscler Thromb Vasc Biol 30(2):239\u2013245. \nhttps:\/\/doi.org\/10.1161\/ATVBAHA.109.197830","journal-title":"Arterioscler Thromb Vasc Biol"},{"issue":"5908","key":"5_CR39","doi-asserted-by":"publisher","first-page":"1702","DOI":"10.1126\/science.1161524","volume":"322","author":"TI Pollin","year":"2008","unstructured":"Pollin TI, Damcott CM, Shen H et al (2008) A null mutation in human APOC3 confers a favorable plasma lipid profile and apparent cardioprotection. Science 322(5908):1702\u20131705. \nhttps:\/\/doi.org\/10.1126\/science.1161524","journal-title":"Science"},{"issue":"1","key":"5_CR40","doi-asserted-by":"publisher","first-page":"22","DOI":"10.1056\/NEJMoa1307095","volume":"371","author":"I Blood","year":"2014","unstructured":"Tg, Hdl Working Group of the Exome Sequencing Project NHL, Blood I et al (2014) Loss-of-function mutations in APOC3, triglycerides, and coronary disease. N Engl J Med 371(1):22\u201331. \nhttps:\/\/doi.org\/10.1056\/NEJMoa1307095","journal-title":"N Engl J Med"},{"issue":"6209","key":"5_CR41","doi-asserted-by":"publisher","first-page":"6","DOI":"10.1126\/science.1258040","volume":"346","author":"NTS-GJ Schirle","year":"2014","unstructured":"Schirle NTS-GJ, MacRae IJ (2014) Structural basis for microRNA targeting. Science 346(6209):6. \nhttps:\/\/doi.org\/10.1126\/science.1258040","journal-title":"Science"},{"issue":"6","key":"5_CR42","doi-asserted-by":"publisher","first-page":"1358","DOI":"10.2337\/diacare.27.6.1358","volume":"27","author":"GM Dallinga-Thie","year":"2004","unstructured":"Dallinga-Thie GM, Berk P II, Bootsma AH et al (2004) Atorvastatin decreases apolipoprotein C-III in apolipoprotein B-containing lipoprotein and HDL in type 2 diabetes: a potential mechanism to lower plasma triglycerides. Diabetes Care 27(6):1358\u20131364. \nhttps:\/\/doi.org\/10.2337\/diacare.27.6.1358","journal-title":"Diabetes Care"},{"key":"5_CR43","doi-asserted-by":"publisher","first-page":"6","DOI":"10.2337\/dc08-0358","volume":"8","author":"EMWG Ooi","year":"2008","unstructured":"Ooi EMWG, Chan DC, Chen MM, Nestel PJ, Sviridov D et al (2008) Dose-dependent effect of rosuvastatin on VLDL-apolipoprotein C-III kinetics in the metabolic syndrome. Diabetes Care 8:6. \nhttps:\/\/doi.org\/10.2337\/dc08-0358","journal-title":"Diabetes Care"},{"issue":"6","key":"5_CR44","doi-asserted-by":"publisher","first-page":"473","DOI":"10.2165\/00044011-199815060-00003","volume":"15","author":"E Swahn","year":"1998","unstructured":"Swahn E, von Schenck H, Olsson AG (1998) Omega-3 ethyl Ester concentrate decreases Total Apolipoprotein CIII and increases Antithrombin III in Postmyocardial infarction patients. Clin Drug Investig 15(6):473\u2013482. \nhttps:\/\/doi.org\/10.2165\/00044011-199815060-00003","journal-title":"Clin Drug Investig"},{"issue":"4","key":"5_CR45","doi-asserted-by":"publisher","first-page":"389","DOI":"10.1080\/13543784.2019.1582028","volume":"28","author":"D Milonas","year":"2019","unstructured":"Milonas D, Tziomalos K (2019) Experimental therapies targeting apolipoprotein C-III for the treatment of hyperlipidemia - spotlight on volanesorsen. Expert Opin Investig Drugs 28(4):389\u2013394. \nhttps:\/\/doi.org\/10.1080\/13543784.2019.1582028","journal-title":"Expert Opin Investig Drugs"},{"issue":"11","key":"5_CR46","doi-asserted-by":"publisher","first-page":"1479","DOI":"10.1161\/CIRCRESAHA.111.300367","volume":"112","author":"MJ Graham","year":"2013","unstructured":"Graham MJ, Lee RG, Bell TA 3rd et al (2013) Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humans. Circ Res 112(11):1479\u20131490. \nhttps:\/\/doi.org\/10.1161\/CIRCRESAHA.111.300367","journal-title":"Circ Res"},{"issue":"9","key":"5_CR47","doi-asserted-by":"publisher","first-page":"2969","DOI":"10.1210\/jc.2011-3213","volume":"97","author":"LBJ Berglund","year":"2012","unstructured":"Berglund LBJ, Goldberg AC, Goldberg IJ, Sacks F, Murad MH, Stalenhoef AF, Endocrine society (2012) Evaluation and treatment of hypertriglyceridemia: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab 97(9):2969\u20132989","journal-title":"J Clin Endocrinol Metab"},{"issue":"8","key":"5_CR48","doi-asserted-by":"publisher","first-page":"1408","DOI":"10.2337\/dc16-0126","volume":"39","author":"A Digenio","year":"2016","unstructured":"Digenio A, Dunbar RL, Alexander VJ et al (2016) Antisense-mediated lowering of plasma Apolipoprotein C-III by Volanesorsen improves Dyslipidemia and insulin sensitivity in type 2 diabetes. Diabetes Care 39(8):1408\u20131415. \nhttps:\/\/doi.org\/10.2337\/dc16-0126","journal-title":"Diabetes Care"},{"issue":"12","key":"5_CR49","doi-asserted-by":"publisher","first-page":"1469","DOI":"10.1016\/j.metabol.2014.08.010","volume":"63","author":"L Wu","year":"2014","unstructured":"Wu L, Parhofer KG (2014) Diabetic dyslipidemia. Metabolism 63(12):1469\u20131479. \nhttps:\/\/doi.org\/10.1016\/j.metabol.2014.08.010","journal-title":"Metabolism"},{"issue":"2","key":"5_CR50","doi-asserted-by":"publisher","first-page":"307","DOI":"10.1161\/01.ATV.0000046230.02211.B4","volume":"23","author":"WH Capell","year":"2003","unstructured":"Capell WH, DeSouza CA, Poirier P et al (2003) Short-term triglyceride lowering with fenofibrate improves vasodilator function in subjects with hypertriglyceridemia. Arterioscler Thromb Vasc Biol 23(2):307\u2013313","journal-title":"Arterioscler Thromb Vasc Biol"},{"issue":"2","key":"5_CR51","doi-asserted-by":"publisher","first-page":"80","DOI":"10.1016\/j.tcm.2019.03.001","volume":"30","author":"V Chyzhyk","year":"2019","unstructured":"Chyzhyk V, Brown AS (2019) Familial chylomicronemia syndrome: a rare but devastating autosomal recessive disorder characterized by refractory hypertriglyceridemia and recurrent pancreatitis. Trends Cardiovasc Med 30(2):80\u201385. \nhttps:\/\/doi.org\/10.1016\/j.tcm.2019.03.001","journal-title":"Trends Cardiovasc Med"},{"key":"5_CR52","doi-asserted-by":"publisher","first-page":"7","DOI":"10.1016\/j.atherosclerosissup.2016.10.002","volume":"23","author":"E Stroes","year":"2017","unstructured":"Stroes E, Moulin P, Parhofer KG, Rebours V, L\u00f6hr JM, Averna M (2017) Diagnostic algorithm for familial chylomicronemia syndrome. Atheroscler Suppl 23:7. \nhttps:\/\/doi.org\/10.1016\/j.atherosclerosissup.2016.10.002","journal-title":"Atheroscler Suppl"},{"issue":"2","key":"5_CR53","doi-asserted-by":"publisher","first-page":"13","DOI":"10.1124\/jpet.115.229740","volume":"356","author":"T Sakurai","year":"2016","unstructured":"Sakurai T, Sakurai A, Vaisman BL, Amar MJ, Liu C et al (2016) Creation of Apolipoprotein C-II (ApoC-II) mutant mice and correction of their hypertriglyceridemia with an ApoC-II mimetic peptide. J Pharmacol Exp Ther 356(2):13. \nhttps:\/\/doi.org\/10.1124\/jpet.115.229740","journal-title":"J Pharmacol Exp Ther"},{"issue":"6","key":"5_CR54","doi-asserted-by":"publisher","first-page":"352","DOI":"10.1038\/nrendo.2015.26","volume":"11","author":"AJ Brahm","year":"2015","unstructured":"Brahm AJ, Hegele RA (2015) Chylomicronaemia--current diagnosis and future therapies. Nat Rev Endocrinol 11(6):352\u2013362. \nhttps:\/\/doi.org\/10.1038\/nrendo.2015.26","journal-title":"Nat Rev Endocrinol"},{"issue":"1","key":"5_CR55","doi-asserted-by":"publisher","first-page":"1","DOI":"10.5551\/jat.10702","volume":"19","author":"T Gotoda","year":"2012","unstructured":"Gotoda T, Shirai K, Ohta T et al (2012) Diagnosis and management of type I and type V hyperlipoproteinemia. J Atheroscler Thromb 19(1):1\u201312","journal-title":"J Atheroscler Thromb"},{"key":"5_CR56","doi-asserted-by":"publisher","first-page":"125","DOI":"10.1186\/1475-2840-11-125","volume":"11","author":"A Tenenbaum","year":"2012","unstructured":"Tenenbaum A, Fisman EZ (2012) Fibrates are an essential part of modern anti-dyslipidemic arsenal: spotlight on atherogenic dyslipidemia and residual risk reduction. Cardiovasc Diabetol 11:125. \nhttps:\/\/doi.org\/10.1186\/1475-2840-11-125","journal-title":"Cardiovasc Diabetol"},{"issue":"5","key":"5_CR57","doi-asserted-by":"publisher","first-page":"1234","DOI":"10.1016\/j.jacl.2018.05.013","volume":"12","author":"DJ Blom","year":"2018","unstructured":"Blom DJ, O\u2019Dea L, Digenio A et al (2018) Characterizing familial chylomicronemia syndrome: baseline data of the APPROACH study. J Clin Lipidol 12(5):1234\u20131243. e1235. \nhttps:\/\/doi.org\/10.1016\/j.jacl.2018.05.013","journal-title":"J Clin Lipidol"},{"issue":"23","key":"5_CR58","doi-asserted-by":"publisher","first-page":"2200","DOI":"10.1056\/NEJMoa1400284","volume":"371","author":"D Gaudet","year":"2014","unstructured":"Gaudet D, Brisson D, Tremblay K et al (2014) Targeting APOC3 in the familial chylomicronemia syndrome. N Engl J Med 371(23):2200\u20132206. \nhttps:\/\/doi.org\/10.1056\/NEJMoa1400284","journal-title":"N Engl J Med"},{"issue":"7","key":"5_CR59","doi-asserted-by":"publisher","first-page":"537","DOI":"10.1080\/14779072.2018.1487290","volume":"16","author":"M Arca","year":"2018","unstructured":"Arca M, Hsieh A, Soran H et al (2018) The effect of volanesorsen treatment on the burden associated with familial chylomicronemia syndrome: the results of the ReFOCUS study. Expert Rev Cardiovasc Ther 16(7):537\u2013546. \nhttps:\/\/doi.org\/10.1080\/14779072.2018.1487290","journal-title":"Expert Rev Cardiovasc Ther"},{"key":"5_CR60","doi-asserted-by":"publisher","first-page":"14","DOI":"10.1016\/j.atherosclerosissup.2018.04.042","volume":"32","author":"D DA Gaudet","year":"2018","unstructured":"DA Gaudet D, Alexander V et al (2018) The APPROACH study: a randomized, double-blind, placebo-controlled, phase 3 study of volanesorsen administered subcutaneously to patients with familial chylomicronemia syndrome (FCS). Atherosclerosis 32:14\u201315. \nhttps:\/\/doi.org\/10.1016\/j.atherosclerosissup.2018.04.042","journal-title":"Atherosclerosis"},{"issue":"3","key":"5_CR61","doi-asserted-by":"publisher","first-page":"17","DOI":"10.1161\/CIRCRESAHA.118.313171","volume":"124","author":"RA Hegele","year":"2019","unstructured":"Hegele RA, Tsimikas S (2019) Lipid-lowering agents. Circ Res 124(3):17. \nhttps:\/\/doi.org\/10.1161\/CIRCRESAHA.118.313171","journal-title":"Circ Res"},{"key":"5_CR62","unstructured":"Akcea and Ionis Announce Approval of WAYLIVRA\u00ae (volanesorsen) in the European Union. (7 May 2019). \nhttps:\/\/ir.akceatx.com\/news-releases\/news-release-details\/akcea-and-ionis-announce-approval-waylivrar-volanesorsen\n\n. Accessed October 30 2019"},{"issue":"12","key":"5_CR63","doi-asserted-by":"publisher","first-page":"1349","DOI":"10.1007\/s40265-019-01168-z","volume":"79","author":"J Paik","year":"2019","unstructured":"Paik J, Duggan S (2019) Volanesorsen: first global approval. Drugs 79(12):1349\u20131354. \nhttps:\/\/doi.org\/10.1007\/s40265-019-01168-z","journal-title":"Drugs"},{"issue":"9","key":"5_CR64","doi-asserted-by":"publisher","first-page":"E1676","DOI":"10.1210\/jc.2014-1878","volume":"99","author":"J Joseph","year":"2014","unstructured":"Joseph J, Shamburek RD, Cochran EK et al (2014) Lipid regulation in lipodystrophy versus the obesity-associated metabolic syndrome: the dissociation of HDL-C and triglycerides. J Clin Endocrinol Metab 99(9):E1676\u2013E1680. \nhttps:\/\/doi.org\/10.1210\/jc.2014-1878","journal-title":"J Clin Endocrinol Metab"},{"issue":"1","key":"5_CR65","doi-asserted-by":"publisher","first-page":"107","DOI":"10.4158\/endp.19.1.v767575m65p5mr06","volume":"19","author":"Y Handelsman","year":"2013","unstructured":"Handelsman Y, Oral EA, Bloomgarden ZT et al (2013) The clinical approach to the detection of lipodystrophy - an AACE consensus statement. Endocr Pract 19(1):107\u2013116","journal-title":"Endocr Pract"},{"issue":"2","key":"5_CR66","doi-asserted-by":"publisher","first-page":"310","DOI":"10.4158\/EP09154.RA","volume":"16","author":"JL Chan","year":"2010","unstructured":"Chan JL, Oral EA (2010) Clinical classification and treatment of congenital and acquired lipodystrophy. Endocr Pract 16(2):310\u2013323. \nhttps:\/\/doi.org\/10.4158\/EP09154.RA","journal-title":"Endocr Pract"},{"issue":"1","key":"5_CR67","doi-asserted-by":"publisher","first-page":"66","DOI":"10.1111\/j.1399-0004.2009.01350.x","volume":"78","author":"L Subramanyam","year":"2010","unstructured":"Subramanyam L, Simha V, Garg A (2010) Overlapping syndrome with familial partial lipodystrophy, Dunnigan variety and cardiomyopathy due to amino-terminal heterozygous missense Lamin a\/C mutations. Clin Genet 78(1):66\u201373. \nhttps:\/\/doi.org\/10.1111\/j.1399-0004.2009.01350.x","journal-title":"Clin Genet"},{"issue":"12","key":"5_CR68","doi-asserted-by":"publisher","first-page":"1220","DOI":"10.1056\/NEJMra025261","volume":"350","author":"A Garg","year":"2004","unstructured":"Garg A (2004) Acquired and inherited lipodystrophies. N Engl J Med 350(12):1220\u20131234. \nhttps:\/\/doi.org\/10.1056\/NEJMra025261","journal-title":"N Engl J Med"},{"issue":"5","key":"5_CR69","doi-asserted-by":"publisher","first-page":"698","DOI":"10.1111\/cen.13311","volume":"86","author":"N Ajluni","year":"2017","unstructured":"Ajluni N, Meral R, Neidert AH et al (2017) Spectrum of disease associated with partial lipodystrophy: lessons from a trial cohort. Clin Endocrinol 86(5):698\u2013707. \nhttps:\/\/doi.org\/10.1111\/cen.13311","journal-title":"Clin Endocrinol"},{"key":"5_CR70","unstructured":"Familial Partial Lipodystrophy. (2015) Rare Disease Database. \nhttps:\/\/rarediseases.org\/rare-diseases\/familial-partial-lipodystrophy\/\n\n. Accessed June 5 2019"},{"issue":"4","key":"5_CR71","doi-asserted-by":"publisher","first-page":"1790","DOI":"10.1210\/jc.2015-3891","volume":"101","author":"A Kassai","year":"2016","unstructured":"Kassai A, Muniyappa R, Levenson AE et al (2016) Effect of Leptin administration on circulating Apolipoprotein CIII levels in patients with Lipodystrophy. J Clin Endocrinol Metab 101(4):1790\u20131797. \nhttps:\/\/doi.org\/10.1210\/jc.2015-3891","journal-title":"J Clin Endocrinol Metab"},{"issue":"13","key":"5_CR72","doi-asserted-by":"publisher","first-page":"1371","DOI":"10.1007\/s40265-018-0968-5","volume":"78","author":"SJ Keam","year":"2018","unstructured":"Keam SJ (2018) Inotersen: first global approval. Drugs 78(13):1371\u20131376. \nhttps:\/\/doi.org\/10.1007\/s40265-018-0968-5","journal-title":"Drugs"},{"issue":"4","key":"5_CR73","doi-asserted-by":"publisher","first-page":"406","DOI":"10.1016\/j.tips.2017.01.003","volume":"38","author":"G Guidotti","year":"2017","unstructured":"Guidotti G, Brambilla L, Rossi D (2017) Cell-penetrating peptides: from basic research to clinics. Trends Pharmacol Sci 38(4):406\u2013424. \nhttps:\/\/doi.org\/10.1016\/j.tips.2017.01.003","journal-title":"Trends Pharmacol Sci"},{"issue":"3","key":"5_CR74","doi-asserted-by":"publisher","first-page":"130","DOI":"10.1089\/nat.2016.0652","volume":"27","author":"F Shabanpoor","year":"2017","unstructured":"Shabanpoor F, Hammond SM, Abendroth F et al (2017) Identification of a peptide for systemic brain delivery of a Morpholino oligonucleotide in mouse models of spinal muscular atrophy. Nucleic Acid Ther 27(3):130\u2013143. \nhttps:\/\/doi.org\/10.1089\/nat.2016.0652","journal-title":"Nucleic Acid Ther"},{"issue":"24","key":"5_CR75","doi-asserted-by":"publisher","first-page":"3909","DOI":"10.1093\/hmg\/ddn293","volume":"17","author":"H Yin","year":"2008","unstructured":"Yin H, Moulton HM, Seow Y et al (2008) Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet 17(24):3909\u20133918. \nhttps:\/\/doi.org\/10.1093\/hmg\/ddn293","journal-title":"Hum Mol Genet"},{"issue":"16","key":"5_CR76","doi-asserted-by":"publisher","first-page":"4213","DOI":"10.1073\/pnas.1613203114","volume":"114","author":"Y Echigoya","year":"2017","unstructured":"Echigoya Y, Nakamura A, Nagata T et al (2017) Effects of systemic multiexon skipping with peptide-conjugated morpholinos in the heart of a dog model of Duchenne muscular dystrophy. Proc Natl Acad Sci U S A 114(16):4213\u20134218. \nhttps:\/\/doi.org\/10.1073\/pnas.1613203114","journal-title":"Proc Natl Acad Sci U S A"},{"key":"5_CR77","doi-asserted-by":"publisher","first-page":"507","DOI":"10.1146\/annurev-bioeng-071910-124709","volume":"13","author":"L Huang","year":"2011","unstructured":"Huang L, Liu Y (2011) In vivo delivery of RNAi with lipid-based nanoparticles. Annu Rev Biomed Eng 13:507\u2013530. \nhttps:\/\/doi.org\/10.1146\/annurev-bioeng-071910-124709","journal-title":"Annu Rev Biomed Eng"},{"issue":"8","key":"5_CR78","doi-asserted-by":"publisher","first-page":"2720","DOI":"10.1021\/mp500259e","volume":"11","author":"A Babu","year":"2014","unstructured":"Babu A, Wang Q, Muralidharan R et al (2014) Chitosan coated polylactic acid nanoparticle-mediated combinatorial delivery of cisplatin and siRNA\/plasmid DNA chemosensitizes cisplatin-resistant human ovarian cancer cells. Mol Pharm 11(8):2720\u20132733. \nhttps:\/\/doi.org\/10.1021\/mp500259e","journal-title":"Mol Pharm"},{"key":"5_CR79","doi-asserted-by":"publisher","first-page":"553","DOI":"10.1093\/nass\/nrn280","volume":"52","author":"PP Seth","year":"2008","unstructured":"Seth PP, Siwkowski A, Allerson CR et al (2008) Design, synthesis and evaluation of constrained methoxyethyl (cMOE) and constrained ethyl (cEt) nucleoside analogs. Nucleic Acids Symp Ser (Oxf) 52:553\u2013554. \nhttps:\/\/doi.org\/10.1093\/nass\/nrn280","journal-title":"Nucleic Acids Symp Ser (Oxf)"},{"issue":"6","key":"5_CR80","doi-asserted-by":"publisher","first-page":"640","DOI":"10.1038\/s41587-019-0106-2","volume":"37","author":"W Shen","year":"2019","unstructured":"Shen W, De Hoyos CL, Migawa MT et al (2019) Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index. Nature biotechnology:1. Nat Biotechnol 37(6):640\u2013650","journal-title":"Nat Biotechnol"},{"issue":"1","key":"5_CR81","doi-asserted-by":"publisher","first-page":"9","DOI":"10.1038\/nrneurol.2017.148","volume":"14","author":"C Rinaldi","year":"2018","unstructured":"Rinaldi C, Wood MJA (2018) Antisense oligonucleotides: the next frontier for treatment of neurological disorders. Nat Rev Neurol 14(1):9\u201321. \nhttps:\/\/doi.org\/10.1038\/nrneurol.2017.148","journal-title":"Nat Rev Neurol"}],"container-title":["Methods in Molecular Biology","Gapmers"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/978-1-0716-0771-8_5","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2020,8,31]],"date-time":"2020-08-31T07:04:24Z","timestamp":1598857464000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/978-1-0716-0771-8_5"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020]]},"ISBN":["9781071607701","9781071607718"],"references-count":81,"URL":"https:\/\/doi.org\/10.1007\/978-1-0716-0771-8_5","relation":{},"ISSN":["1064-3745","1940-6029"],"issn-type":[{"value":"1064-3745","type":"print"},{"value":"1940-6029","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020]]},"assertion":[{"value":"1 September 2020","order":1,"name":"first_online","label":"First Online","group":{"name":"ChapterHistory","label":"Chapter History"}}]}}