Study on development of polymeric micellar gene carrier and evaluation of its functionality

Biological and Pharmaceutical Bulletin

Volumn 36, Issue 7, 2013, Pages 1045-1051

  Oba, Makoto ^a  

^a NAGASAKI UNIVERSITY   (Japan)

Author keywords

Block copolymer; Gene carrier; Gene delivery system; Gene therapy; Polymeric micelle; Polyplex micelle

Indexed keywords

DISULFIDE CROSSLINKED POLYPLEX; DRUG CARRIER; INTRACELLULAR TRAFFICKING CONTROLLABLE POLYPLEX; MACROGOL DETCHABLE POLYPLEX; MACROGOL POLY[N[N (2 AMINOETHYL) 2 AMINOETHYL]ASPARTAMIDE] POLYPLEX; PLASMID DNA; POLYMERIC MICELLAR GENE CARRIER; SMALL INTERFERING RNA; TARGETABLE POLYPLEX; THREE LAYERED POLYPLEX; UNCLASSIFIED DRUG;

CANCER GENE THERAPY; CANCER MODEL; DISULFIDE BOND; DRUG CYTOTOXICITY; DRUG DOSAGE FORM COMPARISON; DRUG EFFECT; DRUG EFFICACY; DRUG TARGETING; EXCIPIENT COMPATIBILITY; GENE DELIVERY SYSTEM; HYDROPHOBICITY; MICELLE; MICELLIZATION; MOLECULAR INTERACTION; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PANCREAS ADENOCARCINOMA; PH MEASUREMENT; PHYSICAL CHEMISTRY; PROCESS DEVELOPMENT; REVIEW;

DNA; DRUG CARRIERS; GENE TRANSFER TECHNIQUES; MICELLES; POLYMERS;

EID: 84880445595     PISSN: 09186158     EISSN: 13475215     Source Type: Journal    
DOI: 10.1248/bpb.b13-00287     Document Type: Review

References (48)

1. Tian J, Xu Z, Smith JS, Hofherr SE, Barry MA, Byrnes AP. Adenovirus activates complement by distinctly different mechanisms in vitro and in vivo: indirect complement activation by virions in vivo. J. Virol., 83, 5648-5658 (2009).
2. Huang X, Yang Y. Innate immune recognition of viruses and viral vectors. Hum. Gene Ther., 20, 293-301 (2009).
3. Thorne BA, Takeya RK, Peluso RW. Manufacturing recombinant adeno-associated viral vectors from producer cell clones. Hum. Gene Ther., 20, 707-714 (2009).
4. Mastrobattista E, van der Aa MAEM, Hennink WE, Crommelin DJ. Artificial viruses: a nanotechnological approach to gene delivery. Nat. Rev. Drug Discov., 5, 115-121 (2006).
5. Mintzer MA, Simanek EE. Nonviral vectors for gene delivery. Chem. Rev., 109, 259-302 (2009).
6. Pedroso de Lima MC, Simões S, Pires P, Faneca H, Düzgünes N. Cationic lipid-DNA complexes in gene delivery: from biophysics to biological applications. Adv. Drug Deliv. Rev., 47, 277-294 (2001).
7. Li W, Szoka FC Jr. Lipid-based nanoparticles for nucleic acid delivery. Pharm. Res., 24, 438-449 (2007).
8. Merdan T, Kopecek J, Kissel T. Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. Adv. Drug Deliv. Rev., 54, 715-758 (2002).
9. Pack DW, Hoffman AS, Pun S, Stayton PS. Design and development of polymers for gene delivery. Nat. Rev. Drug Discov., 4, 581-593 (2005).
10. Kakizawa Y, Kataoka K. Block copolymer micelles for delivery of gene and related compounds. Adv. Drug Deliv. Rev., 54, 203-222 (2002).
11. Itaka K, Yamauchi K, Harada A, Nakamura K, Kawaguchi H, Kataoka K. Polyion complex micelles from plasmid DNA and poly(ethylene glycol)-poly(l- lysine) block copolymer as serum-tolerable polyplex system: physicochemical properties of micelles relevant to gene transfection efficiency. Biomaterials, 24, 4495-4506 (2003).
12. Harada-Shiba M, Yamauchi K, Harada A, Takamisawa I, Shimokado K, Kataoka K. Polyion complex micelles as vectors in gene therapy -pharmacokinetics and in vivo gene transfer. Gene Ther., 9, 407-414 (2002).
13. Kakizawa Y, Harada A, Kataoka K. Glutathione-sensitive stabilization of block copolymer micelles composed of antisense DNA and thiolated poly(ethylene glycol)-block-poly(l-lysine): a potential carrier for systemic delivery of antisense DNA. Biomacromolecules, 2, 491-497 (2001).
14. Miyata K, Kakizawa Y, Nishiyama N, Harada A, Yamasaki Y, Koyama H, Kataoka K. Block catiomer polyplexes with regulated densities of charge and disulfide cross-linking directed to enhance gene expression. J. Am. Chem. Soc., 126, 2355-2361 (2004).
15. Meister A, Anderson ME. Glutathione. Annu. Rev. Biochem., 52, 711-760 (1983).
16. Miyata K, Kakizawa Y, Nishiyama N, Yamasaki Y, Watanabe T, Kohara M, Kataoka K. Freeze-dried formulations for in vivo gene delivery of PEGylated polyplex micelles with disulfide crosslinked cores to the liver. J. Control. Release, 109, 15-23 (2005).
17. Oba M, Vachutinsky Y, Miyata K, Kano MR, Ikeda S, Nishiyama N, Itaka K, Miyazono K, Koyama H, Kataoka K. Antiangiogenic gene therapy of solid tumor by systemic injection of polyplex micelles loading plasmid DNA encoding soluble flt-1. Mol. Pharm., 7, 501-509 (2010).
18. Matsumoto S, Christie RJ, Nishiyama N, Miyata K, Ishii A, Oba M, Koyama H, Yamasaki Y, Kataoka K. Environment-responsive block copolymer micelles with a disulfide cross-linked core for enhanced siRNA delivery. Biomacromolecules, 10, 119-127 (2009).
19. Christie RJ, Miyata K, Matsumoto Y, Nomoto T, Menasco D, Lai TC, Pennisi M, Osada K, Fukushima S, Nishiyama N, Yamasaki Y, Kataoka K. Effect of polymer structure on micelles formed between siRNA and cationic block copolymer comprising thiols and amidines. Biomacromolecules, 12, 3174-3185 (2011).
20. Matsumura Y, Maeda H. A new concept for macromolecular therapeutics in cancer chemotherapy: mechanism of tumoritropic accumulation of proteins and the antitumor agent smancs. Cancer Res., 46, 6387-6392 (1986).
21. Wakebayashi D, Nishiyama N, Yamasaki Y, Itaka K, Kanayama N, Harada A, Nagasaki Y, Kataoka K. Lactose-conjugated polyion complex micelles incorporating plasmid DNA as a targetable gene vector system: their preparation and gene transfecting efficiency against cultured HepG2 cells. J. Control. Release, 95, 653-664 (2004).
22. Oba M, Fukushima S, Kanayama N, Aoyagi K, Nishiyama N, Koyama H, Kataoka K. Cyclic RGD peptide-conjugated polyplex micelles as a targetable gene delivery system directed to cells possessing av β3 and av β5 integrins. Bioconjug. Chem., 18, 1415-1423 (2007).
23. Oba M, Aoyagi K, Miyata K, Matsumoto Y, Itaka K, Nishiyama N, Yamasaki Y, Koyama H, Kataoka K. Polyplex micelles with cyclic RGD peptide ligands and disulfide cross-links directing to the enhanced transfection via controlled intracellular trafficking. Mol. Pharm., 5, 1080-1092 (2008).
24. Mickler FM, Vachutinsky Y, Oba M, Miyata K, Nishiyama N, Kataoka K, Bräuchle C, Ruthardt N. Effect of integrin targeting and PEG shielding on polyplex micelle internalization studied by livecell imaging. J. Control. Release, 156, 364-373 (2011).
25. Haubner R, Gratias R, Diefenbach B, Goodman SL, Jonczyk A, Kessler H. Structural and functional aspects of RGD-containing cyclic pentapeptides as highly potent and selective av β3 antagonists. J. Am. Chem. Soc., 118, 7461-7472 (1996).
26. Vachutinsky Y, Oba M, Miyata K, Hiki S, Kano MR, Nishiyama N, Koyama H, Miyazono K, Kataoka K. Antiangiogenic gene therapy of experimental pancreatic tumor by sFlt-1 plasmid DNA carried by RGD-modified crosslinked polyplex micelles. J. Control. Release, 149, 51-57 (2011).
27. Christie RJ, Matsumoto Y, Miyata K, Nomoto T, Fukushima S, Osada K, Halnaut J, Pittella F, Kim HJ, Nishiyama N, Kataoka K. Targeted polymeric micelles for siRNA treatment of experimental cancer by intravenous injection. ACS Nano, 6, 5174-5189 (2012).
28. Wagner E, Plank C, Zatloukal K, Cotten M, Birnstiel ML. Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: toward a synthetic virus-like gene-transfer vehicle. Proc. Natl. Acad. Sci. U.S.A., 89, 7934-7938 (1992).
29. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B, Behr JP. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. U.S.A., 92, 7297-7301 (1995).
30. Nakanishi M, Park JS, Jang WD, Oba M, Kataoka K. Study on the quantitative aminolysis reaction of poly(β-benzyl l-aspartate) (PBLA) as a platform polymer for functionality materials. React. Funct. Polym., 67, 1361-1372 (2007).
31. Miyata K, Oba M, Nakanishi M, Fukushima S, Yamasaki Y, Koyama H, Nishiyama N, Kataoka K. Polyplexes from poly(aspartamide) bearing 1, 2-diaminoethane side chains induce pH-selective, endosomal membrane destabilization with amplified transfection and negligible cytotoxicity. J. Am. Chem. Soc., 130, 16287-16294 (2008).
32. Uchida H, Miyata K, Oba M, Ishii T, Suma T, Itaka K, Nishiyama N, Kataoka K. Odd-even effect of repeating aminoethylene units in the side chain of N-substituted polyaspartamides on gene transfection profiles. J. Am. Chem. Soc., 133, 15524-15532 (2011).
33. Masago K, Itaka K, Nishiyama N, Chung UI, Kataoka K. Gene delivery with biocompatible cationic polymer: pharmacogenomic analysis on cell bioactivity. Biomaterials, 28, 5169-5175 (2007).
34. Itaka K, Ishii T, Hasegawa Y, Kataoka K. Biodegradable polyamino acid-based polycations as safe and effective gene carrier minimizing cumulative toxicity. Biomaterials, 31, 3707-3714 (2010).
35. Kanayama N, Fukushima S, Nishiyama N, Itaka K, Jang WD, Miyata K, Yamasaki Y, Chung UI, Kataoka K. A PEG-based biocompatible block catiomer with high buffering capacity for the construction of polyplex micelles showing efficient gene transfer toward primary cells. ChemMedChem, 1, 439-444 (2006).
36. Akagi D, Oba M, Koyama H, Nishiyama N, Fukushima S, Miyata T, Nagawa H, Kataoka K. Biocompatible micellar nanovectors achieve efficient gene transfer to vascular lesions without cytotoxicity and thrombus formation. Gene Ther., 14, 1029-1038 (2007).
37. Nomoto T, Matsumoto Y, Miyata K, Oba M, Fukushima S, Nishiyama N, Yamasoba T, Kataoka K. In situ quantitative monitoring of polyplexes and polyplex micelles in the blood circulation using intravital real-time confocal laser scanning microscopy. J. Control. Release, 151, 104-109 (2011).
38. Han M, Bae Y, Nishiyama N, Miyata K, Oba M, Kataoka K. Transfection study using multicellular tumor spheroids for screening non-viral polymeric gene vectors with low cytotoxicity and high transfection efficiencies. J. Control. Release, 121, 38-48 (2007).
39. Han M, Oba M, Nishiyama N, Kano MR, Kizaka-Kondoh S, Kataoka K. Enhanced percolation and gene expression in tumor hypoxia by PEGylated polyplex micelles. Mol. Ther., 17, 1404-1410 (2009).
40. Takae S, Miyata K, Oba M, Ishii T, Nishiyama N, Itaka K, Yamasaki Y, Koyama H, Kataoka K. PEG-detachable polyplex micelles based on disulfide-linked block catiomers as bioresponsive nonviral gene vectors. J. Am. Chem. Soc., 130, 6001-6009 (2008).
41. Kumagai M, Shimoda S, Wakabayashi R, Kunisawa Y, Ishii T, Osada K, Itaka K, Nishiyama N, Kataoka K, Nakano K. Effective transgene expression without toxicity by intraperitoneal administration of PEG-detachable polyplex micelles in mice with peritoneal dissemination. J. Control. Release, 160, 542-551 (2012).
42. Miyata K, Oba M, Kano MR, Fukushima S, Vachutinsky Y, Han M, Koyama H, Miyazono K, Nishiyama N, Kataoka K. Polyplex micelles from triblock copolymers composed of tandemly aligned segments with biocompatible, endosomal escaping, and DNA-condensing functions for systemic gene delivery to pancreatic tumor tissue. Pharm. Res., 25, 2924-2936 (2008).
43. Kano MR, Bae Y, Iwata C, Morishita Y, Yashiro M, Oka M, Fujii T, Komuro A, Kiyono K, Kaminishi M, Hirakawa K, Ouchi Y, Nishiyama N, Kataoka K, Miyazono K. Improvement of cancertargeting therapy, using nanocarriers for intractable solid tumors by inhibition of TGF-beta signaling. Proc. Natl. Acad. Sci. U.S.A., 104, 3460-3465 (2007).
44. Oba M, Miyata K, Osada K, Christie RJ, Sanjoh M, Li W, Fukushima S, Ishii T, Kano MR, Nishiyama N, Koyama H, Kataoka K. Polyplex micelles prepared from ?-cholesteryl PEG-polycation block copolymers for systemic gene delivery. Biomaterials, 32, 652-663 (2011).
45. Kagaya H, Oba M, Miura Y, Koyama H, Ishii T, Shimada T, Takato T, Kataoka K, Miyata T. Impact of polyplex micelles installed with cyclic RGD peptide as ligand on gene delivery to vascular lesions. Gene Ther., 19, 61-69 (2012).
46. Chen Q, Osada K, Ishii T, Oba M, Uchida S, Tockary TA, Endo T, Ge Z, Kinoh H, Kano MR, Itaka K, Kataoka K. Homo-catiomer integration into PEGylated polyplex micelle from block-catiomer for systemic anti-angiogenic gene therapy for fibrotic pancreatic tumors. Biomaterials, 33, 4722-4730 (2012).
47. Iriyama A, Oba M, Ishii T, Nishiyama N, Kataoka K, Tamaki Y, Yanagi Y. Gene transfer using micellar nanovectors inhibits choroidal neovascularization in vivo. PLoS ONE, 6, e28560 (2011).
48. Kim HJ, Oba M, Pittella F, Nomoto T, Cabral H, Matsumoto Y, Miyata K, Nishiyama N, Kataoka K. PEG-detachable cationic polyaspartamide derivatives bearing stearoyl moieties for systemic siRNA delivery toward subcutaneous BxPC3 pancreatic tumor. J. Drug Target., 20, 33-42 (2012).