A selective inhibitor of human C-reactive protein translation is efficacious in vitro and in C-reactive protein transgenic mice and humans

Molecular Therapy Nucleic Acids

Volumn 1, Issue 11, 2012, Pages

  Jones, Nicholas R ^a   Pegues, Melissa A ^a   McCrory, Mark A ^a   Singleton, Walter ^a   Bethune, Claudette ^b   Baker, Brenda F ^b   Norris, Daniel A ^b   Crooke, Rosanne M ^b   Graham, Mark J ^b   Szalai, Alexander J ^a  

^a The University of Alabama at Birmingham   (United States)

^b ISIS PHARMACEUTICALS   (United States)

Author keywords

Antisense therapy; CRP; rheumatoid arthritis

Indexed keywords

ALANINE AMINOTRANSFERASE; ANTISENSE OLIGONUCLEOTIDE; C REACTIVE PROTEIN; COLLAGEN; ISIS 329993; MESSENGER RNA; PLACEBO; UNCLASSIFIED DRUG;

ADULT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; AREA UNDER THE CURVE; ARTHRITIS; ARTICLE; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; DRUG ACTIVITY; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; FEMALE; HUMAN; HUMAN EXPERIMENT; IN VITRO STUDY; LOADING DRUG DOSE; MALE; MOUSE; NONHUMAN; NORMAL HUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTEIN BLOOD LEVEL; SCREENING TEST; TRANSGENIC MOUSE;

EID: 84869476334     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2012.44     Document Type: Article

References (44)

1. Firestein, GS (2003). Evolving concepts of rheumatoid arthritis. Nature 423: 356-361 (Pubitemid 40852711)
2. Goronzy, JJ and Weyand, CM (2009). Developments in the scientifc understanding of rheumatoid arthritis. Arthritis Res Ther 11: 249.
3. Kapetanovic, MC, Lindqvist, E, Saxne, T and Eberhardt, K (2008). Orthopaedic surgery in patients with rheumatoid arthritis over 20 years: prevalence and predictive factors of large joint replacement. Ann Rheum Dis 67: 1412-1416.
4. Kremers, HM, Crowson, CS, Therneau, TM, Roger, VL and Gabriel, SE (2008). High ten-year risk of cardiovascular disease in newly diagnosed rheumatoid arthritis patients: a population-based cohort study. Arthritis Rheum 58: 2268-2274.
5. del Rincón, ID, Williams, K, Stern, MP, Freeman, GL and Escalante, A (2001). High inci-dence of cardiovascular events in a rheumatoid arthritis cohort not explained by traditional cardiac risk factors. Arthritis Rheum 44: 2737-2745. (Pubitemid 34016192)
6. Newkirk, MM, Fournier, MJ and Shiroky, J (1995). Rheumatoid factor avidity in patients with rheumatoid arthritis: identifcation of pathogenic RFs which correlate with disease pa-rameters and with the gal(0) glycoform of IgG. J Clin Immunol 15: 250-257.
7. Mallya, RK, de Beer, FC, Berry, H, Hamilton, ED, Mace, BE and Pepys, MB (1982). Correlation of clinical parameters of disease activity in rheumatoid arthritis with serum concentration of C-reactive protein and erythrocyte sedimentation rate. J Rheumatol 9: 224-228. (Pubitemid 12014862)
8. Volanakis, JE (1982). Complement activation by C-reactive protein complexes. Ann N Y Acad Sci 389: 235-250. (Pubitemid 12013192)
9. Molenaar, ET, Voskuyl, AE, Familian, A, van Mierlo, GJ, Dijkmans, BA and Hack, CE (2001). Complement activation in patients with rheumatoid arthritis mediated in part by C-reactive protein. Arthritis Rheum 44: 997-1002. (Pubitemid 32433603)
10. Mold, C, Gresham, HD and Du Clos, TW (2001). Serum amyloid P component and C-reactive protein mediate phagocytosis through murine FcgammaRs. J Immunol 166: 1200-1205 (Pubitemid 32060280)
11. Lu, J, Marnell, LL, Marjon, KD, Mold, C, Du Clos, TW and Sun, PD (2008). Structural recog-nition and functional activation of FcgammaR by innate pentraxins. Nature 456: 989-992.
12. Vigushin, DM, Pepys, MB and Hawkins, PN (1993). Metabolic and scintigraphic studies of ra-dioiodinated human C-reactive protein in health and disease. J Clin Invest 91: 1351-1357. (Pubitemid 23127332)
13. Walters, MT, Stevenson, FK, Goswami, R, Smith, JL and Cawley, MI (1989). Comparison of serum and synovial fuid concentrations of beta 2-microglobulin and C reactive protein in relation to clinical disease activity and synovial infammation in rheumatoid arthritis. Ann Rheum Dis 48: 905-911 (Pubitemid 19280912)
14. Zamani, B, Jamali, R and Ehteram, H (2012). Synovial fuid adenosine deaminase and high-sensitivity C-reactive protein activity in differentiating monoarthritis. Rheumatol Int 32: 183-188.
15. Wells, G, Becker, JC, Teng, J, Dougados, M, Schiff, M, Smolen, J et al. (2009). Validation of the 28-joint Disease Activity Score (DAS28) and European League Against Rheumatism response criteria based on C-reactive protein against disease progression in patients with rheumatoid arthritis, and comparison with the DAS28 based on erythrocyte sedimentation rate. Ann Rheum Dis 68: 954-960.
16. Kushner, I and Somerville-Volanakis, J (1973). Studies of synovial and serum C-reactive protein in experimental arthritis in rabbits. Proc Soc Exp Biol Med 142: 112-114.
17. Phillips, NC (1982). Exacerbation of experimental poly-D-lysine arthritis by C-reactive pro-tein. Agents Actions 12: 344-347. (Pubitemid 12026818)
18. Jiang, S, Xia, D and Samols, D (2006). Expression of rabbit C-reactive protein in transgenic mice inhibits development of antigen-induced arthritis. Scand J Rheumatol 35: 351-355. (Pubitemid 44601067)
19. Ciliberto, G, Arcone, R, Wagner, EF and Rüther, U (1987). Inducible and tissue-specifc expression of human C-reactive protein in transgenic mice. EMBO J 6: 4017-4022.
20. Szalai, AJ and McCrory, MA (2002). Varied Biologic Functions of C-reactive Protein. Im-munol Res 26: 279-287.
21. Jones, NR, Pegues, MA, McCrory, MA, Kerr, SW, Jiang, H, Sellati, R et al. (2011). Colla-gen-induced arthritis is exacerbated in C-reactive protein-defcient mice. Arthritis Rheum 63: 2641-2650.
22. Pepys, MB, Hirschfeld, GM, Tennent, GA, Gallimore, JR, Kahan, MC, Bellotti, V et al. (2006). Targeting C-reactive protein for the treatment of cardiovascular disease. Nature 440: 1217-1221
23. Okroj, M, Heinegård, D, Holmdahl, R and Blom, AM (2007). Rheumatoid arthritis and the complement system. Ann Med 39: 517-530. (Pubitemid 350035568)
24. Monach, PA, Hueber, W, Kessler, B, Tomooka, BH, BenBarak, M, Simmons, BP et al. (2009). A broad screen for targets of immune complexes decorating arthritic joints high-lights deposition of nucleosomes in rheumatoid arthritis. Proc Natl Acad Sci USA 106: 15867-15872.
25. Wenink, MH, Santegoets, KC, Roelofs, MF, Huijbens, R, Koenen, HJ, van Beek, R et al. (2009). The inhibitory Fc gamma IIb receptor dampens TLR4-mediated immune responses and is selectively up-regulated on dendritic cells from rheumatoid arthritis patients with quiescent disease. J Immunol 183: 4509-4520.
26. Yu, RZ, Kim, TW, Hong, A, Watanabe, TA, Gaus, HJ and Geary, RS (2007). Cross-species pharmacokinetic comparison from mouse to man of a second-generation antisense oligo-nucleotide, ISIS 301012, targeting human apolipoprotein B-100. Drug Metab Dispos 35: 460-468. (Pubitemid 46333910)
27. Yu, RZ, Geary RS, Siwkowski, A, and Levin, AA (2008). Pharmacokinetic/pharmaco-dynamic properties of phosphorothioate 2'-O-(2-methoxyethyl)-modifed an-tisense oligonucleotides in animals and man. In: Crooke SA (ed.). Antisense Drug Technology: Principles, Strategies, and Applications, 2nd edn. CRC Press: Boca Raton, FL. pp. 305-326.
28. Graham, MJ, Crooke, ST, Lemonidis, KM, Gaus, HJ, Templin, MV and Crooke, RM (2001). Hepatic distribution of a phosphorothioate oligodeoxynucleotide within rodents following intravenous administration. Biochem Pharmacol 62: 297-306. (Pubitemid 32575330)
29. Jialal, I, Devaraj, S and Venugopal, SK (2004). C-reactive protein: risk marker or mediator in atherothrombosis? Hypertension 44: 6-11. (Pubitemid 38823477)
30. Kastelein, JJ, Wedel, MK, Baker, BF, Su, J, Bradley, JD, Yu, RZ et al. (2006). Potent reduc-tion of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation 114: 1729-1735 (Pubitemid 44607121)
31. Kwoh TJ. (2008). An overview of clinical safety experience of frst-and second-generation antisense oligodeoxynucleotides. In: Crooke SA (ed.). Antisense Drug Technology: Principles, Strategies, and Applications, 2nd edn. CRC Press. pp. 365-399.
32. Crooke, RM, Graham, MJ, Lemonidis, KM, Whipple, CP, Koo, S and Perera, RJ (2005). An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis. J Lipid Res 46: 872-884.
33. Olofsson, SO and Borèn, J (2005). Apolipoprotein B: a clinically important apolipoprotein which assembles atherogenic lipoproteins and promotes the development of atherosclero-sis. J Intern Med 258: 395-410. (Pubitemid 41546555)
34. Akdim, F, Stroes, ES and Kastelein, JJ (2007). Antisense apolipoprotein B therapy: where do we stand? Curr Opin Lipidol 18: 397-400. (Pubitemid 47051917)
35. Henry SO, Kim T-W, Kramer-Srickland K, Zanardi TA, Fey RA and Levin AA (2008). Toxi-cologic properties of 2'O-methyl chimeric antisense inhibitors in animals and man In: Crooke SA (ed.). Antisense Drug Technology: Principles, Strategies, and Applications, 2nd edn. CRC Press. pp. 327-363.
36. Henry, SP, Geary, RS, Yu, R and Levin, AA (2001). Drug properties of second-generation antisense oligonucleotides: how do they measure up to their predecessors? Curr Opin Investig Drugs 2: 1444-1449. (Pubitemid 32994399)
37. Schurgers, E, Billiau, A and Matthys, P (2011). Collagen-induced arthritis as an animal model for rheumatoid arthritis: focus on interferon-γ J Interferon Cytokine Res 31: 917-926.
38. Crooke, ST (2004). Progress in antisense technology. Annu Rev Med 55: 61-95. (Pubitemid 38316716)
39. Gabay, C and Kushner, I (1999). Acute-phase proteins and other systemic responses to infammation. N Engl J Med 340: 448-454. (Pubitemid 29084777)
40. Whitehead, AS, Zahedi, K, Rits, M, Mortensen, RF and Lelias, JM (1990). Mouse C-reac-tive protein. Generation of cDNA clones, structural analysis, and induction of mRNA during infammation. Biochem J 266: 283-290. (Pubitemid 20069050)
41. Inglis, JJ, Simelyte, E, McCann, FE, Criado, G and Williams, RO (2008). Protocol for the induction of arthritis in C57BL/6 mice. Nat Protoc 3: 612-618. (Pubitemid 351516757)
42. Brand, DD, Latham, KA and Rosloniec, EF (2007). Collagen-induced arthritis. Nat Protoc 2: 1269-1275 (Pubitemid 46901304)
43. Barck, KH, Lee, WP, Diehl, LJ, Ross, J, Gribling, P, Zhang, Y et al. (2004). Quantifcation of cortical bone loss and repair for therapeutic evaluation in collagen-induced arthritis, by micro-computed tomography and automated image analysis. Arthritis Rheum 50: 3377-3386. (Pubitemid 39372016)
44. Felson, DT, Anderson, JJ, Boers, M, Bombardier, C, Furst, D, Goldsmith, C et al. (1995). American College of Rheumatology. Preliminary defnition of improvement in rheumatoid arthritis. Arthritis Rheum 38: 727-735.