medigraphic.com
Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán

2018, Número 5

<< Anterior

Rev Invest Clin 2018; 70 (5)


New Biotechnological Treatments for Lipid Disorders

Valencia-Enciso N, Mendivil CO

Idioma: Ingles.
Referencias bibliográficas: 80
Paginas: 244-254
Archivo PDF: 111.88 Kb.


RESUMEN

Sin resumen.


REFERENCIAS (EN ESTE ARTÍCULO)

  1. Bruzzone G, Corbelli G, Belci P, et al. Cholesterol lowering therapy: treat to target or reduce the global risk? The unresolved problem of residual risk. Curr Pharm Des. 2016;22:5676-86.

  2. Turner RM, Yin P, Hanson A, et al. Investigating the prevalence, predictors, and prognosis of suboptimal statin use early after a non-ST elevation acute coronary syndrome. J Clin Lipidol. 2017; 11:204-14.

  3. Ajufo E, Rader DJ. New therapeutic approaches for familial hypercholesterolemia. Annu Rev Med. 2018;69:113-31.

  4. Chait A, Eckel RH. Lipids, lipoproteins, and cardiovascular disease: clinical pharmacology now and in the future. J Clin Endocrinol Metab. 2016;101:804-14.

  5. Abifadel M, Varret M, Rabès JP, et al. Mutations in PCSK9 cause autosomal dominant hypercholesterolemia. Nat Genet. 2003; 34:154-6.

  6. Seidah NG, Benjannet S, Wickham L, et al. The secretory proprotein convertase neural apoptosis-regulated convertase 1 (NARC-1): liver regeneration and neuronal differentiation. Proc Natl Acad Sci U S A. 2003;100:928-33.

  7. Maxwell KN, Breslow JL. Adenoviral-mediated expression of Pcsk9 in mice results in a low-density lipoprotein receptor knockout phenotype. Proc Natl Acad Sci U S A. 2004;101:7100-5.

  8. Cohen J, Pertsemlidis A, Kotowski IK, et al. Low LDL cholesterol in individuals of African descent resulting from frequent nonsense mutations in PCSK9. Nat Genet. 2005;37:161-5.

  9. Cohen JC, Boerwinkle E, Mosley TH Jr., Hobbs HH. Sequence variations in PCSK9, low LDL, and protection against coronary heart disease. N Engl J Med. 2006;354:1264-72.

  10. Kathiresan S, Myocardial Infarction Genetics Consortium. A PCSK9 missense variant associated with a reduced risk of earlyonset myocardial infarction. N Engl J Med. 2008;358:2299-300.

  11. Zhang DW, Lagace TA, Garuti R, et al. Binding of proprotein convertase subtilisin/kexin type 9 to epidermal growth factorlike repeat A of low density lipoprotein receptor decreases receptor recycling and increases degradation. J Biol Chem. 2007; 282:18602-12.

  12. Mattijssen F, Kersten S. Regulation of triglyceride metabolism by angiopoietin-like proteins. Biochim Biophys Acta. 2012;1821: 782-9.

  13. Koishi R, Ando Y, Ono M, et al. Angptl3 regulates lipid metabolism in mice. Nat Genet. 2002;30:151-7.

  14. Haller JF, Mintah IJ, Shihanian LM, et al. ANGPTL8 requires ANGPTL3 to inhibit lipoprotein lipase and plasma triglyceride clearance. J Lipid Res. 2017;58:1166-73.

  15. Stitziel NO, Khera AV, Wang X, et al. ANGPTL3 deficiency and protection against coronary artery disease. J Am Coll Cardiol. 2017;69:2054-63.

  16. Dewey FE, Gusarova V, Dunbar RL, et al. Genetic and pharmacologic inactivation of ANGPTL3 and cardiovascular disease. N Engl J Med. 2017;377:211-21.

  17. Robciuc MR, Maranghi M, Lahikainen A, et al. Angptl3 deficiency is associated with increased insulin sensitivity, lipoprotein lipase activity, and decreased serum free fatty acids. Arterioscler Thromb Vasc Biol. 2013;33:1706-13.

  18. Mendivil CO, Zheng C, Furtado J, et al. Metabolism of very-lowdensity lipoprotein and low-density lipoprotein containing apolipoprotein C-III and not other small apolipoproteins. Arterioscler Thromb Vasc Biol. 2010;30:239-45.

  19. Mendivil CO, Rimm EB, Furtado J, Sacks FM. Apolipoprotein E in VLDL and LDL with apolipoprotein C-III is associated with a lower risk of coronary heart disease. J Am Heart Assoc. 2013; 2:e000130.

  20. Mendivil CO, Rimm EB, Furtado J, Chiuve SE, Sacks FM. Lowdensity lipoproteins containing apolipoprotein C-III and the risk of coronary heart disease. Circulation. 2011;124:2065-72.

  21. Jensen MK, Rimm EB, Furtado JD, Sacks FM. Apolipoprotein C-III as a potential modulator of the association between HDL-cholesterol and incident coronary heart disease. J Am Heart Assoc. 2012;1:jah3-e000232.

  22. Pollin TI, Damcott CM, Shen H, et al. A null mutation in human APOC3 confers a favorable plasma lipid profile and apparent cardioprotection. Science. 2008;322:1702-5.

  23. Maranhão RC, Carvalho PO, Strunz CC, Pileggi F. Lipoprotein (a): structure, pathophysiology and clinical implications. Arq Bras Cardiol. 2014;103:76-84.

  24. Emerging Risk Factors Collaboration, Erqou S, Kaptoge S, et al. Lipoprotein(a) concentration and the risk of coronary heart disease, stroke, and nonvascular mortality. JAMA. 2009;302:412-23.

  25. Clarke R, Peden JF, Hopewell JC, et al. Genetic variants associated with lp(a) lipoprotein level and coronary disease. N Engl J Med. 2009;361:2518-28.

  26. Köhler G, Milstein C. Continuous cultures of fused cells secreting antibody of predefined specificity. Nature. 1975;256:495-7.

  27. Stoekenbroek RM, Kastelein JJ, Huijgen R. PCSK9 inhibition: the way forward in the treatment of dyslipidemia. BMC Med. 2015; 13:258.

  28. Raal FJ, Stein EA, Dufour R, et al. PCSK9 inhibition with evolocumab (AMG 145) in heterozygous familial hypercholesterolaemia (RUTHERFORD-2): a randomised, double-blind, placebocontrolled trial. Lancet. 2015;385:331-40.

  29. Raal FJ, Honarpour N, Blom DJ, et al. Inhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolaemia (TESLA part B): A randomised, double-blind, placebo-controlled trial. Lancet. 2015;385:341-50.

  30. Raal FJ, Hovingh GK, Blom D, et al. Long-term treatment with evolocumab added to conventional drug therapy, with or without apheresis, in patients with homozygous familial hypercholesterolemia: an interim subset analysis of the open-label TAUSSIG study. Lancet Diabetes Endocrinol. 2017;5:280-90.

  31. Koren MJ, Lundqvist P, Bolognese M, et al. Anti-PCSK9 monotherapy for hypercholesterolemia: the MENDEL-2 randomized, controlled phase III clinical trial of evolocumab. J Am Coll Cardiol. 2014;63:2531-40.

  32. Robinson JG, Nedergaard BS, Rogers WJ, et al. Effect of evolocumab or ezetimibe added to moderate-or high-intensity statin therapy on LDL-C lowering in patients with hypercholesterolemia. JAMA. 2014;311:1870.

  33. Blom DJ, Hala T, Bolognese M, et al. A 52-week placebo-controlled trial of evolocumab in hyperlipidemia. N Engl J Med. 2014;370:1809-19.

  34. Nissen SE, Stroes E, Dent-Acosta RE, et al. Efficacy and tolerability of evolocumab vs ezetimibe in patients with muscle-related statin intolerance: the GAUSS-3 randomized clinical trial. JAMA. 2016;315:1580-90.

  35. Sabatine MS, Giugliano RP, Wiviott SD, et al. Efficacy and safety of evolocumab in reducing lipids and cardiovascular events. N Engl J Med. 2015;372:1500-9.

  36. Sabatine MS, Giugliano RP, Keech AC, et al. Evolocumab and clinical outcomes in patients with cardiovascular disease. N Engl J Med. 2017;376:1713-22.

  37. Giugliano RP, Mach F, Zavitz K, et al. Cognitive function in a randomized trial of evolocumab. N Engl J Med. 2017;377:633-43.

  38. Kastelein JJ, Ginsberg HN, Langslet G, et al. ODYSSEY FH I and FH II: 78 week results with alirocumab treatment in 735 patients with heterozygous familial hypercholesterolaemia. Eur Heart J. 2015;36:2996-3003.

  39. Moriarty PM, Parhofer KG, Babirak SP, et al. Alirocumab in patients with heterozygous familial hypercholesterolaemia undergoing lipoprotein apheresis: the ODYSSEY ESCAPE trial. Eur Heart J. 2016;37:3588-95.

  40. Kereiakes DJ, Robinson JG, Cannon CP, et al. Efficacy and safety of the proprotein convertase subtilisin/kexin type 9 inhibitor alirocumab among high cardiovascular risk patients on maximally tolerated statin therapy: the ODYSSEY COMBO I study. Am Heart J. 2015;169:906-15E+15.

  41. Cannon CP, Cariou B, Blom D, et al. Efficacy and safety of alirocumab in high cardiovascular risk patients with inadequately controlled hypercholesterolaemia on maximally tolerated doses of statins: The ODYSSEY COMBO II randomized controlled trial. Eur Heart J. 2015;36:1186-94.

  42. Bays H, Gaudet D, Weiss R, et al. Alirocumab as add-on to atorvastatin versus other lipid treatment strategies: ODYSSEY OPTIONS I randomized trial. J Clin Endocrinol Metab. 2015;100:3140-8.

  43. Moriarty PM, Thompson PD, Cannon CP, et al. Efficacy and safety of alirocumab vs ezetimibe in statin-intolerant patients, with a statin rechallenge arm: the ODYSSEY ALTERNATIVE randomized trial. J Clin Lipidol. 2015;9:758-69.

  44. Robinson JG, Farnier M, Krempf M, et al. Efficacy and safety of alirocumab in reducing lipids and cardiovascular events. N Engl J Med. 2015;372:1489-99.

  45. Steg P, The ODYSSEY OUTCOMES investigators. Presented at the American College of Cardiology Annual Scientific Session (ACC 2018), Orlando, FL; 2018. Available from: (accessed August 23, 2018)

  46. Ridker PM, Revkin J, Amarenco P, et al. Cardiovascular efficacy and safety of bococizumab in high-risk patients. N Engl J Med. 2017;376:1527-39.

  47. Beyer TP, Eacho PI, Schroeder KM, et al. A PCSK9 antibody that blocks binding to LDLR while allowing normal PCSK9 inactivation by furin is afforded a reduced clearance rate and a longer duration of effect in mice. Atheroscler Thromb Vasc Biol. 2015; 35:A538.

  48. Kastelein JJ, Nissen SE, Rader DJ, et al. Safety and efficacy of LY3015014, a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 (PCSK9): a randomized, placebo-controlled phase 2 study. Eur Heart J. 2016;37:1360-9.

  49. Gusarova V, Alexa CA, Wang Y, et al. ANGPTL3 blockade with a human monoclonal antibody reduces plasma lipids in dyslipidemic mice and monkeys. J Lipid Res. 2015;56:1308-17.

  50. Gaudet D, Gipe DA, Pordy R, et al. ANGPTL3 inhibition in homozygous familial hypercholesterolemia. N Engl J Med. 2017; 377:296-7.

  51. Miller CM, Harris EN. Antisense oligonucleotides: treatment strategies and cellular internalization. RNA Dis. 2016;3:e1393.

  52. Toth PP. Antisense therapy and emerging applications for the management of dyslipidemia. J Clin Lipidol. 2011;5:441-9.

  53. Crooke ST. Molecular mechanisms of antisense oligonucleotides. Nucleic Acid Ther. 2017;27:70-7.

  54. Besseling J, Hovingh GK, Stroes ES. Antisense oligonucleotides in the treatment of lipid disorders: pitfalls and promises. Neth J Med. 2013;71:118-22.

  55. Kurreck J. Antisense technologies. Improvement through novel chemical modifications. Eur J Biochem. 2003;270:1628-44.

  56. Agrawal S, Jiang Z, Zhao Q, et al. Mixed-backbone oligonucleotides as second generation antisense oligonucleotides: in vitro and in vivo studies. Proc Natl Acad Sci U S A. 1997;94:2620-5.

  57. Raal FJ, Santos RD, Blom DJ, et al. 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. 2010;375:998-1006.

  58. Stein EA, Dufour R, Gagne C, et al. Apolipoprotein B synthesis inhibition with mipomersen in heterozygous familial hypercholesterolemia: results of a randomized, double-blind, placebocontrolled trial to assess efficacy and safety as add-on therapy in patients with coronary artery disease. Circulation. 2012; 126:2283-92.

  59. Santos RD, Duell PB, East C, et al. Long-term efficacy and safety of mipomersen in patients with familial hypercholesterolaemia: 2-year interim results of an open-label extension. Eur Heart J. 2015;36:566-75.

  60. Duell PB, Santos RD, Kirwan BA, et al. Long-term mipomersen treatment is associated with a reduction in cardiovascular events in patients with familial hypercholesterolemia. J Clin Lipidol. 2016;10:1011-21.

  61. Graham MJ, Lee RG, Brandt TA, et al. Cardiovascular and metabolic effects of ANGPTL3 antisense oligonucleotides. N Engl J Med. 2017;377:222-32.

  62. Gaudet D, Alexander VJ, Baker BF, et al. Antisense inhibition of apolipoprotein C-III in patients with hypertriglyceridemia. N Engl J Med. 2015;373:438-47.

  63. Digenio A, Dunbar RL, Alexander VJ, et al. Antisense-mediated lowering of plasma apolipoprotein C-III by volanesorsen improves dyslipidemia and insulin sensitivity in type 2 diabetes. Diabetes Care. 2016;39:1408-15.

  64. Gaudet D, Brisson D, Tremblay K, et al. Targeting APOC3 in the familial chylomicronemia syndrome. N Engl J Med. 2014; 371:2200-6.

  65. Graham MJ, Viney N, Crooke RM, Tsimikas S. Antisense inhibition of apolipoprotein (a) to lower plasma lipoprotein (a) levels in humans. J Lipid Res. 2016;57:340-51.

  66. Tsimikas S, Viney NJ, Hughes SG, et al. Antisense therapy targeting apolipoprotein(a): a randomised, double-blind, placebocontrolled phase 1 study. Lancet. 2015;386:1472-83.

  67. Viney NJ, van Capelleveen JC, Geary RS, et al. Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomised, double-blind, placebo-controlled, dose-ranging trials. Lancet. 2016;388:2239-53.

  68. Ozcan G, Ozpolat B, Coleman RL, Sood AK, Lopez-Berestein G. Preclinical and clinical development of siRNA-based therapeutics. Adv Drug Deliv Rev. 2015;87:108-19.

  69. Carthew RW, Sontheimer EJ. Origins and mechanisms of miRNAs and siRNAs. Cell. 2009;136:642-55.

  70. Kim DH, Rossi JJ. Strategies for silencing human disease using RNA interference. Nat Rev Genet. 2007;8:173-84.

  71. Khvorova A. Oligonucleotide therapeutics - A new class of cholesterol- lowering drugs. N Engl J Med. 2017;376:4-7.

  72. Juliano R, Alam MR, Dixit V, Kang H. Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides. Nucleic Acids Res. 2008;36:4158-71.

  73. Fitzgerald K, White S, Borodovsky A, et al. A highly durable RNAi therapeutic inhibitor of PCSK9. N Engl J Med. 2017;376:41-51.

  74. Mali P, Yang L, Esvelt KM, et al. RNA-guided human genome engineering via cas9. Science. 2013;339:823-6.

  75. Musunuru K. Genome editing: the recent history and perspective in cardiovascular diseases. J Am Coll Cardiol. 2017;70:2808-21.

  76. Cong L, Ran FA, Cox D, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339:819-23.

  77. Gilbert LA, Larson MH, Morsut L, et al. CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes. Cell. 2013;154:442-51.

  78. Ran FA, Cong L, Yan WX, et al. In vivo genome editing using Staphylococcus aureus cas9. Nature. 2015;520:186-91.

  79. Chadwick AC, Wang X, Musunuru K. In vivo base editing of PCSK9 (Proprotein convertase subtilisin/Kexin Type 9) as a therapeutic alternative to genome editing. Arterioscler Thromb Vasc Biol. 2017;37:1741-7.

  80. Chadwick AC, Evitt NH, Lv W, Musunuru K. Reduced blood lipid levels with in vivo CRISPR-cas9 base editing of ANGPTL3. Circulation. 2018;137:975-7.




2020     |     www.medigraphic.com