Delivery of siRNA to the mouse lung via a functionalized lipopolyamine

Molecular Therapy

Volumn 20, Issue 1, 2012, Pages 91-100

  Polach, Kevin J ^a   Matar, Majed ^a   Rice, Jennifer ^a   Slobodkin, Gregory ^a   Sparks, Jeff ^a   Congo, Richard ^a   Rea Ramsey, Angela ^a   McClure, Diane ^a   Brunhoeber, Elaine ^a   Krampert, Monika ^b   Schuster, Andrea ^b   Jahn Hofmann, Kerstin ^b   John, Matthias ^b   Vornlocher, Hans Peter ^b   Fewell, Jason G ^a   Anwer, Khursheed ^a   Geick, Anke ^b  

^a EGEN Inc   (United States)

^b Roche Kulmbach GmbH   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CAVEOLIN 1; CELL MARKER; LIPOPOLYAMINE; LIPOSOME; MACROGOL DERIVATIVE; NANOPARTICLE; POLYAMINE DERIVATIVE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BIOACCUMULATION; BIOSAFETY; BLOOD CHEMISTRY; CELL TYPE; CHEMICAL MODIFICATION; COMPLEX FORMATION; CONTROLLED STUDY; ENDOTHELIUM CELL; FEMALE; GENE SILENCING; GENE TARGETING; HISTOPATHOLOGY; IMMUNOHISTOCHEMISTRY; IN VIVO STUDY; LIPOSOMAL GENE DELIVERY SYSTEM; LUNG PARENCHYMA; MOUSE; NANOFABRICATION; NONHUMAN; PHYSICAL CHEMISTRY; RNA INTERFERENCE; TISSUE DISTRIBUTION; TOXICITY TESTING;

MUS;

EID: 84856956282     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.210     Document Type: Article

References (49)

1. Tiemann, K and Rossi, JJ (2009). RNAi-based therapeutics-current status, challenges and prospects. EMBO Mol Med 1: 142-151.
2. Grimm, D (2009). Small silencing RNAs: state-of-the-art. Adv Drug Deliv Rev 61: 672-703.
3. Vaishnaw, AK, Gollob, J, Gamba-Vitalo, C, Hutabarat, R, Sah, D, Meyers, R et al. (2010). A status report on RNAi therapeutics. Silence 1: 14.
4. Soutschek, J, Akinc, A, Bramlage, B, Charisse, K, Constien, R, Donoghue, M et al. (2004). Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432: 173-178. (Pubitemid 39545841)
5. Akhtar, S and Benter, IF (2007). Nonviral delivery of synthetic siRNAs in vivo. J Clin Invest 117: 3623-3632. (Pubitemid 350224068)
6. Samad, A, Sultana, Y and Aqil, M (2007). Liposomal drug delivery systems: an update review. Curr Drug Deliv 4: 297-305. (Pubitemid 47554274)
7. Akinc, A, Zumbuehl, A, Goldberg, M, Leshchiner, ES, Busini, V, Hossain, N et al. (2008). A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat Biotechnol 26: 561-569. (Pubitemid 351668042)
8. Farokhzad, OC (2008). Nanotechnology for drug delivery: the perfect partnership. Expert Opin Drug Deliv 5: 927-929.
9. Li, SD, Chen, YC, Hackett, MJ and Huang, L (2008). Tumor-targeted delivery of siRNA by self-assembled nanoparticles. Mol Ther 16: 163-169.
10. Bennewitz, MF and Saltzman, WM (2009). Nanotechnology for delivery of drugs to the brain for epilepsy. Neurotherapeutics 6: 323-336.
11. Farokhzad, OC and Langer, R (2009). Impact of nanotechnology on drug delivery. ACS Nano 3: 16-20.
12. Gao, K and Huang, L (2009). Nonviral methods for siRNA delivery. Mol Pharm 6: 651-658.
13. Reischl, D and Zimmer, A (2009). Drug delivery of siRNA therapeutics: potentials and limits of nanosystems. Nanomedicine 5: 8-20.
14. Tseng, YC, Mozumdar, S and Huang, L (2009). Lipid-based systemic delivery of siRNA. Adv Drug Deliv Rev 61: 721-731.
15. Whitehead, KA, Langer, R and Anderson, DG (2009). Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov 8: 129-138.
16. Higuchi, Y, Kawakami, S and Hashida, M (2010). Strategies for in vivo delivery of siRNAs: recent progress. BioDrugs 24: 195-205.
17. Schroeder, A, Levins, CG, Cortez, C, Langer, R and Anderson, DG (2010). Lipid-based nanotherapeutics for siRNA delivery. J Intern Med 267: 9-21.
18. Semple, SC, Akinc, A, Chen, J, Sandhu, AP, Mui, BL, Cho, CK et al. (2010). Rational design of cationic lipids for siRNA delivery. Nat Biotechnol 28: 172-176.
19. Akinc, A, Goldberg, M, Qin, J, Dorkin, JR, Gamba-Vitalo, C, Maier, M et al. (2009). Development of lipidoid-siRNA formulations for systemic delivery to the liver. Mol Ther 17: 872-879.
20. Santel, A, Aleku, M, Keil, O, Endruschat, J, Esche, V, Fisch, G et al. (2006). A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium. Gene Ther 13: 1222-1234. (Pubitemid 44184716)
21. Juliano, R, Bauman, J, Kang, H and Ming, X (2009). Biological barriers to therapy with antisense and siRNA oligonucleotides. Mol Pharm 6: 686-695.
22. Behlke, MA (2006). Progress towards in vivo use of siRNAs. Mol Ther 13: 644-670.
23. Santel, A, Aleku, M, Keil, O, Endruschat, J, Esche, V, Durieux, B et al. (2006). RNA interference in the mouse vascular endothelium by systemic administration of siRNAlipoplexes for cancer therapy. Gene Ther 13: 1360-1370. (Pubitemid 44323692)
24. Davis, ME, Zuckerman, JE, Choi, CH, Seligson, D, Tolcher, A, Alabi, CA et al. (2010). Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 464: 1067-1070.
25. Akinc, A, Querbes, W, De, S, Qin, J, Frank-Kamenetsky, M, Jayaprakash, KN et al. (2010). Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol Ther 18: 1357-1364.
26. Bisgaier, CL, Siebenkas, MV and Williams, KJ (1989). Effects of apolipoproteins A-IV and A-I on the uptake of phospholipid liposomes by hepatocytes. J Biol Chem 264: 862-866. (Pubitemid 19038065)
27. Love, KT, Mahon, KP, Levins, CG, Whitehead, KA, Querbes, W, Dorkin, JR et al. (2010). Lipid-like materials for low-dose, in vivo gene silencing. Proc Natl Acad Sci USA 107: 1864-1869.
28. Tao, W, Davide, JP, Cai, M, Zhang, GJ, South, VJ, Matter, A et al. (2010). Noninvasive imaging of lipid nanoparticle-mediated systemic delivery of small-interfering RNA to the liver. Mol Ther 18: 1657-1666.
29. Alexis, F, Pridgen, E, Molnar, LK and Farokhzad, OC (2008). Factors affecting the clearance and biodistribution of polymeric nanoparticles. Mol Pharm 5: 505-515.
30. Immordino, ML, Dosio, F and Cattel, L (2006). Stealth liposomes: review of the basic science, rationale, and clinical applications, existing and potential. Int J Nanomedicine 1: 297-315.
31. Klibanov, AL, Maruyama, K, Torchilin, VP and Huang, L (1990). Amphipathic polyethyleneglycols effectively prolong the circulation time of liposomes. FEBS Lett 268: 235-237. (Pubitemid 20233874)
32. Robbins, M, Judge, A and MacLachlan, I (2009). siRNA and innate immunity. Oligonucleotides 19: 89-102.
33. Maus, UA, Wellmann, S, Hampl, C, Kuziel, WA, Srivastava, M, Mack, M et al. (2005). CCR2-positive monocytes recruited to inflamed lungs downregulate local CCL2 chemokine levels. Am J Physiol Lung Cell Mol Physiol 288: L350-L358. (Pubitemid 40105374)
34. Moore, BB, Murray, L, Das, A, Wilke, CA, Herrygers, AB and Toews, GB (2006). The role of CCL12 in the recruitment of fibrocytes and lung fibrosis. Am J Respir Cell Mol Biol 35: 175-181. (Pubitemid 44175316)
35. Szymczak, WA and Deepe, GS Jr (2009). The CCL7-CCL2-CCR2 axis regulates IL-4 production in lungs and fungal immunity. J Immunol 183: 1964-1974.
36. Bauer, JW, Baechler, EC, Petri, M, Batliwalla, FM, Crawford, D, Ortmann, WA et al. (2006). Elevated serum levels of interferon-regulated chemokines are biomarkers for active human systemic lupus erythematosus. PLoS Med 3: e491.
37. Lee, PY, Li, Y, Kumagai, Y, Xu, Y, Weinstein, JS, Kellner, ES et al. (2009). Type I interferon modulates monocyte recruitment and maturation in chronic inflammation. Am J Pathol 175: 2023-2033.
38. Judge, AD, Bola, G, Lee, AC and MacLachlan, I (2006). Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo. Mol Ther 13: 494-505. (Pubitemid 43257617)
39. Beyerle, A, Braun, A, Merkel, O, Koch, F, Kissel, T and Stoeger, T (2011). Comparative in vivo study of poly(ethylene imine)/siRNA complexes for pulmonary delivery in mice. J Control Release 151: 51-56.
40. Ghosn, B, Singh, A, Li, M, Vlassov, AV, Burnett, C, Puri, N et al. (2010). Efficient gene silencing in lungs and liver using imidazole-modified chitosan as a nanocarrier for small interfering RNA. Oligonucleotides 20: 163-172.
41. Günther, M, Lipka, J, Malek, A, Gutsch, D, Kreyling, W and Aigner, A (2011). Polyethylenimines for RNAi-mediated gene targeting in vivo and siRNA delivery to the lung. Eur J Pharm Biopharm 77: 438-449.
42. Merkel, OM, Mintzer, MA, Librizzi, D, Samsonova, O, Dicke, T, Sproat, B et al. (2010). Triazine dendrimers as nonviral vectors for in vitro and in vivo RNAi: the effects of peripheral groups and core structure on biological activity. Mol Pharm 7: 969-983.
43. Varkouhi, AK, Lammers, T, Schiffelers, RM, van Steenbergen, MJ, Hennink, WE and Storm, G (2011). Gene silencing activity of siRNA polyplexes based on biodegradable polymers. Eur J Pharm Biopharm 77: 450-457.
44. Garbuzenko, OB, Saad, M, Betigeri, S, Zhang, M, Vetcher, AA, Soldatenkov, VA et al. (2009). Intratracheal versus intravenous liposomal delivery of siRNA, antisense oligonucleotides and anticancer drug. Pharm Res 26: 382-394.
45. Gutbier, B, Kube, SM, Reppe, K, Santel, A, Lange, C, Kaufmann, J et al. (2010). RNAimediated suppression of constitutive pulmonary gene expression by small interfering RNA in mice. Pulm Pharmacol Ther 23: 334-344.
46. Garbuzenko, OB, Saad, M, Pozharov, VP, Reuhl, KR, Mainelis, G and Minko, T (2010). Inhibition of lung tumor growth by complex pulmonary delivery of drugs with oligonucleotides as suppressors of cellular resistance. Proc Natl Acad Sci USA 107: 10737-10742.
47. Damha, MJ and Ogilvie, KK (1993). Oligoribonucleotide synthesis. The silylphosphoramidite method. Methods Mol Biol 20: 81-114.
48. Iyer, RP, Uznanski, B, Boal, J, Storm, C, Egan, W, Matsukura, M et al. (1990). Abasic oligodeoxyribonucleoside phosphorothioates: synthesis and evaluation as anti-HIV-1 agents. Nucleic Acids Res 18: 2855-2859.
49. Constien, R, Forde, A, Liliensiek, B, Gröne, HJ, Nawroth, P, Hämmerling, G et al. (2001). Characterization of a novel EGFP reporter mouse to monitor Cre recombination as demonstrated by a Tie2 Cre mouse line. Genesis 30: 36-44.