Proof-of-concept Studies for siRNA-mediated Gene Silencing for Coagulation Factors in Rat and Rabbit

Molecular Therapy Nucleic Acids

Volumn 4, Issue 1, 2015, Pages e224-

  Chen, Zhu ^a   Luo, Bin ^a   Cai, Tian Quan ^a   Thankappan, Anil ^a   Xu, Yiming ^a   Wu, Weizhen ^a   DiMuzio, Jillian ^a   Lifsted, Traci ^a   DiPietro, Marty ^a   Disa, Jyoti ^a   Ng, Bruce ^a   Leander, Karen ^a   Clark, Seth ^a   Hoos, Lizbeth ^a   Zhou, Yuchen ^a   Jochnowitz, Nina ^a   Jachec, Christine ^a   Szczerba, Peter ^a   Gindy, Marian E ^a   Strapps, Walter ^a   Sepp Lorenzino, Laura ^a   Seiffert, Dietmar A ^a   Lubbers, Laura ^a   Tadin Strapps, Marija ^a   more..

^a MERCK RESEARCH LABORATORIES   (United States)

Author keywords

coagulation; Factor X; lipid nanoparticle; plasma kallikrein; siRNA

Indexed keywords

BLOOD CLOTTING FACTOR; BLOOD CLOTTING FACTOR 10; NANOPARTICLE; PREKALLIKREIN; SMALL INTERFERING RNA;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BLEEDING TIME; BLOOD CLOTTING TIME; CONTROLLED STUDY; DRUG DELIVERY SYSTEM; GENE SILENCING; MALE; NONHUMAN; PRIORITY JOURNAL; RABBIT; RAT; THROMBOSIS;

EID: 84927734274     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.75     Document Type: Article

References (38)

1. Bakhtiyari, S, Haghani, K, Basati, G and Karimfar, MH (2013). siRNA therapeutics in the treatment of diseases. Ther Deliv 4: 45-57.
2. Tadin-Strapps, M, Peterson, LB, Cumiskey, AM, Rosa, RL, Mendoza, VH, Castro-Perez, J et al. (2011). siRNA-induced liver ApoB knockdown lowers serum LDL-cholesterol in a mouse model with human-like serum lipids. J Lipid Res 52: 1084-1097.
3. Olearczyk, J, Gao, S, Eybye, M, Yendluri, S, Andrews, L, Bartz, S et al. (2014). Targeting of hepatic angiotensinogen using chemically modified siRNAs results in significant and sustained blood pressure lowering in a rat model of hypertension. Hypertens Res 37: 405-412.
4. Safdar, H, Cheung, KL, Salvatori, D, Versteeg, HH, Laghmani, el H, Wagenaar, GT et al. (2013). Acute and severe coagulopathy in adult mice following silencing of hepatic antithrombin and protein C production. Blood 121: 4413-4416.
5. Tep, S, Mihaila, R, Freeman, A, Pickering, V, Huynh, F, Huyhn, F et al. (2012). Rescue of Mtp siRNA-induced hepatic steatosis by DGAT2 siRNA silencing. J Lipid Res 53: 859-867.
6. Tomari, Y and Zamore, PD (2005). Perspective: machines for RNAi. Genes Dev 19: 517-529.
7. Sepp-Lorenzino, L and Ruddy, M (2008). Challenges and opportunities for local and systemic delivery of siRNA and antisense oligonucleotides. Clin Pharmacol Ther 84: 628-632.
8. Wooddell, CI, Rozema, DB, Hossbach, M, John, M, Hamilton, HL, Chu, Q et al. (2013). Hepatocyte-targeted RNAi therapeutics for the treatment of chronic hepatitis B virus infection. Mol Ther 21: 973-985.
9. Furie, B and Furie, BC (1988). The molecular basis of blood coagulation. Cell 53: 505-518.
10. Chen, Z, Seiffert, D and Hawes, B (2014). Inhibition of Factor XI activity as a promising antithrombotic strategy. Drug Discov Today 19: 1435-1439.
11. Wong, PC, Crain, EJ, Watson, CA and Xin, B (2009). Favorable therapeutic index of the direct factor Xa inhibitors, apixaban and rivaroxaban, compared with the thrombin inhibitor dabigatran in rabbits. J Thromb Haemost 7: 1313-1320.
12. Vilahur, G, Padro, T and Badimon, L (2011). Atherosclerosis and thrombosis: insights from large animal models. J Biomed Biotechnol 2011: 907575.
13. Neth, P, Arnhold, M, Nitschko, H and Fink, E (2001). The mRNAs of prekallikrein, factors XI and XII, and kininogen, components of the contact phase cascade are differentially expressed in multiple non-hepatic human tissues. Thromb Haemost 85: 1043-1047.
14. Renné, T and Gruber, A (2012). Plasma kallikrein: novel functions for an old protease. Thromb Haemost 107: 1012-1013.
15. Gruber, A (2014). The role of the contact pathway in thrombus propagation. Thromb Res 133(suppl 1): S45-S47.
16. van Montfoort, ML and Meijers, JC (2013). Anticoagulation beyond direct thrombin and factor Xa inhibitors: indications for targeting the intrinsic pathway? Thromb Haemost 110: 223-232.
17. Bird, JE, Smith, PL, Wang, X, Schumacher, WA, Barbera, F, Revelli, JP et al. (2012). Effects of plasma kallikrein deficiency on haemostasis and thrombosis in mice: murine ortholog of the Fletcher trait. Thromb Haemost 107: 1141-1150.
18. Revenko, AS, Gao, D, Crosby, JR, Bhattacharjee, G, Zhao, C, May, C et al. (2011). Selective depletion of plasma prekallikrein or coagulation factor XII inhibits thrombosis in mice without increased risk of bleeding. Blood 118: 5302-5311.
19. Girolami, A, Scarparo, P, Candeo, N and Lombardi, AM (2010). Congenital prekallikrein deficiency. Expert Rev Hematol 3: 685-695.
20. Wong, PC, Crain, EJ, Xin, B, Wexler, RR, Lam, PY, Pinto, DJ et al. (2008). Apixaban, an oral, direct and highly selective factor Xa inhibitor: in vitro, antithrombotic and antihemostatic studies. J Thromb Haemost 6: 820-829.
21. Lu, G, DeGuzman, FR, Hollenbach, SJ, Karbarz, MJ, Abe, K, Lee, G et al. (2013). A specific antidote for reversal of anticoagulation by direct and indirect inhibitors of coagulation factor Xa. Nat Med 19: 446-451.
22. Yeh, CH, Fredenburgh, JC and Weitz, JI (2012). Oral direct factor Xa inhibitors. Circ Res 111: 1069-1078.
23. Novobrantseva, TI, Borodovsky, A, Wong, J, Klebanov, B, Zafari, M, Yucius, K et al. (2012). Systemic RNAi-mediated Gene Silencing in Nonhuman Primate and Rodent Myeloid Cells. Mol Ther Nucleic Acids 1: e4.
24. Shi, B, Keough, E, Matter, A, Leander, K, Young, S, Carlini, E et al. (2011). Biodistribution of small interfering RNA at the organ and cellular levels after lipid nanoparticle-mediated delivery. J Histochem Cytochem 59: 727-740.
25. Pannen, BH and Robotham, JL (1995). The acute-phase response. New Horiz 3: 183-197.
26. Giovannini, I, Chiarla, C, Giuliante, F, Vellone, M and Nuzzo, G (2004). Modulation of plasma fibrinogen levels in acute-phase response after hepatectomy. Clin Chem Lab Med 42: 261-265.
27. Walker, B and Lynas, JF (2001). Strategies for the inhibition of serine proteases. Cell Mol Life Sci 58: 596-624.
28. Henry BL, Desai UR (2014). Discovery methodology for the development of direct factor VIIa inhibitors. Expert Opin Drug Discov 9: 859-872.
29. Beaubien, G, Rosinski-Chupin, I, Mattei, MG, Mbikay, M, Chrétien, M and Seidah, NG (1991). Gene structure and chromosomal localization of plasma kallikrein. Biochemistry 30: 1628-1635.
30. Xu, Y, Cai, TQ, Castriota, G, Zhou, Y, Hoos, L, Jochnowitz, N et al. (2014). Factor XIIa inhibition by Infestin-4: in vitro mode of action and in vivo antithrombotic benefit. Thromb Haemost 111: 694-704.
31. Liu, J, Gao, BB, Clermont, AC, Blair, P, Chilcote, TJ, Sinha, S et al. (2011). Hyperglycemia-induced cerebral hematoma expansion is mediated by plasma kallikrein. Nat Med 17: 206-210.
32. Frost, C, Wang, J, Nepal, S, Schuster, A, Barrett, YC, Mosqueda-Garcia, R et al. (2013). Apixaban, an oral, direct factor Xa inhibitor: single dose safety, pharmacokinetics, pharmacodynamics and food effect in healthy subjects. Br J Clin Pharmacol 75: 476-487.
33. Dewerchin, M, Liang, Z, Moons, L, Carmeliet, P, Castellino, FJ, Collen, D et al. (2000). Blood coagulation factor X deficiency causes partial embryonic lethality and fatal neonatal bleeding in mice. Thromb Haemost 83: 185-190.
34. Lee, G, Duan-Porter, W and Metjian, AD (2012). Acquired, non-amyloid related factor X deficiency: review of the literature. Haemophilia 18: 655-663.
35. Strapps, WR, Pickering, V, Muiru, GT, Rice, J, Orsborn, S, Polisky, BA et al. (2010). The siRNA sequence and guide strand overhangs are determinants of in vivo duration of silencing. Nucleic Acids Res 38: 4788-4797.
36. Gindy, ME, Leone, AM and Cunningham, JJ (2012). Challenges in the pharmaceutical development of lipid-based short interfering ribonucleic acid therapeutics. Expert Opin Drug Deliv 9: 171-182.
37. Gindy, ME, Feuston, B, Glass, A, Arrington, L, Haas, RM, Schariter, J et al. (2014). Stabilization of Ostwald ripening in low molecular weight amino lipid nanoparticles for systemic delivery of siRNA therapeutics. Mol Pharm 11: 4143-4153.
38. Ariani, F, Mari, F, Pescucci, C, Longo, I, Bruttini, M, Meloni, I et al. (2004). Real-time quantitative PCR as a routine method for screening large rearrangements in Rett syndrome: Report of one case of MECP2 deletion and one case of MECP2 duplication. Hum Mutat 24: 172-177.