Antisense therapy for cardiovascular diseases

Current Pharmaceutical Design

Volumn 21, Issue 30, 2015, Pages 4417-4426

  Phillips, M Ian ^a   Costales, Jessica ^a   Lee, Robert J ^a   Oliveira, Edilamar ^b   Burns, Andrew B ^a  

^a KECK GRADUATE INSTITUTE   (United States)

^b UNIVERSITY OF SÃO PAULO   (Brazil)

Author keywords

Antisense oligonucleotides; Eteplirsen ; Exon skipping; Hyperlipidemia; Hypertension; Kynamro ; Metabolic syndrome

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MIPOMERSEN;

ANTISENSE THERAPY; ARTICLE; CARDIOVASCULAR DISEASE; DRUG MECHANISM; DRUG UPTAKE; DUCHENNE MUSCULAR DYSTROPHY; EXON; EXON SKIPPING; FAMILIAL HYPERCHOLESTEROLEMIA; HEART FUNCTION; HEMORRHAGIC FEVER; HUMAN; HYPERCHOLESTEROLEMIA; HYPERGLYCEMIA; HYPERLIPIDEMIA; HYPERTENSION; METABOLIC SYNDROME X; OBESITY; PRIORITY JOURNAL; CARDIOVASCULAR DISEASES; GENETICS; MUTATION;

CARDIOVASCULAR DISEASES; EXONS; HUMANS; MUTATION; OLIGONUCLEOTIDES, ANTISENSE;

EID: 84946054384     PISSN: 13816128     EISSN: 18734286     Source Type: Journal    
DOI: 10.2174/1381612821666150803150402     Document Type: Article

References (68)

1. Zamecnik PC, Stephenson ML. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. Proc Natl Acad Sci USA 1978; 75(1): 280-4.
2. Reynolds T. First antisense drug trials planned in leukemia. J Natl Cancer Inst 1992; 84(5): 288-90.
3. Phillips MI, Wielbo D, Gyurko R. Antisense inhibition of hypertension: a new strategy for renin-angiotensin candidate genes. Kidney Int 1994; 46(6): 1554-6.
4. Crooke ST. Molecular mechanisms of antisense drugs: RNase H. Antisense Nucleic Acid Drug Develop 1998; 8(2): 133-4.
5. Food and Drug Agency. FDA approves new orphan drug Kynamro to treat inherited cholesterol disorder 2013; Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm 337195.htm, 2015.
6. Crooke ST. Vitravene™—another piece in the mosaic. Antisense Nucleic Acid Drug Develop 1998; 8(4): vii-viii.
7. Kole R, Krainer AR, Altman S. RNA therapeutics: beyond RNA interference and antisense oligonucleotides. Nat Rev Drug Discov 2012; 11(2): 125-40.
8. Dias N, Stein CA. Antisense oligonucleotides: basic concepts and mechanisms. Mol Cancer Ther 2002; 1(5): 347-55.
9. Phillips MI Antisense a promise waiting to be filled In. Antisense Therapeutics. 2nd ed. Humana Press 2005; pp. 3-4.
10. Bennett CF, Swayze EE. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu Rev Pharmacol Toxicol 2010; 50: 259-93.
11. Wu H, Lima WF, Zhang H, Fan A, Sun H, Crooke ST. Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs. J Biol Chem 2004; 279(17): 17181-9.
12. Vickers TA, Koo S, Bennett CF, Crooke ST, Dean NM, Baker BF. Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents. A comparative analysis. J Biol Chem 2003; 278(9): 7108-18.
13. Aartsma-Rus A, Kaman W, Bremmer-Bout M, et al. Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells. Gene Ther 2004; 11(18): 1391-8.
14. Phillips MI, Mohuczy-Dominiak D, Coffey M, et al. Prolonged reduction of high blood pressure with an in vivo, nonpathogenic, adeno-associated viral vector delivery of AT1-R mRNA antisense. Hypertension 1997; 29(1 Pt 2): 374-80.
15. Juliano R, Alam MR, Dixit V, Kang H. Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides. Nucleic Acids Res 2008; 36(12): 4158-71.
16. ISIS Pharmaceuticals. Platform. 2015; Available at: http://isispharm.com/backup/Antisense- Technology/Publications/Category-48.htm, 2015.
17. Idera. Platform. 2015; Available at: http://www.iderapharma.com/, 2015.
18. Sarepta Therapeutics. Platforms. 2015; Available at: Available from http://www.sarepta.com/technology/technology-platform, 2015.
19. Li B, Hughes JA, Phillips MI. Uptake and efflux of intact antisense phosphorothioate deoxyoligonucleotide directed against angiotensin receptors in bovine adrenal cells. Neurochem Int 1997; 31(3): 393-403.
20. Chan JH, Lim S, Wong W. Antisense oligonucleotides: from design to therapeutic application. Clin Exp Pharmacol Physiol 2006; 33(5_6): 533-40.
21. Wojtkowiak-Szlachcic A, Taylor K, Stepniak-Konieczna E, et al. Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy. Nucleic Acids Res 2015; 43(6): 3318-31.
22. Aartsma-Rus A, van Ommen GJ. Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA 2007; 13(10): 1609-24.
23. Lennox KA, Owczarzy R, Thomas DM, Walder JA, Behlke MA. Improved performance of anti-miRNA oligonucleotides using anovel non-nucleotide modifier. Mol Ther Nucleic Acids 2013; 2(8): e117.
24. Zhang YC, Bui JD, Shen L, Phillips MI. Antisense inhibition of beta(1)-adrenergic receptor mRNA in a single dose produces a profound and prolonged reduction in high blood pressure in spontaneously hypertensive rats. Circulation 2000; 101(6): 682-8.
25. Visser ME, Witztum JL, Stroes ES, Kastelein JJ. Antisense oligonucleotides for the treatment of dyslipidaemia. Eur Heart J 2012; 33(12): 1451-8.
26. Volberding P. Global HIV/AIDS Medicine: Elsevier Health Sciences 2008.
27. ISIS. Isis Pharmaceuticals and Eli Lilly and Company Announce Results of Affinitak(TM) Phase III Clinical Trial in Non-Small Cell Lung Cancer. 2002; Available at: http://ir.isispharm.com/phoenix.zhtml?c=222170&p=irol-newsArticle_pf&ID=1290234&highlight =, 2015.
28. Agrawal S, Crooke ST. Antisense research and application. USA: Springer Science & Business Media 1998.
29. Farman CA, Kornbrust DJ. Oligodeoxynucleotide studies in primates: antisense and immune stimulatory indications. Toxicol Pathol 2003; 31 Suppl: 119-22.
30. ISIS Pharmaceuticals. Pipeline. 2015; http://www.isispharm.com/Pipeline/index.htm 2015.
31. Sarepta Therapeutics. Pipeline. 2015; http: //www.sarepta.com/ourpipeline 2015.
32. Nguyen NT, Magno CP, Lane KT, Hinojosa MW, Lane JS. Association of hypertension, diabetes, dyslipidemia, and metabolic syndrome with obesity: findings from the National Health and Nutrition Examination Survey, 1999 to 2004. J Am Coll Surg 2008; 207(6): 928-34.
33. Wilson C, Tran M, Salazar K, Young M, Taegtmeyer H. Western diet, but not high fat diet, causes derangements of fatty acid metabolism and contractile dysfunction in the heart of Wistar rats. Biochem J 2007; 406: 457-67.
34. Phillips MI. Gene therapy for hypertension: sense and antisense strategies. Expert Opin Biol Ther 2001; 1(4): 655-62.
35. Gyurko R, Wielbo D, Phillips MI. Antisense inhibition of AT 1 receptor mRNA and angiotensinogen mRNA in the brain of spontaneously hypertensive rats reduces hypertension of neurogenic origin. Regul Pept 1993; 49(2): 167-74.
36. Kalra SP, Bagnasco M, Otukonyong EE, Dube MG, Kalra PS. Rhythmic, reciprocal ghrelin and leptin signaling: new insight in the development of obesity. Regul Pept 2003; 111(1): 1-11.
37. Sloop KW, Cao JX, Siesky AM, et al. Hepatic and glucagon-like peptide-1-mediated reversal of diabetes by glucagon receptor antisense oligonucleotide inhibitors. J Clin Invest 2004; 113(11): 1571-81.
38. Elchebly M, Payette P, Michaliszyn E, et al. Increased insulin sensitivity and obesity resistance in mice lacking the protein tyrosine phosphatase-1B gene. Science 1999; 283(5407): 1544-8.
39. Liu G. Technology evaluation: ISIS-113715, Isis. Curr Opin Mol Ther 2004; 6(3): 331-6.
40. Jeong HS, Park KK, Park KK, et al. Effect of antisense TGF-beta1 oligodeoxynucleotides in streptozotocin- induced diabetic rat kidney. J Korean Med Sci 2004; 19(3): 374-83.
41. Mehta JL, Sanada N, Hu CP, et al. Deletion of LOX-1 reduces atherogenesis in LDLR knockout mice fed high cholesterol diet. Circ Res 2007; 100(11): 1634-42.
42. Tang X, Mohuczy D, Zhang YC, Kimura B, Galli SM, Phillips MI. Intravenous angiotensinogen antisense in AAV-based vector decreases hypertension. Am J Physiol 1999; 277(6 Pt 2): H2392-9.
43. Gyurko R, Tran D, Phillips MI. Time course of inhibition of hypertension by antisense oligonucleotides targeted to AT1 angiotensin receptor mRNA in spontaneously hypertensive rats. Am J Hypertens 1997; 10(S4): 56S-62.
44. US patent 6,248,724, Moore MD, Phillips MI, Mohuczy D. Antisense oligonucleotide compositions targeted to angiotensin converting enzyme MRNA and methods of use 2001.
45. US patent 6,489,307. Phillips MI, Zhang Y. Reducing the level of transcription of beta 1-adrenoceptor polypeptide-specific mRNA in a mammalian host cell, by administering oligonucleotide; reduces receptor expression; hypotensive effect 2002.
46. Krakoff LR, Gillespie RL, Ferdinand KC, et al. Hypertension recommendations from the eighth joint national committee panel members raise concerns for elderly black and female populations. J Am Coll Cardiol 2014; 64(4): 394-402.
47. Szalai AJ, McCrory MA, Xing D, et al. Inhibiting C-reactive protein for the treatment of cardiovascular disease: promising evidence from rodent models. Mediators Inflamm 2014; 2014: 353614.
48. Food and Drug Association. Clinical Trials Searchable Database. 2015; Available at: Clinicaltrials.gov 2015.
49. Peng JF, Kimura B, Fregly MJ, Phillips MI. Reduction of coldinduced hypertension by antisense oligodeoxynucleotides to angiotensinogen mRNA and AT1-receptor mRNA in brain and blood. Hypertension 1998; 31(6): 1317-23.
50. Kagiyama S, Varela A, Phillips MI, Galli SM. Antisense inhibition of brain renin-angiotensin system decreased blood pressure in chronic 2-kidney, 1 clip hypertensive rats. Hypertension 2001; 37(2 Pt 2): 371-5.
51. Soutschek J, Akinc A, Bramlage B, et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 2004; 432(7014): 173-8.
52. Kimura B, Mohuczy D, Tang X, Phillips MI. Attenuation of hypertension and heart hypertrophy by adeno-associated virus delivering angiotensinogen antisense. Hypertension 2001; 37(2 Pt 2): 376-80.
53. Phillips MI. Gene therapy for hypertension: the preclinical data. Hypertension 2001; 38(3 Pt 2): 543-8.
54. Phillips MI. Antisense inhibition and adeno-associated viral vector delivery for reducing hypertension. Hypertension 1997; 29(1 Pt 2): 177-87.
55. Graham MJ, Lee RG, Bell TA, 3rd, et al. Antisense oligonucleotide inhibition of apolipoprotein C-III reduces plasma triglycerides in rodents, nonhuman primates, and humans. Circ Res 2013; 112(11): 1479-90.
56. Kiberstis PA. A surfeit of suspects. Science 2005; 307(5708): 369-9.
57. Yoon MJ, Lee GY, Chung JJ, Ahn YH, Hong SH, Kim JB. Adiponectin increases fatty acid oxidation in skeletal muscle cells by sequential activation of AMP-activated protein kinase, p38 mitogen- activated protein kinase, and peroxisome proliferator-activated receptor alpha. Diabetes 2006; 55(9): 2562-70.
58. Schmidt-Ott KM, Kagiyama S, Phillips MI. The multiple actions of angiotensin II in atherosclerosis. Regul Pept 2000; 93(1): 65-77.
59. Saonere J. Antisense therapy, a magic bullet for the treatment of various diseases: Present and future prospects. J Med Genetics Genom 2011; 3: 77-83.
60. Koo T, Wood MJ. Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy. Hum Gene Ther 2013; 24(5): 479-88.
61. Summerton J. Morpholino antisense oligomers: the case for an RNase H-independent structural type. Biochim Biophys Acta 1999; 1489(1): 141-58.
62. Song E, Lee S, Wang J, et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med 2003; 9(3): 347-51.
63. Cech TR. Ribozymes, the first 20 years. Biochem Soc Trans 2002; 30(Pt 6): 1162-6.
64. Osman EY, Miller MR, Robbins KL, et al. Morpholino antisense oligonucleotides targeting intronic repressor element 1 improve phenotype in SMA mouse models. Hum Mol Genet 2014; 23(18): 4832-45.
65. Milhavet O, Gary DS, Mattson MP. RNA interference in biology and medicine. Pharmacol Rev 2003; 55(4): 629-48.
66. Bates SH, Myers MG. The role of leptin®STAT3 signaling in neuroendocrine function: an integrative perspective. J Mol Med 2004; 82(1): 12-20.
67. Ueki K, Kondo T, Kahn CR. Suppressor of cytokine signaling 1 (SOCS-1) and SOCS-3 cause insulin resistance through inhibition of tyrosine phosphorylation of insulin receptor substrate proteins by discrete mechanisms. Mol Cell Biol 2004; 24(12): 5434-46.
68. Howard JK, Cave BJ, Oksanen LJ, Tzameli I, Bjørbæk C, Flier JS. Enhanced leptin sensitivity and attenuation of diet-induced obesity in mice with haploinsufficiency of Socs3. Nat Med 2004; 10(7): 734-8.