Using microRNA as an alternative treatment for hyperlipidemia and cardiovascular disease: Cardio-miRs in the pipeline

Journal of Cardiovascular Pharmacology

Volumn 62, Issue 3, 2013, Pages 247-254

  Hennessy, Elizabeth J ^a   Moore, Kathryn J ^a  

^a NEW YORK UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

MicroRNA; Therapeutics

Indexed keywords

ABC TRANSPORTER A1; ANTISENSE OLIGONUCLEOTIDE; APOLIPOPROTEIN B; APOLIPOPROTEIN B100; APOLIPOPROTEIN C3; C REACTIVE PROTEIN; DRUG CARRIER; HYDROXYMETHYLGLUTARYL COENZYME A REDUCTASE INHIBITOR; KEXIN; LENTIVIRUS VECTOR; LIPOSOME; LOMITAPIDE; LOW DENSITY LIPOPROTEIN CHOLESTEROL; MGN 1374; MGN 9103; MICRORNA; MICRORNA 107; MICRORNA 122; MICRORNA 208; MICRORNA 21; MICRORNA 33; MICRORNA 33 INHIBITOR; MICRORNA INHIBITOR; MICRORNA MIMETIC; MICROSOMAL TRIGLYCERIDE TRANSFER PROTEIN; MIPOMERSEN; MIRAVIRSEN; SMALL INTERFERING RNA; SUBTILISIN; UNCLASSIFIED DRUG; UNINDEXED DRUG;

ANTIVIRAL ACTIVITY; CARDIOVASCULAR DISEASE; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG MECHANISM; DRUG TARGETING; DRUG TOLERABILITY; FAMILIAL HYPERCHOLESTEROLEMIA; FATTY LIVER; GENE EXPRESSION REGULATION; GENE OVEREXPRESSION; GENE SILENCING; GENE TARGETING; HEART INFARCTION; HEART MUSCLE FIBROSIS; HEPATITIS C; HUMAN; INJECTION SITE REACTION; LIVER TOXICITY; METABOLIC DISORDER; METABOLIC SYNDROME X; MICELLE; MYOPATHY; NON INSULIN DEPENDENT DIABETES MELLITUS; NONHUMAN; PRIORITY JOURNAL; REVIEW; RNA DEGRADATION; RNA INTERFERENCE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; APOLIPOPROTEINS B; CARDIOVASCULAR DISEASES; GENE EXPRESSION REGULATION; GENETIC THERAPY; HUMANS; HYPERLIPIDEMIAS; MICRORNAS; OLIGONUCLEOTIDES, ANTISENSE; RNA, SMALL INTERFERING; THERAPIES, INVESTIGATIONAL; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 84884672205     PISSN: 01602446     EISSN: 15334023     Source Type: Journal    
DOI: 10.1097/FJC.0b013e31829d48bf     Document Type: Review

References (80)

1. Go AS, et al. Heart disease and stroke statistics - 2013 update: A report from the American Heart Association. Circulation. 2013;127:e6-e245
2. Harper CR, Jacobson TA. The broad spectrum of statin myopathy: From myalgia to rhabdomyolysis. Curr Opin Lipidol. 2007;18:401-408
3. Maningat P, Breslow JL. Needed: Pragmatic clinical trials for statinintolerant patients. N Engl J Med. 2011;365:2250-2251
4. The ENCODE (ENCyclopedia of DNA Elements) Project. Science. 2004;306:636-640
5. Dunham I, et al. An integrated encyclopedia of DNA elements in the human genome. Nature. 2012;489:57-74
6. Aartsma-Rus A, et al. Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms. Mol Ther. 2009;17: 548-553
7. Baker BF, et al. 2'-O-(2-Methoxy)ethyl-modified anti-intercellular adhesion molecule 1 (ICAM-1) oligonucleotides selectively increase the ICAM-1 mRNA level and inhibit formation of the ICAM-1 translation initiation complex in human umbilical vein endothelial cells. J Biol Chem. 1997;272:11994-12000
8. Chan JH, Lim S, Wong WS. Antisense oligonucleotides: From design to therapeutic application. Clin Exp Pharmacol Physiol. 2006;33:533-540
9. Bell DA, Hooper AJ, Burnett Jr. Mipomersen, an antisense apolipoprotein B synthesis inhibitor. Expert Opin Investig Drugs. 2011;20:265-272
10. Gebhard C, Huard G, Kritikou EA, et al. Apolipoprotein B antisense inhibition - update on mipomersen. Curr Pharm Des. 2013
11. Vogt A, Parhofer KG. The potential of mipomersen, an ApoB synthesis inhibitor, to reduce necessity for LDL-apheresis in patients with heterozygous familial hypercholesterolemia and coronary artery disease. Expert Opin Pharmacother. 2013
12. Gregory RI, Chendrimada TP, Cooch N, et al. Human RISC couples microRNA biogenesis and posttranscriptional gene silencing. Cell. 2005; 123:631-640
13. Qiu S, Adema CM, Lane T. A computational study of off-target effects of RNA interference. Nucleic Acids Res. 2005;33:1834-1847
14. Obbard DJ, Gordon KH, Buck AH, et al. The evolution of RNAi as a defence against viruses and transposable elements. Philos Trans R Soc Lond B Biol Sci. 2009;364:99-115
15. Rivera S, Yuan F. Critical issues in delivery of RNAi therapeutics in vivo. Curr Pharm Biotechnol. 2012;13:1279-1291
16. Lee RC, Feinbaum RL, Ambros V. The C. elegans heterochronic gene lin-4 encodes small RNAs with antisense complementarity to lin-14. Cell. 1993;75:843-854
17. Wightman B, Ha I, Ruvkun G. Posttranscriptional regulation of the heterochronic gene lin-14 by lin-4 mediates temporal pattern formation in C. elegans. Cell. 1993;75:855-862
18. Kozomara A, Griffiths-Jones S. miRBase: Integrating microRNA annotation and deep-sequencing data. Nucleic Acids Res. 2011;39:D152-D157
19. Selbach M, et al. Widespread changes in protein synthesis induced by microRNAs. Nature. 2008;455:58-63
20. Yang JS, et al. Widespread regulatory activity of vertebrate microRNA. * species. RNA. 2011;17:312-326
21. Mah SM, Buske C, Humphries RK, et al. miRNA*: A passenger stranded in RNA-induced silencing complex? Crit Rev Eukaryot Gene Expr. 2010;20:141-148
22. Marco A, Macpherson JI, Ronshaugen M, et al. MicroRNAs from the same precursor have different targeting properties. Silence. 2012;3:8
23. Yao B, La LB, Chen YC, et al. Defining a new role of GW182 in maintaining miRNA stability. EMBO Rep. 2012;13:1102-1108
24. Sacco LD, Masotti A. Recent insights and novel bioinformatics tools to understand the role of microRNAs binding to 5' untranslated region. Int J Mol Sci. 2012;14:480-495
25. Reczko M, Maragkakis M, Alexiou P, et al. Functional microRNA targets in protein coding sequences. Bioinformatics. 2012;28:771-776
26. Orom UA, Nielsen FC, Lund AH. MicroRNA-10a binds the 5'UTR of ribosomal protein mRNAs and enhances their translation. Mol Cell. 2008;30:460-471
27. Chekulaeva M, et al. miRNA repression involves GW182-mediated recruitment of CCR4-NOT through conserved W-containing motifs. Nat Struct Mol Biol. 2011;18:1218-1226
28. Wu L, Fan J, Belasco JG. MicroRNAs direct rapid deadenylation of mRNA. Proc Natl Acad Sci U S A 2006;103:4034-4039
29. Guo H, Ingolia NT, Weissman JS, et al. Mammalian microRNAs predominantly act to decrease target mRNA levels. Nature. 2010;466:835-840
30. Bazzini AA, Lee MT, Giraldez AJ. Ribosome profiling shows that miR- 430 reduces translation before causing mRNA decay in zebrafish. Science. 2012;336:233-237
31. Djuranovic S, Nahvi A, Green R. miRNA-mediated gene silencing by translational repression followed by mRNA deadenylation and decay. Science. 2012;336:237-240
32. Friedman RC, Farh KK, Burge CB, et al. Most mammalian mRNAs are conserved targets of microRNAs. Genome Res. 2009;19:92-105
33. Lewis BP, Burge CB, Bartel DP. Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets. Cell. 2005;120:15-20
34. Bartel DP. MicroRNAs: Target recognition and regulatory functions. Cell. 2009;136:215-233
35. Grimson A, et al. MicroRNA targeting specificity in mammals: Determinants beyond seed pairing. Mol Cell. 2007;27:91-105
36. Ambros V. The functions of animal microRNAs. Nature. 2004;431:350-355
37. Kloosterman WP, Plasterk RH. The diverse functions of microRNAs in animal development and disease. Dev Cell. 2006;11:441-450
38. Baek D, et al. The impact of microRNAs on protein output. Nature. 2008; 455:64-71
39. Chi SW, Zang JB, Mele A, et al. Argonaute HITS-CLIP decodes micro- RNA-mRNA interaction maps. Nature. 2009;460:479-486
40. Lal A, et al. Capture of microRNA-bound mRNAs identifies the tumor suppressor miR-34a as a regulator of growth factor signaling. PLoS Genet. 2011;7:e1002363
41. Orom UA, Lund AH. Isolation of microRNA targets using biotinylated synthetic microRNAs. Methods. 2007;43:162-165
42. Easow G, Teleman AA, Cohen SM. Isolation of microRNA targets by miRNP immunopurification. RNA. 2007;13:1198-1204
43. Broderick JA, Zamore PD. MicroRNA therapeutics. Gene Ther. 2011;18: 1104-1110
44. Grunweller A, Hartmann RK. Locked nucleic acid oligonucleotides: The next generation of antisense agents? Bio Drugs. 2007;21:235-243
45. Semple SC, et al. Rational design of cationic lipids for siRNA delivery. Nat Biotechnol. 2010;28:172-176
46. Love KT, et al. Lipid-like materials for low-dose, in vivo gene silencing. Proc Natl Acad Sci U S A 2010;107:1864-1869
47. Soutschek J, et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature. 2004;432:173-178
48. Stenvang J, Petri A, Lindow M, et al. Inhibition of microRNA function by antimiR oligonucleotides. Silence. 2012;3:1
49. van Rooij E, Olson EN.MicroRNA therapeutics for cardiovascular disease: Opportunities and obstacles. Nat Rev Drug Discov. 2012;11:860-872
50. Dorn GW II. Therapeutic potential of microRNAs in heart failure. Curr Cardiol Rep. 2010;12:209-215
51. Krutzfeldt J, et al. Silencing of microRNAs in vivo with 'antagomirs'. Nature. 2005;438:685-689
52. Jopling CL, Yi M, Lancaster AM, et al.Modulation of hepatitis C virus RNA abundance by a liver-specific MicroRNA. Science. 2005;309:1577-1581
53. Lanford RE, et al. Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science. 2010;327:198-201
54. Janssen HL, et al. Treatment of HCV infection by targeting microRNA. N Engl J Med. 2013
55. Janssen HL, et al. A randomized, double-blind, placebo (plb) controlled safety and anti-viral proof of concept study of miravirsen (MIR), an oligonucleotide targeting miR-122, in treatment naiv́ e patients with genotype 1 (gt1) chronic HCV infection. Paper presented at: The 62nd Annual Meeting of the American Association for the Study of Liver Diseases: The Liver Meeting; 2011; San Francisco, CA.
56. Esau C, et al. miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting. Cell Metab. 2006;3:87-98
57. Ebert MS, Neilson JR, Sharp PA. MicroRNA sponges: Competitive inhibitors of small RNAs in mammalian cells. Nat Methods. 2007;4:721-726
58. Kluiver J, et al. Generation of miRNA sponge constructs. Methods. 2012; 58:113-117
59. Lu J, et al. MicroRNA expression profiles classify human cancers. Nature. 2005;435:834-838
60. Vickers KC, Palmisano BT, Shoucri BM, et al. MicroRNAs are transported in plasma and delivered to recipient cells by high-density lipoproteins. Nat Cell Biol. 2011;13:423-433
61. Gentner B, et al. Stable knockdown of microRNA in vivo by lentiviral vectors. Nat Methods. 2009;6:63-66
62. Rayner KJ, et al. MiR-33 contributes to the regulation of cholesterol homeostasis. Science. 2010;328:1570-1573
63. Rayner KJ, et al. Antagonism of miR-33 in mice promotes reverse cholesterol transport and regression of atherosclerosis. J Clin Invest. 2011; 121:2921-2931
64. Geisler A, et al. microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors. Gene Ther. 2011;18:199-209
65. Montgomery RL, et al. Therapeutic inhibition of miR-208a improves cardiac function and survival during heart failure. Circulation. 2011; 124:1537-1547
66. Grueter CE, et al. A cardiac microRNA governs systemic energy homeostasis by regulation of MED13. Cell. 2012;149:671-683
67. Conaway RC, Conaway JW. Function and regulation of the Mediator complex. Curr Opin Genet Dev. 2011;21:225-230
68. Rayner KJ, et al. Inhibition of miR-33a/b in non-human primates raises plasma HDL and lowers VLDL triglycerides. Nature. 2011;478:404-407
69. Trajkovski M, et al. MicroRNAs 103 and 107 regulate insulin sensitivity. Nature. 2011;474:649-653
70. van Rooij E, et al. Dysregulation of microRNAs after myocardial infarction reveals a role of miR-29 in cardiac fibrosis. Proc Natl Acad Sci U S A 2008;105:13027-13032
71. Thum T, et al. MicroRNA-21 contributes to myocardial disease by stimulating MAP kinase signalling in fibroblasts. Nature. 2008;456:980-984
72. Chau BN, et al. MicroRNA-21 promotes fibrosis of the kidney by silencing metabolic pathways. Sci Transl Med. 2012;4:121ra118
73. Patrick DM, et al. Stress-dependent cardiac remodeling occurs in the absence of microRNA-21 in mice. J Clin Invest. 2010;120:3912-3916
74. Morrisey EE. The magic and mystery of miR-21. J Clin Invest. 2010; 120:3817-3819
75. Hullinger TG, et al. Inhibition of miR-15 protects against cardiac ischemic injury. Circ Res. 2012;110:71-81
76. van Rooij E, et al. A signature pattern of stress-responsive microRNAs that can evoke cardiac hypertrophy and heart failure. Proc Natl Acad Sci U S A 2006;103:18255-18260
77. Sun X, Feinberg MW. Knocking out viral myocarditis: Reality or a miRage? Circ Res. 2012;111:388-391
78. Corsten MF, et al. MicroRNA profiling identifies microRNA-155 as an adverse mediator of cardiac injury and dysfunction during acute viral myocarditis. Circ Res. 2012;111:415-425
79. Caruso P, et al. A role for miR-145 in pulmonary arterial hypertension: Evidence from mouse models and patient samples. Circ Res. 2012;111: 290-300
80. Bernardo BC, et al. Therapeutic inhibition of the miR-34 family attenuates pathological cardiac remodeling and improves heart function. Proc Natl Acad Sci U S A 2012;109:17615-17620