Evaluation of long-term gene expression in mouse liver using PhiC31 integrase and hydrodynamic injection

Biological and Pharmaceutical Bulletin

Volumn 35, Issue 7, 2012, Pages 1182-1186

  Umemoto, Yoshiaki ^a   Kawakami, Shigeru ^a   Otani, Yuki ^a   Higuchi, Yuriko ^a   Yamashita, Fumiyoshi ^a   Hashida, Mitsuru ^a,b  

^a KYOTO UNIVERSITY   (Japan)

^b KYOTO UNIVERSITY   (Japan)

Author keywords

Gene vector; Hepatocyte; Non viral gene delivery; Plasmid DNA

Indexed keywords

AMINOTRANSFERASE; COMPLEMENTARY DNA; GENOMIC DNA; INTEGRASE; LUCIFERASE; PHIC31 INTEGRASE; PLASMID DNA; UNCLASSIFIED DRUG;

AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DNA DETERMINATION; DNA TRANSFECTION; ENZYME ACTIVITY; ENZYME ASSAY; ENZYME RELEASE; EVALUATION RESEARCH; FEMALE; GENE EXPRESSION PROFILING; GENE EXPRESSION REGULATION; LIVER INJURY; LIVER PARENCHYMA; MOUSE; NONHUMAN; PROTEIN EXPRESSION; QUANTITATIVE ANALYSIS;

ANIMALS; DNA; FEMALE; GENE EXPRESSION; INTEGRASES; LIVER; LUCIFERASES; MICE; MICE, INBRED C57BL; MICE, INBRED ICR; TRANSFECTION;

EID: 84863550119     PISSN: 09186158     EISSN: 13475215     Source Type: Journal    
DOI: 10.1248/bpb.b12-00083     Document Type: Article

References (29)

1. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, Romana S, Radford-Weiss I, Gross F, Valensi F, Delabesse E, Macintyre E, Sigaux F, Soulier J, Leiva LE, Wissler M, Prinz C, Rabbitts TH, Le Deist F, Fischer A, Cavazzana-Calvo M. LMO2- associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science, 302, 415-419 (2003).
2. Li Z, Düllmann J, Schiedlmeier B, Schmidt M, von Kalle C, Meyer J, Forster M, Stocking C, Wahlers A, Frank O, Ostertag W, Kühlcke K, Eckert HG, Fehse B, Baum C. Murine leukemia induced by retroviral gene marking. Science, 296, 497 (2002).
3. Nienhuis AW, Dunbar CE, Sorrentino BP. Genotoxicity of retroviral integration in hematopoietic cells. Mol. Ther., 13, 1031-1049 (2006).
4. Nakanishi H, Higuchi Y, Kawakami S, Yamashita F, Hashida M. piggyBac transposon-mediated long-term gene expression in mice. Mol. Ther., 18, 707-714 (2010).
5. Rodríguez CI, Buchholz F, Galloway J, Sequerra R, Kasper J, Ayala R, Stewart AF, Dymecki SM. High-efficiency deleter mice show that FLPe is an alternative to Cre-loxP. Nat. Genet., 25, 139-140 (2000).
6. Wu S, Ying G, Wu Q, Capecchi MR. Toward simpler and faster genome-wide mutagenesis in mice. Nat. Genet., 39, 922-930 (2007).
7. Groth AC, Olivares EC, Thyagarajan B, Calos MP. A phage integrase directs efficient site-specific integration in human cells. Proc. Natl. Acad. Sci. U.S.A., 97, 5995-6000 (2000).
8. Thorpe HM, Smith MC. In vitro site-specific integration of bacteriophage DNA catalyzed by a recombinase of the resolvase/invertase family. Proc. Natl. Acad. Sci. U.S.A., 95, 5505-5510 (1998).
9. Thyagarajan B, Olivares EC, Hollis RP, Ginsburg DS, Calos MP. Site-specific genomic integration in mammalian cells mediated by phage phiC31 integrase. Mol. Cell. Biol., 21, 3926-3934 (2001).
10. Chalberg TW, Portlock JL, Olivares EC, Thyagarajan B, Kirby PJ, Hillman RT, Hoelters J, Calos MP. Integration specificity of phage phiC31 integrase in the human genome. J. Mol. Biol., 357, 28-48 (2006).
11. Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP. Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat. Biotechnol., 20, 1124-1128 (2002).
12. Aneja MK, Imker R, Rudolph C. Phage phiC31 integrase-mediated genomic integration and long-term gene expression in the lung after nonviral gene delivery. J. Gene Med., 9, 967-975 (2007).
13. Bertoni C, Jarrahian S, Wheeler TM, Li Y, Olivares EC, Calos MP, Rando TA. Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration. Proc. Natl. Acad. Sci. U.S.A., 103, 419-424 (2006).
14. Maucksch C, Aneja MK, Hennen E, Bohla A, Hoffmann F, Elfinger M, Rosenecker J, Rudolph C. Cell type differences in activity of the Streptomyces bacteriophage phiC31 integrase. Nucleic Acids Res., 36, 5462-5471 (2008).
15. Ehrhardt A, Xu H, Huang Z, Engler JA, Kay MA. A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase. Mol. Ther., 11, 695-706 (2005).
16. Keravala A, Lee S, Thyagarajan B, Olivares EC, Gabrovsky VE, Woodard LE, Calos MP. Mutational derivatives of phiC31 integrase with increased efficiency and specificity. Mol. Ther., 17, 112-120 (2009).
17. Held PK, Olivares EC, Aguilar CP, Finegold M, Calos MP, Grompe M. In vivo correction of murine hereditary tyrosinemia type I by phiC31 integrase-mediated gene delivery. Mol. Ther., 11, 399-408 (2005).
18. Lee S, Hong SW, Choi HS, Lee LY, Nam C, Rhee Y, Chung UI, Lim SK. Experimental parathyroid hormone gene therapy using phiC31 integrase. Endocr. J., 55, 1033-1041 (2008).
19. Woodard LE, Hillman RT, Keravala A, Lee S, Calos MP. Effect of nuclear localization and hydrodynamic delivery-induced cell division on phiC31 integrase activity. Gene Ther., 17, 217-226 (2010).
20. Fu Q, Jia S, Sun Z, Tian F, Du J, Zhou Y, Wang Y, Wang X, Zhan L. PhiC31 integrase and liver-specific regulatory elements confer high-level, long-term expression of firefly luciferase in mouse liver. Biotechnol. Lett., 31, 1151-1157 (2009).
21. Woodard LE, Keravala A, Jung WE, Wapinski OL, Yang Q, Felsher DW, Calos MP. Impact of hydrodynamic injection and phiC31 integrase on tumor latency in a mouse model of MYC-induced hepatocellular carcinoma. PLoS ONE, 5, e11367 (2010).
22. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther., 6, 1258-1266 (1999).
23. Wurdinger T, Badr C, Pike L, de Kleine R, Weissleder R, Breakefield XO, Tannous BA. A secreted luciferase for ex vivo monitoring of in vivo processes. Nat. Methods, 5, 171-173 (2008).
24. Feng DM, He CX, Miao CY, Lu B, Wu WJ, Ding YF, Xue JL. Conditions affecting hydrodynamics-based gene delivery into mouse liver in vivo. Clin. Exp. Pharmacol. Physiol., 31, 850-855 (2004).
25. Nishikawa M, Nakayama A, Takahashi Y, Fukuhara Y, Takakura Y. Reactivation of silenced transgene expression in mouse liver by rapid, large-volume injection of isotonic solution. Hum. Gene Ther., 19, 1009-1020 (2008).
26. Wang BY, Xu GL, Zhou CH, Tian L, Xue JL, Chen JZ, Jia W. PhiC31 integrase interacts with TTRAP and inhibits NFkappaB activation. Mol. Biol. Rep., 37, 2809-2816 (2010).
27. Kobayashi N, Nishikawa M, Hirata K, Takakura Y. 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 (2004).
28. Miao CH, Thompson AR, Loeb K, Ye X. Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo. Mol. Ther., 3, 947-957 (2001).
29. Chavez CL, Keravala A, Woodard LE, Hillman RT, Stowe TR, Chu JN, Calos MP. Kinetics and longevity of phiC31 integrase in mouse liver and cultured cells. Hum. Gene Ther., 21, 1287-1297 (2010).