Gene silencing approaches for the management of dyslipidaemia

Trends in Pharmacological Sciences

Volumn 34, Issue 4, 2013, Pages 198-205

  Norata, Giuseppe Danilo ^a,b,c   Tibolla, Gianpaolo ^a,d   Catapano, Alberico Luigi ^a,d  

^a UNIVERSITY OF MILAN   (Italy)

^b Ospedale Bassini   (Italy)

^c QUEEN MARY UNIVERSITY OF LONDON   (United Kingdom)

^d IRCCS MULTIMEDICA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ACYLTRANSFERASE; ALN PCS; ALN PCS 02; ANGIOPOIETIN LIKE PROTEIN 3; APOCIIIRX; APOLIPOPROTEIN A; APOLIPOPROTEIN B; APOLIPOPROTEIN C3; ASO 144367; ASO BMS PCSK9RX; DIACYLGLYCEROL ACYL TRANSFERASE 1; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; KEXIN; LCQ 908; LIPOPROTEIN; LNA SPC 5001; MICRORNA; MICRORNA 33; MIPOMERSEN; NUCLEIC ACID; OLIGODEOXYNUCLEOTIDE DERIVATIVE; SMALL INTERFERING RNA; SUBTILISIN; UNCLASSIFIED DRUG;

CARDIOVASCULAR RISK; DRUG MEGADOSE; DRUG RESPONSE; DRUG TOLERABILITY; DYSLIPIDEMIA; GENE; GENE SILENCING; HUMAN; LIPID BLOOD LEVEL; LIPID METABOLISM; NONHUMAN; PCSK9 GENE; PRIORITY JOURNAL; REVIEW; RNA INTERFERENCE;

ANIMALS; DYSLIPIDEMIAS; GENE SILENCING; GENETIC THERAPY; HUMANS; MICRORNAS;

EID: 84876023437     PISSN: 01656147     EISSN: 18733735     Source Type: Journal    
DOI: 10.1016/j.tips.2013.01.010     Document Type: Review

References (59)

1. A.L. Catapano 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 Suppl. 1 2011 S1 S44
2. R.M. Crooke Antisense oligonucleotides as therapeutics for hyperlipidaemias Expert Opin. Biol. Ther. 5 2005 907 917
3. M.E. Visser Antisense oligonucleotides for the treatment of dyslipidaemia Eur. Heart J. 33 2012 1451 1458
4. A. Fire Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans Nature 391 1998 806 811
5. J. Martinez Single-stranded antisense siRNAs guide target RNA cleavage in RNAi Cell 110 2002 563 574
6. S.T. Crooke Progress in antisense technology Annu. Rev. Med. 55 2004 61 95
7. J.M. Campbell Oligodeoxynucleoside phosphorothioate stability in subcellular extracts, culture media, sera and cerebrospinal fluid J. Biochem. Biophys. Methods 20 1990 259 267
8. J. Kurreck Antisense technologies. Improvement through novel chemical modifications Eur. J. Biochem. 270 2003 1628 1644
9. R.Z. Yu Cross-species comparison of in vivo PK/PD relationships for second-generation antisense oligonucleotides targeting apolipoprotein B-100 Biochem. Pharmacol. 77 2009 910 919
10. D.A. Braasch Biodistribution of phosphodiester and phosphorothioate siRNA Bioorg. Med. Chem. Lett. 14 2004 1139 1143
11. K.A. Whitehead Knocking down barriers: advances in siRNA delivery Nat. Rev. Drug Discov. 8 2009 129 138
12. A. Akinc A combinatorial library of lipid-like materials for delivery of RNAi therapeutics Nat. Biotechnol. 26 2008 561 569
13. B. Shi Biodistribution of small interfering RNA at the organ and cellular levels after lipid nanoparticle-mediated delivery J. Histochem. Cytochem. 59 2011 727 740
14. R.M. Crooke An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis J. Lipid Res. 46 2005 872 884
15. F. Akdim Efficacy of apolipoprotein B synthesis inhibition in subjects with mild-to-moderate hyperlipidaemia Eur. Heart J. 32 2011 2650 2659
16. M.E. Visser Mipomersen, an apolipoprotein B synthesis inhibitor, lowers low-density lipoprotein cholesterol in high-risk statin-intolerant patients: a randomized, double-blind, placebo-controlled trial Eur. Heart J. 33 2012 1142 1149
17. B.G. Nordestgaard Lipoprotein(a) as a cardiovascular risk factor: current status Eur. Heart J. 31 2010 2844 2853
18. R. Clarke Genetic variants associated with Lp(a) lipoprotein level and coronary disease N. Engl. J. Med. 361 2009 2518 2528
19. P.R. Kamstrup Genetically elevated lipoprotein(a) and increased risk of myocardial infarction JAMA 301 2009 2331 2339
20. E. Merki Antisense oligonucleotide lowers plasma levels of apolipoprotein (a) and lipoprotein (a) in transgenic mice J. Am. Coll. Cardiol. 57 2011 1611 1621
21. M. Abifadel Mutations in PCSK9 cause autosomal dominant hypercholesterolemia Nat. Genet. 34 2003 154 156
22. A.J. Hooper The C679X mutation in PCSK9 is present and lowers blood cholesterol in a Southern African population Atherosclerosis 193 2007 445 448
23. Z. Zhao Molecular characterization of loss-of-function mutations in PCSK9 and identification of a compound heterozygote Am. J. Hum. Genet. 79 2006 514 523
24. K.E. Berge Missense mutations in the PCSK9 gene are associated with hypocholesterolemia and possibly increased response to statin therapy Arterioscler. Thromb. Vasc. Biol. 26 2006 1094 1100
25. S. Rashid Decreased plasma cholesterol and hypersensitivity to statins in mice lacking Pcsk9 Proc. Natl. Acad. Sci. U.S.A. 102 2005 5374 5379
26. S.W. Park Post-transcriptional regulation of low density lipoprotein receptor protein by proprotein convertase subtilisin/kexin type 9a in mouse liver J. Biol. Chem. 279 2004 50630 50638
27. G. Tibolla Proprotein convertase subtilisin/kexin type 9 (PCSK9): from structure-function relation to therapeutic inhibition Nutr. Metab. Cardiovasc. Dis. 21 2011 835 843
28. C.S.A. Dias Effects of AMG145, a fully human monoclonal antibody against PCSK9, on low-density lipoprotein cholesterol in subjects taking statins: a phase 1, randomized, double-blind, placebo-controlled, ascending study TSG-14 J. Am. Coll. Cardiol. (Suppl.) 59 2012 E1379
29. E.M. Roth Atorvastatin with or without an antibody to PCSK9 in primary hypercholesterolemia N. Engl. J. Med. 367 2012 1891 1900
30. M. Frank-Kamenetsky Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates Proc. Natl. Acad. Sci. U.S.A. 105 2008 11915 11920
31. M.J. Graham Antisense inhibition of proprotein convertase subtilisin/kexin type 9 reduces serum LDL in hyperlipidemic mice J. Lipid Res. 48 2007 763 767
32. N. Gupta A locked nucleic acid antisense oligonucleotide (LNA) silences PCSK9 and enhances LDLR expression in vitro and in vivo PLoS ONE 5 2010 e10682
33. E.A. Stein Effect of a monoclonal antibody to PCSK9 on LDL cholesterol N. Engl. J. Med. 366 2012 1108 1118
34. A.M. Krieg Targeting LDL cholesterol with LNA Mol. Ther. Nucleic Acids 1 2012 e6
35. T.I. Pollin A null mutation in human APOC3 confers a favorable plasma lipid profile and apparent cardioprotection Science 322 2008 1702 1705
36. D.P. Bartel MicroRNAs: genomics, biogenesis, mechanism, and function Cell 116 2004 281 297
37. G. Condorelli microRNAs in heart disease: putative novel therapeutic targets? Eur. Heart J. 31 2010 649 658
38. G.D. Norata MicroRNAs and lipoproteins: a connection beyond atherosclerosis? Atherosclerosis 2012 10.1016/j.atherosclerosis.2012.11.019
39. J. Elmen Antagonism of microRNA-122 in mice by systemically administered LNA-antimiR leads to up-regulation of a large set of predicted target mRNAs in the liver Nucleic Acids Res. 36 2008 1153 1162
40. C. Esau miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting Cell Metab. 3 2006 87 98
41. W.C. Tsai MicroRNA-122 plays a critical role in liver homeostasis and hepatocarcinogenesis J. Clin. Invest. 122 2012 2884 2897
42. S.H. Hsu Essential metabolic, anti-inflammatory, and anti-tumorigenic functions of miR-122 in liver J. Clin. Invest. 122 2012 2871 2883
43. S.H. Najafi-Shoushtari MicroRNA-33 and the SREBP host genes cooperate to control cholesterol homeostasis Science 328 2010 1566 1569
44. K.J. Rayner MiR-33 contributes to the regulation of cholesterol homeostasis Science 328 2010 1570 1573
45. M.S. Brown, and J.L. Goldstein The SREBP pathway: regulation of cholesterol metabolism by proteolysis of a membrane-bound transcription factor Cell 89 1997 331 340
46. A. Davalos miR-33a/b contribute to the regulation of fatty acid metabolism and insulin signaling Proc. Natl. Acad. Sci. U.S.A. 108 2011 9232 9237
47. K.J. Rayner Inhibition of miR-33a/b in non-human primates raises plasma HDL and lowers VLDL triglycerides Nature 478 2011 404 407
48. K.J. Rayner Antagonism of miR-33 in mice promotes reverse cholesterol transport and regression of atherosclerosis J. Clin. Invest. 121 2011 2921 2931
49. K.C. Vickers MicroRNA-27b is a regulatory hub in lipid metabolism and is altered in dyslipidemia Hepatology 2012 10.1002/hep.25846
50. C.T. Johansen Genetic determinants of plasma triglycerides J. Lipid Res. 52 2011 189 206
51. H.R. Wurie Diacylglycerol acyltransferase 2 acts upstream of diacylglycerol acyltransferase 1 and utilizes nascent diglycerides and de novo synthesized fatty acids in HepG2 cells FEBS J. 279 2012 3033 3047
52. A.M. Birch DGAT1 inhibitors as anti-obesity and anti-diabetic agents Curr. Opin. Drug Discov. Dev. 13 2010 489 496
53. J.T. Haas DGAT1 mutation is linked to a congenital diarrheal disorder J. Clin. Invest. 122 2012 4680 4684
54. M.C. Meuwese ACAT inhibition and progression of carotid atherosclerosis in patients with familial hypercholesterolemia: the CAPTIVATE randomized trial JAMA 301 2009 1131 1139
55. J.M. Brown Targeted depletion of hepatic ACAT2-driven cholesterol esterification reveals a non-biliary route for fecal neutral sterol loss J. Biol. Chem. 283 2008 10522 10534
56. -/- mice Arterioscler. Thromb. Vasc. Biol. 26 2006 1814 1820
57. E. Demetz Inhibition of hepatic scavenger receptor-class B type I by RNA interference decreases atherosclerosis in rabbits Atherosclerosis 222 2012 360 366
58. T. Miida, and S. Hirayama Impacts of angiopoietin-like proteins on lipoprotein metabolism and cardiovascular events Curr. Opin. Lipidol. 21 2010 70 75
59. L. Pisciotta Characterization of three kindreds with familial combined hypolipidemia caused by loss-of-function mutations of ANGPTL3 Circ. Cardiovasc. Genet. 5 2011 42 50