Inhibition of the alternative complement pathway by antisense oligonucleotides targeting complement factor B improves lupus nephritis in mice

Immunobiology

Volumn 221, Issue 6, 2016, Pages 701-708

  Grossman, Tamar R ^a   Hettrick, Lisa A ^a   Johnson, Robert B ^a   Hung, Gene ^a   Peralta, Raechel ^a   Watt, Andrew ^a   Henry, Scott P ^a   Adamson, Peter ^b   Monia, Brett P ^a   McCaleb, Michael L ^a  

^a ISIS PHARMACEUTICALS   (United States)

^b GLAXOSMITHKLINE   (United Kingdom)

Author keywords

Antisense oligonucleotides; Complement; Complement factor B; Lupus nephritis; Therapeutics

Indexed keywords

ALTERNATIVE COMPLEMENT PATHWAY C3 C5 CONVERTASE; ANTISENSE OLIGONUCLEOTIDE; COMPLEMENT COMPONENT C3; COMPLEMENT MEMBRANE ATTACK COMPLEX; IMMUNOGLOBULIN A; IMMUNOGLOBULIN G; IMMUNOGLOBULIN M; SODIUM CHLORIDE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; COMPLEMENT ACTIVATION; COMPLEMENT DEPOSITION; CONTROLLED STUDY; DISEASE MODEL; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG POTENCY; DRUG TARGETING; DRUG TOLERABILITY; ENZYME INHIBITION; FEMALE; LUPUS ERYTHEMATOSUS NEPHRITIS; MOUSE; NONHUMAN; PATHOGENESIS; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEINURIA; SURVIVAL RATE; TREATMENT OUTCOME; ANIMAL; ANTIGEN ANTIBODY COMPLEX; CELL CULTURE; COMPLEMENT ALTERNATIVE PATHWAY; GENETICS; HUMAN; IMMUNOLOGY; KIDNEY; LIVER CELL; METABOLISM; MURPHY ROTHS LARGE LYMPHOPROLIFERATIVE MOUSE; NEW ZEALAND BLACK MOUSE; PATHOLOGY; PHYSIOLOGY; SYSTEMIC LUPUS ERYTHEMATOSUS;

ANIMALS; ANTIGEN-ANTIBODY COMPLEX; CELLS, CULTURED; COMPLEMENT C3; COMPLEMENT FACTOR B; COMPLEMENT PATHWAY, ALTERNATIVE; DISEASE MODELS, ANIMAL; HEPATOCYTES; HUMANS; KIDNEY; LUPUS ERYTHEMATOSUS, SYSTEMIC; LUPUS NEPHRITIS; MICE; MICE, INBRED MRL LPR; MICE, INBRED NZB; OLIGONUCLEOTIDES, ANTISENSE; PROTEINURIA;

EID: 84939625000     PISSN: 01712985     EISSN: 18783279     Source Type: Journal    
DOI: 10.1016/j.imbio.2015.08.001     Document Type: Article

References (37)

1. Baker B.F., Lot S.S., Condon T.P., Cheng-Flournoy S., Lesnik E.A., Sasmor H.M., Bennett C.F. 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.
2. Bao L., Quigg R.J. Complement in lupus nephritis: the good, the bad, and the unknown. Semin Nephrol. 2007, 27:69.
3. Bennett C.F., Swayze E.E. RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu. Rev. Pharmacol. Toxicol. 2010, 50:259.
4. Biesecker G., Katz S., Koffler D. Renal localization of the membrane attack complex in systemic lupus erythematosus nephritis. J. Exp. Med. 1981, 154:1779.
5. Botto M., Kirschfink M., Macor P., Pickering M.C., Wurzner R., Tedesco F. Complement in human diseases: lessons from complement deficiencies. Mol. Immunol. 2009, 46:2774.
6. Cameron J.S., Vick R.M., Ogg C.S., Seymour W.M., Chantler C., Turner D.R. Plasma C and C4 concentrations in management of glomerulonephritis. Br. Med. J. 1973, 3:668.
7. Carroll M.C. A protective role for innate immunity in systemic lupus erythematosus. Nat. Rev. Immunol. 2004, 4:825.
8. Clynes R., Dumitru C., Ravetch J.V. Uncoupling of immune complex formation and kidney damage in autoimmune glomerulonephritis. Science 1998, 279:1052.
9. Crooke S.T. Antisense Drug Technology: Principles, Strategies, and Applications 2008, 273. CRC Press, Boca Raton. 2nd ed.
10. Drake C.G., Rozzo S.J., Vyse T.J., Palmer E., Kotzin B.L. Genetic contributions to lupus-like disease in (NZB × NZW)F1 mice. Immunol. Rev. 1995, 144:51.
11. Keating G.M., Lyseng-Williamson K.A., McKeage K. Eculizumab: a guide to its use in paroxysmal nocturnal hemoglobinuria. BioDrugs 2012, 26:125.
12. Koskimies S., Lokki M.L., Höckerstedt K. Changes in plasma complement C4 and factor B allotypes after liver transplantation. Complement Inflamm. 1991, 8(5-6):257-260.
13. Lachmann P.J. An attempt to characterize the lupus erythematosus cell antigen. Immunology 1961, 4:153.
14. Lachmann P.J. The amplification loop of the complement pathways. Adv. Immunol. 2009, 104:115.
15. Li N., Li Q., Tian X.Q., Qian H.Y., Yang Y.J. Mipomersen is a promising therapy in the management of hypercholesterolemia: a meta-analysis of randomized controlled trials. Am. J. Cardiovasc. Drugs 2014, 14:367.
16. Madaio M.P., Harrington J.T. The diagnosis of glomerular diseases acute glomerulonephritis and the nephrotic syndrome. Arch. Intern. Med. 2001, 161:25.
17. Marsh J.E., Zhou W., Sacks S.H. Local tissue complement synthesis-fine tuning a blunt instrument. Arch. Immol. Ther. Exp. (Warsz) 2001, 49(Suppl. 1):S41-S46.
18. Morgan B.P., Gasque P. Extrahepatic complement biosynthesis: where, when and why?. Clin. Exp. Immunol. 1997, 107:1-7.
19. Muller-Eberhard H.J., Schreiber R.D. Molecular biology and chemistry of the alternative pathway of complement. Adv. Immunol. 1980, 29:1.
20. Perrin L.H., Lambert P.H., Miescher P.A. Complement breakdown products in plasma from patients with systemic lupus erythematosus and patients with membranoproliferative or other glomerulonephritis. J. Clin. Invest. 1975, 56:165.
21. Quigg R.J., Lim A., Haas M., Alexander J.J., He C., Carroll M.C. Immune complex glomerulonephritis in C4- and C3-deficient mice. Kidney Int. 1998, 53:320.
22. Quistorff B., Dich J., Grunnet N. Preparation of isolated rat liver hepatocytes. Methods Mol. Biol. 1990, 5:151.
23. Ricklin D., Lambris J.D. Complement-targeted therapeutics. Nat. Biotechnol. 2007, 25:1265.
24. Ripoche J., Mitchell J.A., Erdei A., Madin C., Moffatt B., Mokoena T., Gordon S., Sim R.B. Interferon gamma induces synthesis of complement alternative pathway proteins by human endothelial cells in culture. J. Exp. Med. 1988, 168:1917.
25. Rovin B.H., Parikh S.V. Lupus nephritis: the evolving role of novel therapeutics. Am. J. Kidney Dis. 2014, 63:677.
26. Sahu A., Lambris J.D. Complement inhibitors: a resurgent concept in anti-inflammatory therapeutics. Immunopharmacology 2000, 49:133.
27. Schreiber R.D., Pangburn M.K., Lesavre P.H., Muller-Eberhard H.J. Initiation of the alternative pathway of complement: recognition of activators by bound C3b and assembly of the entire pathway from six isolated proteins. Proc. Natl. Acad. Sci. U. S. A. 1978, 75:3948.
28. Sekine H., Reilly C.M., Molano I.D., Garnier G., Circolo A., Ruiz P., Holers V.M., Boackle S.A., Gilkeson G.S. Complement component C3 is not required for full expression of immune complex glomerulonephritis in MRL/lpr mice. J. Immunol. 2001, 166:6444.
29. Slade C., Bosco J., Unglik G., Bleasel K., Nagel M., Winship I. Deficiency in complement factor B. N. Engl. J. Med. 2013, 369:1667.
30. Sterner R.M., Hartono S.P., Grande J.P. The Pathogenesis of lupus nephritis. J. Clin. Cell Immunol. 2014, 5.
31. Strunk R.C., Eidlen D.M., Mason R.J. Pulmonary alveolar type II epithelial cells synthesize and secrete proteins of the classical and alternative complement pathways. J. Clin. Invest. 1988, 81:1419.
32. Theofilopoulos A.N., Dixon F.J. Murine models of systemic lupus erythematosus. Adv. Immunol. 1985, 37:269.
33. Walport M.J. Complement and systemic lupus erythematosus. Arthritis Res. 2002, 4(Suppl 3):S279.
34. Wang Y., Hu Q., Madri J.A., Rollins S.A., Chodera A., Matis L.A. Amelioration of lupus-like autoimmune disease in NZB/WF1 mice after treatment with a blocking monoclonal antibody specific for complement component C5. Proc. Natl. Acad. Sci. U. S. A. 1996, 93:8563.
35. Watanabe H., Garnier G., Circolo A., Wetsel R.A., Ruiz P., Holers V.M., Boackle S.A., Colten H.R., Gilkeson G.S. Modulation of renal disease in MRL/lpr mice genetically deficient in the alternative complement pathway factor B. J. Immunol. 2000, 164:786.
36. Watson M.L., Rao J.K., Gilkeson G.S., Ruiz P., Eicher E.M., Pisetsky D.S., Matsuzawa A., Rochelle J.M., Seldin M.F. Genetic analysis of MRL-lpr mice: relationship of the Fas apoptosis gene to disease manifestations and renal disease-modifying loci. J. Exp. Med. 1992, 176:1645.
37. Whaley K. Biosynthesis of the complement components and the regulatory proteins of the alternative complement pathway by human peripheral blood monocytes. J. Exp. Med. 1980, 151:501.