Sustained polymeric delivery of gene silencing antisense ODNs, siRNA, DNAzymes and ribozymes: In vitro and in vivo studies

Journal of Drug Targeting

Volumn 12, Issue 6, 2004, Pages 393-404

  Khan, Alim ^a,b   Benboubetra, Mustapha ^a,c   Sayyed, Pakeeza Z ^b   Ng, Keng Wooi ^a   Fox, Stephen ^a   Beck, Gordon ^d   Benter, Ibrahim F ^e   Akhtar, Saghir ^a,b  

^a CARDIFF UNIVERSITY   (United Kingdom)

^b ASTON UNIVERSITY   (United Kingdom)

^c FERHAT ABBAS UNIVERSITY   (Algeria)

^d ROCHE PRODUCTS LTD   (United Kingdom)

^e KUWAIT UNIVERSITY   (Kuwait)

Author keywords

Antisense oligonucleotides; DNA enzymes; Microspheres; PLGA; RNA enzyme; siRNA; Whole body autoradiography

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DEOXYRIBONUCLEASE; FLUOROURACIL; MICROSPHERE; POLYLACTIDE; POLYMER; RIBOZYME; SMALL INTERFERING RNA;

ANIMAL TISSUE; ARTICLE; CELLULAR DISTRIBUTION; CONTROLLED STUDY; DRUG DISTRIBUTION; DRUG ELIMINATION; DRUG RELEASE; DRUG STABILITY; DRUG TISSUE LEVEL; FEMALE; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE SEQUENCE; GENE SILENCING; GENETIC STABILITY; IN VITRO STUDY; IN VIVO STUDY; ISOTOPE LABELING; KIDNEY; LIVER; MOUSE; NONHUMAN; PRIORITY JOURNAL; SEQUENCE ANALYSIS; SPLEEN; WHOLE BODY AUTORADIOGRAPHY;

ANIMALS; AUTORADIOGRAPHY; DNA, CATALYTIC; DRUG DELIVERY SYSTEMS; FLUOROURACIL; GENE SILENCING; KIDNEY; LACTIC ACID; LIVER; MICE; MICE, INBRED BALB C; MICROSPHERES; OLIGONUCLEOTIDES, ANTISENSE; POLYGLYCOLIC ACID; POLYMERS; RNA, CATALYTIC; RNA, SMALL INTERFERING; SPLEEN; TIME FACTORS;

EID: 4644293047     PISSN: 1061186X     EISSN: None     Source Type: Journal    
DOI: 10.1080/10611860400003858     Document Type: Article

References (49)

1. Akhtar, S. and Lewis, K.J. (1997) "Antisense oligodeoxynucleotide delivery to cultured macrophages is improved by incorporation into sustained release biodegradable polymer microspheres", Int. J. Pharm. 151, 57-67.
2. Akhtar, S., Hughes, M.D., Khan, A., Bibby, M., Hussain, M., Nawaz, Q., Double, J. and Sayyed, P. (2000) "The delivery of antisense therapeutics", Adv. Drug. Deliv. Rev. 44, 3-21.
3. Alvarez-Salas, L.M., Benitez-Hess, M.L. and DiPaolo, J.A. (2004) "Advances in the development of ribozymes and antisense oligodeoxynucleotides as antiviral agents for human papilloma-viruses", Antivir. Ther. 8, 265-278.
4. Beale, G., Hollins, A.J., Benboubetra, M., Sohail, M., Fox, S.P., Benter, I. and Akhtar, S. (2003) "Gene silencing nucleic acids designed by scanning arrays: Anti-EGFR activity of siRNA, ribozyme and DNA enzymes targeting a single hybridization-accessible region using the same delivery system", J. Drug Target. 11, 449-456.
5. Burgin, A.B., Jr, Gonzalez, C., Matulic-Adamic, J., Karpeisky, A.M., Usman, N., McSwiggen, J.A., and Beigelman, L. (1996) "Chemically modified hammerhead ribozymes with improved catalytic rates", Biochemistry 35(45), 14090-14097.
6. Carome, M.A., Kang, Y.H., Bohen, E.M. and Nicholson, D.E. (1997) "Distribution of the cellular uptake of phosphorothioate oligodeoxynucleotides in the rat kidney in vivo", Nephron 75, 82-87.
7. Cohen, I., Yoshioka, T., Luccarelli, M., Hwang, L.H. and Langer, R. (1991) "Controlled delivery systems for proteins based on poly(lactid/glycolic acid) microspheres", Pharm. Res. 28, 2018-2022.
8. Curtis, C.G., Cross, S.AM., McCulloch, R.J. and Powell, G.M. (1981) In: Treherne, J.E. and Rubery, P.H., eds, Whole Body Autoradiography (Academic Press Inc., London, UK).
9. Davidson, B.L. (2003) "Hepatic diseases-hitting the target with inhibitory RNAs", N. Engl. J. Med. 349, 2357-2359.
10. Dykxhoorn, D.M., Novina, C.D. and Sharp, P.A. (2003) "Killing the messenger: short RNAs that silence gene expression", Nat. Rev. Mol. Cell Biol. 4, 457.
11. Fattal, E., Vauthier, C., Aynie, I., Nakada, Y., Lambert, G., Malvy, C. and Couvreur, P. (1998) "Biodegradable polyalkylcyanoacrylate nanoparticles for the delivery of oligonucleotides", J. Controlled Rel. 53, 137-143.
12. Feng, Y., Kong, Y.Y., Wang, Y. and Qi, G.R. (2001) "Intracellular inhibition of the replication of hepatitis B virus by hammerhead ribozymes", J. Gastroenterol. Hepatol. 16, 1125-1130.
13. Fire, A., Xu, S., Montgomery, M.K., Kostas, S.A., Driver, S.E. and Mello, C.C. (1998) "Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans", Nature 391, 806-811.
14. Gilmore, I., Fox, S.P., Hollins, A.J., Sohail, M. and Akhtar, S. (2004) "The design and exogenous delivery of siRNA for post-transcriptional gene silencing", J. Drug Target.
15. Graham, M.J., Freier, S.M., Crooke, R.M., Ecker, D.J., Maslova, R.N. and Lesnik, E.A. (1993) "Tritium labelling of antisense oligonucleotides by exchange with tritiated water", Nucl. Acids Res. 21, 3737-3743.
16. Hoffart, V., Ubrich, N., Lamprecht, A., Bachelier, K., Vigneron, C., Lecompte, T., Hoffman, M. and Maincent, P. (2003) "Microencapsulation of low molecular weight heparin into polymeric particles designed with biodegradable and nonbiodegradable polycationic polymers", Drug Deliv. 10, 1-7.
17. Holle, L., Hicks, L., Song, W., Holle, E., Wagner, T. and Yu, X. (2004) "Bcl-2 targeting siRNA expressed by a T7 vector system inhibits human tumor cell growth in vitro", Int. J. Oncol. 24, 615-621.
18. Hudson, J.W., Alarcon, V.B. and Elinson, R.P. (1996) "Identification of new localized RNAs in the Xenopus oocyte by differential display PCR", Dev. Genet. 19, 190-198.
19. Hughes, M.D., Hussain, M., Nawaz, Q., Sayyed, P. and Akhtar, S. (2001) "The cellular delivery of antisense oligonucleotides and ribozymes", Drug Discov. Today 6, 303-315.
20. Husmann, M., Schenderlein, S., Luck, M., Lindner, H. and Kleinebudde, P. (2002) "Polymer erosion in PLGA microparticles produced by phase separation method", Int. J. Pharm. 242, 277-280.
21. Inagaki, H., Bishop, A.E., Eimoto, T. and Polak, J.M. (1992) "Autoradiographic localization of endothelin-1 binding sites in human colonic cancer tissue", Pathology 68, 263-267.
22. Johnson, B.A., McClain, S.G., Doran, E.R., Tice, G. and Kirsh, M.A. (1990) "Rapid purification of synthetic oligonucleotides: A convenient alternative to HPLC and polyacrylamide gel electrophoresis", Biotechniques 8, 424-429.
23. Khan, A., Sommer, W., Fuxe, K. and Akhtar, S. (2000) "Site-specific administration of antisense oligonucleotides using biodegradable polymer microspheres provides sustained delivery and improved subcellular biodistribution in the neostriatum of the rat brain", J. Drug Target. 8, 319-334.
24. Labarre, P., Papon, J., Moreau, M.F., Madelmont, J.C. and Veyre, A. (1998) "A new quantitative method to evaluate the biodistribution of a radiolabelled tracer for melanoma using whole-body cryosectioning and a gaseous detector: comparison with conventional tissue combustion technology", Eur. J. Nucl. Med. 25, 109-114.
25. Lambert, G., Fattal, E. and Couvreur, P. (2001) "Nanoparticulate systems for the delivery of antisense oligonucleotides", Adv. Drug Deliv. Rev. 47, 99-112.
26. Lee, C.H., Ni, Y.N., Chen, C.C., Chou, C. and Chang, F.H. (2003) "Synergistic effect of polyethylenimine and cationic liposomes in nucleic acid delivery to human cancer cells", Biochim. Biophys. Acta 1611, 55-62.
27. Lewis, K.J., Irwin, W.J. and Akhtar, S. (1998) "Development of sustained-release biodegradable polymer system for site-specific delivery of oligonucleotides: Characterisation of P(LA-GA) copolymer microspheres in vitro", J. Drug Target. 5, 291-302.
28. MacKellar, C., Graham, D., Will, D.W., Burgess, S. and Brown, T. (1992) "Synthesis and physical properties of anti-HIV antisense oligonucleotides bearing terminal lipophilic groups", Nucleic Acids Res. 11, 3411-3417.
29. McManus, M.T. and Sharp, P.A. (2002) "Gene silencing in mammals by small interfering RNAs", Nat. Rev. Genet. 3, 737-747.
30. Miyagishi, M., Sumimoto, H., Miyoshi, H., Kawakami, Y. and Taira, K. (2004) "Optimization of an siRNA-expression system with an improved hairpin and its significant suppressive effects in mammalian cells", J. Gene Med. 6, 715-723.
31. Moore, V.A., Dunnion, D.J., Brown, T., Irwin, W.J. and Akhtar, S. (1997) "Interaction of oligonucleotide-conjugates with the dipeptide transporter system in Caco-2 cells", Biochem. Pharmacol. 53, 1223-1228.
32. Mumtaz, M.M., Farooqui, M.Y.H., Cannon-Cooke, E.P. and Ahmed, A.E. (1997) "Propionitrile: whole body autoradiography, conventional toxicokinetic and metabolism studies in rats", Toxicol. Ind. Health 13, 27-41.
33. Nicklin, P.L., Craig, S.J. and Philips, J.A. (1998) "Pharmacokinetic properties of phosphorothionates in animals-absorption, distribution, metabolism and elimination", In: Crooke, S., ed., Antisense Research and Application (Springer, New York ), pp 141-168.
34. Okada, H. and Toguchi, H. (1995) "Biodegradable microspheres in drug delivery", Crit. Rev. Ther. Drug Carrier. Syst. 12, 1-99.
35. Panyam, J. and Labhasetwar, V. (2003) "Biodegradable nanoparticles for drug and gene delivery to cells and tissue", Adv. Drug Deliv. Rev. 55, 329-347.
36. Park, T.G. (1995) "Degradation of poly(lactic-co-glycolic acid) microspheres: effect of copolymer composition", Biomaterials 16, 1123-1130.
37. Pouton, C.W. and Akhtar, S. (1996) "Biosynthetic polyhydroxyalkanoates and their potential in drug delivery", Adv. Drug Deliv. Rev. 18, 133-162.
38. Prabhu, S., Sullivan, J.L. and Betageri, G.V. (2002) "Comparative assessment of in vitro release kinetics of calcitonin polypeptide from biodegradable microspheres", Drug Deliv. 9, 195-198.
39. Rifai, N., Chao, F.F., Pham, Q., Thiessen, J. and Soldin, S.J. (1996) "The role of lipoproteins in the transport and uptake of cyclosporine and dihydro-tacrolimus into HepG2 and JURKAT cell lines", Clin. Biochem. 29, 149-155.
40. Sambrook, J., Fritsh, E.F. and Maniatis, T. (1989) Molecular Cloning. A laboratory Manual, 2nd Ed. (Cold Spring Harbour Laboratory Press, Cold Spring, Harbour, NY, USA) Vol. 1-3.
41. Santoro, S.W. and Joyce, G.F. (1997) "A general purpose RNA-cleaving DNA enzyme", Proc. Natl Acad. Sci. USA 94, 4262-4666.
42. Santoro, S.W. and Joyce, G.F. (1998) "Mechanism and utility of an RNA-cleaving DNA enzyme", Biochemistry 22, 13330-13342.
43. Sioud, M. (2004). Therapeutic SiRNAs, TIPS, Jan.
44. Ullberg, S. (1977) "The technique of whole body autography: cryosectioning of large specimens", In: Alvfeldt, O., ed., Science Tools (LKB-Producer AB, Swden).
45. Vlassov, V.V., Balakireva, L.A. and Yakubov, L.A. (1994) "Transport of oligonucleotides across natural and model membranes", Biochim. Biophys. Acta 1197, 95-108.
46. Wilson, A.G.E. and Kraus, L.J. (1995) "Application of direct analytical and storage phosphor techniques in quantitating whole body autoradiography data", Toxicol. Methods 5, 15-20.
47. Zamore, P.D., Tuschl, T., Sharp, P.A. and Bartel, D.P. (2000) "RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals", Cell 101, 25-33.
48. Zane, P.A., Obuck, A.J., Walter, R.E., Robertson, P. and Tripp, S.L. (1997) "Validation of procedures for quantitative whole body autoradiography using digital imaging", J. Pharm. Sci. 86, 733-738.
49. Zhang, Q., Ohannesian, D.W. and Erickson, L.C. (2004) "Hammerhead ribozyme-mediated sensitization of human tumor cells after treatment with 1,3-bis(2-chloroethyl)-1-nitrosourea", J. Pharmacol. Exp. Ther., Jan 23 [Epub ahead of print].