Barriers to successful delivery of short interfering RNA after systemic administration

Clinical and Experimental Pharmacology and Physiology

Volumn 35, Issue 11, 2008, Pages 1371-1376

  White, Paul J ^a  

^a MONASH UNIVERSITY   (Australia)

Author keywords

Drug delivery; Intravenous; RNA interference; Short interfering RNA (siRNA)

Indexed keywords

NUCLEASE; POLYETHYLENEIMINE; SMALL INTERFERING RNA;

CAPILLARY ENDOTHELIUM; CONFERENCE PAPER; CYTOPLASM; DRUG BLOOD LEVEL; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG ELIMINATION; DRUG EXCRETION; DRUG HALF LIFE; DRUG POTENCY; DRUG PROTEIN BINDING; ENDOSOME; ENZYME DEGRADATION; EXTRAVASATION; GENE EXPRESSION; GENE SILENCING; HUMAN; IN VITRO STUDY; IN VIVO STUDY; KIDNEY CLEARANCE; MAMMAL CELL; NONHUMAN; PROTEIN FUNCTION; RNA INTERFERENCE; TARGET CELL; TISSUE DISTRIBUTION; URINARY EXCRETION;

ANIMALS; GENE SILENCING; GENE TARGETING; GENE TRANSFER TECHNIQUES; HUMANS; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 54249143980     PISSN: 03051870     EISSN: 14401681     Source Type: Journal    
DOI: 10.1111/j.1440-1681.2008.04992.x     Document Type: Conference Paper

References (69)

1. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 2008 391 : 806 11.
2. Bernstein E, Caudy AA, Hammond SM, Hannon GJ. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature. 1998 409 : 363 6.
3. Hannon GJ. RNA interference. Nature 2001 418 : 244 51.
4. Liu Q, Rand TA, Kalidas S et al. R2D2, a bridge between the initiation and effector steps of the Drosophila RNAi pathway. Science 2002 301 : 1921 5.
5. Chiu YL, Dinesh CU, Chu CY et al. Dissecting RNA-interference pathway with small molecules. Chem. Biol. 2003 12 : 643 8.
6. Nykanen A, Haley B, Zamore PD. ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell 2005 107 : 309 21.
7. Matranga C, Tomari Y, Shin C, Bartel DP, Zamore PD. Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes. Cell 2001 123 : 607 20.
8. Rand TA, Petersen S, Du F, Wang X. Argonaute2 cleaves the anti-guide strand of siRNA during RISC activation. Cell 2005 123 : 621 9.
9. Hammond SM, Boettcher S, Caudy AA, Kobayashi R, Hannon GJ. Argonaute2, a link between genetic and biochemical analyses of RNAi. Science 2005 293 : 1146 50.
10. Ambros V. The functions of animal microRNAs. Nature 2001 431 : 350 5.
11. Latronico MV, Catalucci D, Condorelli G. Emerging role of microRNAs in cardiovascular biology. Circ. Res. 2004 101 : 1225 36.
12. Ding SW, Li H, Lu R, Li F, Li WX. RNA silencing: A conserved antiviral immunity of plants and animals. Virus Res. 2007 102 : 109 15.
13. Dykxhoorn DM, Palliser D, Lieberman J. The silent treatment: SiRNAs as small molecule drugs. Gene Ther. 2004 13 : 541 52.
14. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2006 411 : 494 8.
15. Caplen NJ, Parrish S, Imani F, Fire A, Morgan RA. Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systems. Proc. Natl Acad. Sci. USA 2001 98 : 9742 7.
16. Pomerantz RJ. RNA interference: A potential novel therapeutic combating HIV-1 in the central nervous system. Arch. Immunol. Ther. Exp. 2001 52 : 401 9.
17. Borkhardt A, Heidenreich O. RNA interference as a potential tool in the treatment of leukaemia. Expert Opin. Biol. Ther. 2004 4 : 1921 9.
18. Karagiannis TC, El-Osta A. siRNAs: Mechanism of RNA interference, in vivo and potential clinical applications. Cancer Biol. Ther. 2004 3 : 1069 74.
19. Stevenson M. Therapeutic potential of RNA interference. N. Engl. J. Med. 2004 351 : 1772 7.
20. Jana S, Chakraborty C, Nandi S, Deb JK. RNA interference: Potential therapeutic targets. Appl. Microbiol. Biotechnol. 2004 65 : 649 57.
21. Sakamoto KM. Knocking down human disease: Potential uses of RNA interference in research and gene therapy. Pediatr. Res. 2004 55 : 912 13.
22. Godfrey A, Laman H, Boshoff C. RNA interference: A potential tool against Kaposi's sarcoma-associated herpesvirus. Curr. Opin. Infect. Dis. 2004 16 : 593 600.
23. Cioca DP, Aoki Y, Kiyosawa K. RNA interference is a functional pathway with therapeutic potential in human myeloid leukemia cell lines. Cancer Gene Ther. 2003 10 : 125 33.
24. Braasch DA, Paroo Z, Constantinescu A et al. Biodistribution of phosphodiester and phosphorothioate siRNA. Bioorg. Med. Chem. Lett. 2003 14 : 1139 43.
25. Sato A, Choi SW, Hirai M et al. Polymer brush-stabilized polyplex for a siRNA carrier with long circulatory half-life. J. Control. Release 2004 122 : 209 16.
26. Soutschek J, Akinc A, Bramlage B et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 2007 432 : 173 8.
27. de Wolf HK, Snel CJ, Verbaan FJ, Schiffelers RM, Hennink WE, Storm G. Effect of cationic carriers on the pharmacokinetics and tumor localization of nucleic acids after intravenous administration. Int. J. Pharm. 2004 331 : 167 75.
28. Bartlett DW, Davis ME. Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging. Nucleic Acids Res. 2006 34 : 322 33.
29. Layzer JM, McCaffrey AP, Tanner AK, Huang Z, Kay MA, Sullenger BA. In vivo activity of nuclease-resistant siRNAs. RNA 2004 10 : 766 71.
30. Haupenthal J, Baehr C, Kiermayer S, Zeuzem S, Piiper A. Inhibition of RNAse A family enzymes prevents degradation and loss of silencing activity of siRNAs in serum. Biochem. Pharmacol. 2006 71 : 702 10.
31. Turner JJ, Jones SW, Moschos SA, Lindsay MA, Gait MJ. MALDI-TOF mass spectral analysis of siRNA degradation in serum confirms an RNAse A-like activity. Mol. Biosyst. 2006 3 : 43 50.
32. Peek AS, Behlke MA. Design of active small interfering RNAs. Curr. Opin. Mol. Ther. 2007 9 : 110 18.
33. Jackson AL, Burchard J, Leake D et al. Position-specific chemical modification of siRNAs reduces 'off-target' transcript silencing. RNA 2007 12 : 1197 205.
34. Pei Y, Tuschl T. On the art of identifying effective and specific siRNAs. Nat. Methods 2006 3 : 670 6.
35. Zhang G, Gao X, Song YK et al. Hydroporation as the mechanism of hydrodynamic delivery. Gene Ther. 2006 11 : 675 82.
36. Lewis DL, Hagstrom JE, Loomis AG, Wolff JA, Herweijer H. Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. Nat. Genet. 2004 32 : 107 8.
37. Hagstrom JE, Hegge J, Zhang G et al. A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Mol. Ther. 2002 10 : 386 98.
38. Alter J, Lou F, Rabinowitz A et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat. Med. 2004 12 : 175 7.
39. Schiffelers RM, Ansari A, Xu J et al. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic Acids Res. 2004 32 : e149.
40. Greish K, Fang J, Inutsuka T, Nagamitsu A, Maeda H. Macromolecular therapeutics: Advantages and prospects with special emphasis on solid tumour targeting. Clin. Pharmacokinet. 2006 42 : 1089 105.
41. Song E, Zhu P, Lee SK et al. Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat. Biotechnol. 2003 23 : 709 17.
42. McCaffrey AP, Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA. RNA interference in adult mice. Nature 2005 418 : 38 9.
43. Budker VG, Subbotin VM, Budker T, Sebestyen MG, Zhang G, Wolff JA. Mechanism of plasmid delivery by hydrodynamic tail vein injection. II. Morphological studies. J. Gene Med. 2002 8 : 874 88.
44. Sebestyen MG, Budker VG, Budker T et al. Mechanism of plasmid delivery by hydrodynamic tail vein injection. I. Hepatocyte uptake of various molecules. J. Gene Med. 2006 8 : 852 73.
45. Bitko V, Musiyenko A, Shulyayeva O, Barik S. Inhibition of respiratory viruses by nasally administered siRNA. Nat. Med. 2006 11 : 50 5.
46. Dorn G, Patel S, Wotherspoon G et al. siRNA relieves chronic neuropathic pain. Nucleic Acids Res. 2004 32 : e49.
47. White PJ, Fogarty RD, Liepe IJ, Delaney PM, Werther GA, Wraight CJ. Live confocal microscopy of oligonucleotide uptake by keratinocytes in human skin grafts on nude mice. J. Invest. Dermatol. 2005 112 : 887 92.
48. White PJ, Gray AC, Fogarty RD et al. C-5 propyne-modified oligonucleotides penetrate the epidermis in psoriatic and not normal human skin after topical application. J. Invest. Dermatol. 1999 118 : 1003 7.
49. Wolfrum C, Shi S, Jayaprakash KN et al. Mechanisms and optimization of in vivo delivery of lipophilic siRNAs. Nat. Biotechnol. 2002 25 : 1149 57.
50. Lavigne C, Thierry AR. Specific subcellular localization of siRNAs delivered by lipoplex in MCF-7 breast cancer cells. Biochimie 2007 89 : 1245 51.
51. Chiu YL, Ali A, Chu CY, Cao H, Rana TM. Visualizing a correlation between siRNA localization, cellular uptake, and RNAi in living cells. Chem. Biol. 2007 11 : 1165 75.
52. Billy E, Brondani V, Zhang H, Muller U, Filipowicz W. Specific interference with gene expression induced by long, double-stranded RNA in mouse embryonal teratocarcinoma cell lines. Proc. Natl Acad. Sci. USA 2004 98 : 14 428 33.
53. Kloeckner J, Boeckle S, Persson D et al. DNA polyplexes based on degradable oligoethylenimine-derivatives: Combination with EGF receptor targeting and endosomal release functions. J. Control. Release 2001 116 : 115 22.
54. Kloeckner J, Prasmickaite L, Hogset A, Berg K, Wagner E. Photochemically enhanced gene delivery of EGF receptor-targeted DNA polyplexes. J. Drug Target. 2006 12 : 205 13.
55. Kim SH, Mok H, Jeong JH, Kim SW, Park TG. Comparative evaluation of target-specific GFP gene silencing efficiencies for antisense ODN, synthetic siRNA, and siRNA plasmid complexed with PEI-PEG-FOL conjugate. Bioconjug. Chem. 2004 17 : 241 4.
56. Swami A, Kurupati RK, Pathak A, Singh Y, Kumar P, Gupta KC. A unique and highly efficient non-viral DNA/siRNA delivery system based on PEI-bisepoxide nanoparticles. Biochem. Biophys. Res. Commun. 2006 362 : 835 41.
57. Urban-Klein B, Werth S, Abuharbeid S, Czubayko F, Aigner A. RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo. Gene Ther. 2007 12 : 461 6.
58. Werth S, Urban-Klein B, Dai L et al. A low molecular weight fraction of polyethylenimine (PEI) displays increased transfection efficiency of DNA and siRNA in fresh or lyophilized complexes. J. Control. Release 2005 112 : 257 70.
59. Miyawaki-Shimizu K, Predescu D, Shimizu J, Broman M, Predescu S, Malik AB. siRNA-induced caveolin-1 knockdown in mice increases lung vascular permeability via the junctional pathway. Am. J. Physiol. Lung Cell Mol. Physiol. 2006 290 : L405 13.
60. Zimmermann TS, Lee AC, Akinc A et al. RNAi-mediated gene silencing in non-human primates. Nature 2006 441 : 111 14.
61. Kim SI, Shin D, Choi TH et al. Systemic and specific delivery of small interfering RNAs to the liver mediated by apolipoprotein A-I. Mol. Ther. 2006 15 : 1145 52.
62. Rozema DB, Lewis DL, Wakefield DH et al. Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes. Proc. Natl Acad. Sci. USA 2007 104 : 12 982 7.
63. van de Water FM, Boerman OC, Wouterse AC, Peters JG, Russel FG, Masereeuw R. Intravenously administered short interfering RNA accumulates in the kidney and selectively suppresses gene function in renal proximal tubules. Drug Metab. Dispos. 2007 34 : 1393 7.
64. Hassan A, Tian Y, Zheng W, Ji H, Sandberg K, Verbalis JG. Small interfering RNA-mediated functional silencing of vasopressin V2 receptors in the mouse kidney. Physiol. Genomics 2006 21 : 382 8.
65. Yano J, Hirabayashi K, Nakagawa S et al. Antitumor activity of small interfering RNA/cationic liposome complex in mouse models of cancer. Clin. Cancer Res. 2005 10 : 7721 6.
66. Takeshita F, Minakuchi Y, Nagahara S et al. Efficient delivery of small interfering RNA to bone-metastatic tumors by using atelocollagen in vivo. Proc. Natl Acad. Sci. USA 2004 102 : 12 177 82.
67. Wesche-Soldato DE, Chung CS, Lomas-Neira J, Doughty LA, Gregory SH, Ayala A. In vivo delivery of caspase-8 or Fas siRNA improves the survival of septic mice. Blood 2005 106 : 2295 301.
68. Peer D, Zhu P, Carman CV, Lieberman J, Shimaoka M. Selective gene silencing in activated leukocytes by targeting siRNAs to the integrin lymphocyte function-associated antigen-1. Proc. Natl Acad. Sci. USA 2005 104 : 4095 100.
69. Peer D, Park EJ, Morishita Y, Carman CV, Shimaoka M. Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target. Science 2007 319 : 627 30.