Design of PCR-amplified DNA fragments for in vivo gene delivery: Size-dependency on stability and transgene expression

Journal of Pharmaceutical Sciences

Volumn 96, Issue 9, 2007, Pages 2251-2261

  Hirata, Kazuhiro ^a   Nishikawa, Makiya ^a   Kobayashi, Naoki ^a   Takahashi, Yuki ^a   Takakura, Yoshinobu ^a  

^a KYOTO UNIVERSITY   (Japan)

Author keywords

Gene transfer; Hydrodynamics based procedure; PCR amplified fragment; Plasmid DNA

Indexed keywords

DNA FRAGMENT; FIREFLY LUCIFERASE; GREEN FLUORESCENT PROTEIN; PLASMID DNA;

AMPLIFIED FRAGMENT LENGTH POLYMORPHISM; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL CULTURE; CONTROLLED STUDY; CPG ISLAND; DRUG STABILITY; GENE EXPRESSION; HUMAN; HUMAN CELL; HYDRODYNAMICS BASED TRANSFECTION; IN VIVO GENE TRANSFER; MELANOMA B16; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; POLYMERASE CHAIN REACTION; TRANSGENE;

EID: 34748906174     PISSN: 00223549     EISSN: 15206017     Source Type: Journal    
DOI: 10.1002/jps.20879     Document Type: Article

References (42)

1. Nishikawa M, Huang L. 2001. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther 12:861-870.
2. Liu F, Song Y, Liu D. 1999. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 6:1258-1266.
3. Zhang G, Budker V, Wolff JA. 1999. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum Gene Ther 10:1735-1737.
4. Kobayashi N, Nishikawa M, Takakura Y. 2005. The hydrodynamics-based procedure for controlling the pharmacokinetics of gene medicines at whole body, organ and cellular levels. Adv Drug Deliv Rev 57: 713-731.
5. Budker V, Zhang G, Knechtle S, Wolff JA. 1996. Naked DNA delivered intraportally expresses efficiently in hepatocytes. Gene Ther 3:593-598.
6. Maruyama H, Higuchi N, Nishikawa Y, Hirahara H, Iino N, Kameda S, Kawachi H, Yaoita E, Gejyo F, Miyazaki J. 2002. Kidney-targeted naked DNA transfer by retrograde renal vein injection in rats. Hum Gene Ther 13:455-468.
7. Liang KW, Nishikawa M, Liu F, Sun B, Ye Q, Huang L. 2004. Restoration of dystrophin expression in mdx mice by intravascular injection of naked DNA containing full-length dystrophin cDNA. Gene Ther 11:901-908.
8. Liu F, Huang L. 2002. Electric gene transfer to the liver following systemic administration of plasmid DNA. Gene Ther 9:1116-1119.
9. Sakai M, Nishikawa M, Thanaketpaisarn O, Yamashita F, Hashida M. 2005. Hepatocyte-targeted gene transfer by combination of vascularly delivered plasmid DNA and in vivo electroporation. Gene Ther 12:607-616.
10. Thanaketpaisarn O, Nishikawa M, Yamashita F, Hashida M. 2005. Tissue-specific characteristics of in vivo electric gene transfer by tissue and intravenous injection of plasmid DNA. Pharm Res 22:883-891.
11. Somiari S, Glasspool-Malone J, Drabick JJ, Gilbert RA, Heller R, Jaroszeski MJ, Malone RW. 2000. Theory and in vivo application of electroporative gene delivery. Mol Ther 2:178-187.
12. Kobayashi N, Nishikawa M, Hirata K, Takakura Y. 2004. Hydrodynamics-based procedure involves transient hyperpermeability in the hepatic cellular membrane: Implication of a nonspecific process in efficient intracellular gene delivery. J Gene Med 6: 584-592.
13. Zhang G, Gao X, Song YK, Vollmer R, Stolz DB, Gasiorowski JZ, Dean DA, Liu D. 2004. Hydroporation as the mechanism of hydrodynamic delivery. Gene Ther 11:675-682.
14. Bird AP. 1985. CpG rich islands and the function of DNA methylation. Nature 321:209-213.
15. Krieg AM2002. CpG motifs in bacterial DNA and their immune effects. Annu Rev Immunol 20:709-760.
16. Tan Y, Li S, Pitt BR, Huang L. 1999. The inhibitory role of CpG immunostimulatory motifs in cationic lipid vector-mediated transgene expression in vivo. Hum Gene Ther 10:2153-2161.
17. Boyes J, Bird A. 1992. Repression of genes by DNA methylation depends on CpG density and promoter strength: Evidence for involvement of a methyl-CpG binding protein. EMBO J 11:327-333.
18. Yew NS, Zhao H, Przybylska M, Wu IH, Tousignant JD, Scheule RK, Cheng SH. 2002. CpG-depleted plasmid DNA vectors with enhanced safety and long-term gene expression in vivo. Mol Ther 5:731-738.
19. Tolmachov O, Palaszewski I, Bigger B, Coutelle C. 2006. RecET driven chromosomal gene targeting to generate a RecA deficient Escherichia coli strain for Cre mediated production of minicircle DNA. BMC Biotechnol 6:17.
20. Chen ZY, He CY, Kay MA. 2005. Improved production and purification of minicircle DNA vector free of plasmid bacterial sequences and capable of persistent transgene expression in vivo. Hum Gene Ther 16:126-131.
21. Chen ZY, He CY, Ehrhardt A, Kay MA. 2003. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 8:495-500.
22. Weber M, Moller K, Welzeck M, Schorr J. 1995. Effects of lipopolysaccharide on transfection efficiency in eukaryotic cells. Biotechniques 19:930-940.
23. Li S, Brisson M, He Y, Huang L. 1997. Delivery of a PCR amplified DNA fragment into cells: A model for using synthetic genes for gene therapy. Gene Ther 4:449-454.
24. Hofman CR, Dileo JP, Li Z, Li S, Huang L. 2001. Efficient in vivo gene transfer by PCR amplified fragment with reduced inflammatory activity. Gene Ther 8:71-74.
25. Lukacs GL, Haggie P, Seksek O, Lechardeur D, Freedman N, Verkman AS. 2000. Size-dependent DNA mobility in cytoplasm and nucleus. J Biol Chem 275:1625-1629.
26. Dauty E, Verkman AS. 2005. Actin cytoskeleton as the principal determinant of size-dependent DNA mobility in cytoplasm: A new barrier for non-viral gene delivery. J Biol Chem 280:7823-7828.
27. Nomura T, Yasuda K, Yamada T, Okamoto S, Mahato RI, Watanabe Y, Takakura Y, Hashida M. 1999. Gene expression and antitumor effects following direct interferon (IFN)-gamma gene transfer with naked plasmid DNA and DC-chol liposome complexes in mice. Gene Ther 6:121-129.
28. Poste G, Doll J, Hart IR, Fidler IJ. 1980. In vitro selection of murine B16 melanoma variants with enhanced tissue-invasive properties. Cancer Res 40:1636-1644.
29. Kobayashi N, Matsui Y, Kawase A, Hirata K, Miyagishi M, Taira K, Nishikawa M, Takakura Y. 2004. Vector-based in vivo RNA interference: Dose-and time-dependent suppression of transgene expression. J Pharmacol Exp Ther 308:688-693.
30. Nishikawa M, Takakura Y, Hashida M. 2005. Theoretical considerations involving the pharmacokinetics of plasmid DNA. Adv Drug Deliv Rev 57:675-688.
31. Qin L, Ding Y, Pahud DR, Chang E, Imperiale MJ, Bromberg JS. 1997. Promoter attenuation in gene therapy: Interferon-gamma and tumor necrosis factor-alpha inhibit transgene expression. Hum Gene Ther 8:2019-2029.
32. Hagstrom JE, Ludtke JJ, Bassik MC, Sebestyen MG, Adam SA, Wolff JA. 1997. Nuclear import of DNA in digitonin-permeabilized cells. J Cell Sci 110:2323-2331.
33. Dowty ME, Williams P, Zhang G, Hagstrom JE, Wolff JA. 1995. Plasmid DNA entry into postmitotic nuclei of primary rat myotubes. Proc Natl Acad Sci USA 92:4572-4576.
34. Dean DA. 1997. Import of plasmid DNA into the nucleus is sequence specific. Exp Cell Res 230:293-302.
35. Dean DA, Dean BS, Muller S, Smith LC. 1999. Sequence requirements for plasmid nuclear entry. Exp Cell Res 253:713-722.
36. Ehrhardt A, Xu H, Huang Z, Engler JA, Kay MA. 2005. A direct comparison of two nonviral gene therapy vectors for somatic integration: In vivo evaluation of the bacteriophage integrase C31 and the Sleeping Beauty transposase. Mol Ther 11: 695-706.
37. Guo ZS, Wang LH, Eisensmith RC, Woo SL. 1996. Evaluation of promoter strength for hepatic gene expression in vivo following adenovirus-mediated gene transfer. Gene Ther 3:802-810.
38. Loser P, Jennings GS, Strauss M, Sandig V. 1998. Reactivation of the previously silenced cytomegalovirus major immediate-early promoter in the mouse liver: Involvement of NFκB. J Virol 72:180-190.
39. Yew NS, Cheng SH. 2004. Reducing the immunostimulatory activity of CpG-containing plasmid DNA vectors for non-viral gene therapy. Expert Opin Drug Deliv 1:115-125.
40. Nakai H, Montini E, Fuess S, Storm TA, Meuse L, Finegold M, Grompe M, Kay MA. 2003. Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice. Mol Ther 7:101-111.
41. Chen ZY, Yant SR, He CY, Meuse L, Shen S, Kay MA. 2001. Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver. Mol Ther 3:403-410.
42. Kameda S, Maruyama H, Higuchi N, Nakamura G, lino N, Nishikawa Y, Miyazaki J, Gejyo F. 2003. Hydrodynamics-based transfer of PCR-amplified DNA fragments into rat liver. Biochem Biophys Res Commun 309:929-936.