Enhanced gene expression of systemically administered plasmid DNA in the liver with therapeutic ultrasound and microbubbles

IEEE Transactions on Ultrasonics, Ferroelectrics, and Frequency Control

Volumn 60, Issue 1, 2013, Pages 88-96

  Raju, Balasundar I ^a   Leyvi, Evgeniy ^a   Seip, Ralf ^a   Sethuraman, Shriram ^a   Luo, Xiaoyan ^b   Bird, Andrew ^b   Li, Songtao ^b   Koeberl, Dwight ^b  

^a PHILIPS RESEARCH LABORATORIES   (Netherlands)

^b DUKE UNIVERSITY MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CLINICAL PRACTICES; ENHANCED GENE EXPRESSION; GLUCOSE-6-PHOSPHATASE; IMAGE-GUIDED; KNOCKOUT MICE; LOW TOXICITY; MICE LIVER; MICROBUBBLES; MRNA EXPRESSION; NAKED PLASMID DNA; PILOT STUDIES; PLASMID DNA; PROTEIN EXPRESSIONS; REVERSE TRANSCRIPTION-POLYMERASE CHAIN REACTION; SYSTEMIC ADMINISTRATION; TAIL VEINS; THERAPEUTIC AGENTS; THERAPEUTIC ULTRASOUND; TISSUE ANALYSIS; ULTRASOUND TREATMENTS; WESTERN BLOTS; WESTERN-BLOT ANALYSIS; WILD TYPES;

BIOMOLECULES; GENE EXPRESSION; MAMMALS; POLYMERASE CHAIN REACTION; PROTEINS; RNA; ULTRASONICS;

DISEASES;

DNA; GLUCOSE 6 PHOSPHATASE;

ANIMAL; ARTICLE; CHEMISTRY; GENE THERAPY; GENETIC TRANSFECTION; GENETICS; LIVER; METABOLISM; METHODOLOGY; MICROBUBBLE; MOUSE; MOUSE MUTANT; PILOT STUDY; PLASMID; ULTRASOUND; WESTERN BLOTTING;

ANIMALS; BLOTTING, WESTERN; DNA; GENETIC THERAPY; GLUCOSE-6-PHOSPHATASE; LIVER; MICE; MICE, KNOCKOUT; MICROBUBBLES; PILOT PROJECTS; PLASMIDS; TRANSFECTION; ULTRASONICS;

MLCS; MLOWN;

EID: 84872036386     PISSN: 08853010     EISSN: None     Source Type: Journal    
DOI: 10.1109/TUFFC.2013.2540     Document Type: Article

References (31)

1. C. S. Manno, V. R. Arruda, G. F. Pierce, B. Glader, M. Ragni, J. Rasko, M. C. Ozelo, K. Hoots, P. Blatt, B. Konkle, M. Dake, R. Kaye, M. Razavi, A. Zajko, J. Zehnder, H. Nakai, A. Chew, D. Leonard, J. F. Wright, R. R. Lessard, J. M. Sommer, M. Tigges, D. Sabatino, A. Luk, H. Jiang, F. Mingozzi, L. Couto, H. C. Ertl, K. A. High, and M. A. Kay, "Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response," Nat. Med., vol. 12, no. 3, pp. 342-347, Mar. 2006. (Pubitemid 43355084)
2. S. Hernot and A. L. Klibanov, "Microbubbles in ultrasound-triggered drug and gene delivery," Adv. Drug Deliv. Rev., vol. 60, pp. 1153-1166, Jun. 30, 2008.
3. C .-Y. Lin, Y.-L. Huang, J.-R. Li, F.-H. Chang, and W.-L. Lin, "Effects of focused ultrasound and microbubbles on the vascular permeability of nanoparticles delivered into mouse tumors," Ultrasound Med. Biol., vol. 36, pp. 1460-1469, Sep. 2010.
4. S. Braun, "Muscular gene transfer using nonviral vectors," Curr. Gene Ther., vol. 8, no. 5, pp. 391-405, 2008.
5. K. W. Ferrara, "Driving delivery vehicles with ultrasound," Adv. Drug Deliv. Rev., vol. 60, no. 10, pp. 1097-1102, 2008.
6. V. Frenkel, "Ultrasound mediated delivery of drugs and genes to solid tumors," Adv. Drug Deliv. Rev., vol. 60, no. 10, pp. 1193-1208, 2008.
7. C. R. Mayer and R. Bekeredjian, "Ultrasonic gene and drug delivery to the cardiovascular system," Adv. Drug Deliv. Rev., vol. 60, no. 10, pp. 1177-1192, 2008.
8. C. M. H. Newman and T. Bettinger, "Gene therapy progress and prospects: Ultrasound for gene transfer," Gene Ther., vol. 14, no. 6, pp. 465-475, 2007. (Pubitemid 46390919)
9. A. I. Daud, R. C. DeConti, S. Andrews, P. Urbas, A. I. Riker, V. K. Sondak, P. N. Munster, D. M. Sullivan, K. E. Ugen, J. L. Messina, and R. Heller, "Phase I trial of interleukin-12 plasmid electroporation in patients with metastatic melanoma," J. Clin. Oncol., vol. 26, no. 36, pp. 5896-5903, 2008.
10. C. H. Miao, A. A. Brayman, K. R. Loeb, P. Q. Ye, L. Zhou, P. Mourad, and L. A. Crum, "Ultrasound enhances gene delivery of human factor IX plasmid," Hum. Gene Ther., vol. 16, pp. 893-905, Jul. 2005. (Pubitemid 40982337)
11. Z. P. Shen, A. A. Brayman, L. Chen, and C. H. Miao, "Ultrasound with microbubbles enhances gene expression of plasmid DNA in the liver via intraportal delivery," Gene Ther., vol. 15, no. 16, pp. 1147-1155, 2008.
12. S. Tsunoda, O. Mazda, Y. Oda, Y. Iida, S. Akabame, T. Kishida, S. Y. Masaharu, H. Asada, S. Gojo, J. Imanishi, H. Matsubara, and T. Yoshikawa, "Sonoporation using microbubble BR14 promotes pDNA/siRNA transduction to murine heart," Biochem. Biophys. Res. Commun., vol. 336, no. 1, pp. 118-127, 2005. (Pubitemid 41253411)
13. K. J. Lei, L. L. Shelly, C. J. Pan, J. B. Sidbury, and J. Y. Chou, "Mutations in the glucose-6-phosphatase gene that cause glycogenstorage-disease type-1a," Science, vol. 262, pp. 580-583, Oct. 22, 1993. (Pubitemid 23350674)
14. S. W. Moses, "Historical highlights and unsolved problems in glycogen storage disease type 1," Eur. J. Pediatr., vol. 161, pp. S2-S9, Oct. 2002.
15. D. D. Koeberl, C. Pinto, B. Sun, S. Li, D. M. Kozink, D. K. Benjamin Jr., A. K. Demaster, M. A. Kruse, V. Vaughn, S. Hillman, A. Bird, M. Jackson, T. Brown, P. S. Kishnani, and Y.-T. Chen, "AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia," Mol. Ther., vol. 16, pp. 665-672, Apr. 2008. (Pubitemid 351426167)
16. A. Zingone, H. Hiraiwa, C. J. Pan, B. C. Lin, H. W. Chen, J. M. Ward, and J. Y. Chou, "Correction of glycogen storage disease type 1a in a mouse model by gene therapy," J. Biol. Chem., vol. 275, pp. 828-832, Jan. 14, 2000. (Pubitemid 30051122)
17. R. Seip, B. I. Raju, E. Leyvi, C. T. Chin, L. Songtao, C. Rouse, D. Koeberl, and W. Fodor, "Enhanced gene transfection of plasmid DNA in the liver with ultrasound and microbubbles," in 2010 IEEE Ultrasonics Symp., pp. 103-106.
18. K. J. Lei, H. W. Chen, C. J. Pan, J. M. Ward, B. Mosinger, E. J. Lee, H. Westphal, B. C. Mansfield, and J. Y. Chou, "Glucose-6-phosphatase dependent substrate transport in the glycogen storage disease type-1a mouse," Nat. Genet., vol. 13, pp. 203-209, Jun. 1996. (Pubitemid 26000441)
19. X. Luo, G. Hall, S. Li, A. Bird, P. J. Lavin, M. P. Winn, A. R. Kemper, T. T. Brown, and D. D. Koeberl, "Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector," Mol. Ther., vol. 19, no. 11, pp. 1961-1970, Nov. 2011.
20. C. J. Pan, K. J. Lei, B. Annabi, W. Hemrika, and J. Y. Chou, "Transmembrane topology of glucose-6-phosphatase," J. Biol. Chem., vol. 273, pp. 6144-6148, Mar. 1998. (Pubitemid 28144695)
21. D. D. Koeberl, B. D. Sun, T. V. Damodaran, T. Brown, D. S. Millington, D. K. Benjamin Jr., A. Bird, A. Schneider, S. Hillman, M. Jackson, R. M. Beaty, and Y. T. Chen, "Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia," Gene Ther., vol. 13, no. 17, pp. 1281-1289, Sep. 2006. (Pubitemid 44264082)
22. R. Seip, C. T. Chin, C. S. Hall, B. I. Raju, A. Ghanem, and K. Tiemann, "Targeted ultrasound-mediated delivery of nanoparticles: On the development of a new HIFU-based therapy and imaging device," IEEE Trans. Biomed. Eng., vol. 57, pp. 61-70, Jan. 2010.
23. A. Amalfitano, A. J. McVie-Wylie, H. Hu, T. L. Dawson, N. Raben, P. Plotz, and Y. T. Chen, "Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase," Proc. Natl. Acad. Sci. USA, vol. 96, pp. 8861-8866, Aug. 3, 1999. (Pubitemid 29374673)
24. K. M. Podetz-Pedersen, J. B. Bell, T. W. J. Steele, A. Wilber, W. T. Shier, L. R. Belur, R. S. McIvor, and P. B. Hackett, "Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons," Hum. Gene Ther., vol. 21, no. 2, pp. 210-220, 2010.
25. J. Alter, C. A. Sennoga, D. M. Lopes, R. J. Eckersley, and D. J. Wells, "Microbubble stability is a major determinant of the efficiency of ultrasound and microbubble mediated in vivo gene transfer," Ultrasound Med. Biol., vol. 35, pp. 976-984, Jun. 2009.
26. M. Iwasaki, Y. Adachi, T. Nishiue, K. Minamino, Y. Suzuki, Y. M. Zhang, K. J. Nakano, Y. Koike, J. F. Wang, H. Mukaide, S. Taketani, F. Yuasa, H. Tsubouchi, E. Gohda, T. Iwasaka, and S. Ikehara, "Hepatocyte growth factor delivered by ultrasound-mediated destruction of microbubbles induces proliferation of cardiomyocytes and amelioration of left ventricular contractile function in doxorubicin-induced cardiomyopathy," Stem Cells, vol. 23, no. 10, pp. 1589-1597, 2005. (Pubitemid 41741008)
27. X. S. Li, Z. G. Wang, H. T. Ran, X. L. Li, Q. Y. Yuan, Y. Y. Zheng, J. L. Ren, L. Su, W. F. Zhang, Q. Li, and C. S. Xu, "Experimental research on therapeutic angiogenesis induced by hepatocyte growth factor directed by ultrasound-targeted microbubble destruction in rats," J. Ultrasound Med., vol. 27, no. 3, pp. 453-460, 2008. (Pubitemid 351428128)
28. D. Mukherjee, J. Wong, B. Griffin, S. G. Ellis, T. Porter, S. Sen, and J. D. Thomas, "Ten-fold augmentation of endothelial uptake of vascular endothelial growth factor with ultrasound after systemic administration," J. Am. Coll. Cardiol., vol. 35, no. 6, pp. 1678-1686, 2000. (Pubitemid 30307516)
29. G. Danialou, A. S. Comtois, R. W. R. Dudley, J. Nalbantoglu, R. Gilbert, G. Karpati, D. H. Jones, and B. J. Petrof, "Ultrasound increases plasmid-mediated gene transfer to dystrophic muscles without collateral damage," Mol. Ther., vol. 6, no. 5, pp. 687-693, 2002. (Pubitemid 35363702)
30. A. Delalande, M. F. Bureau, P. Midoux, A. Bouakaz, and C. Pichon, "Ultrasound-assisted microbubbles gene transfer in tendons for gene therapy," Ultrasonics, vol. 50, pp. 269-272, Feb. 2010.
31. Z. Y. Chen, M. X. Xie, X. F. Wang, Q. Lv, and S. W. Ding, "Efficient gene delivery to myocardium with ultrasound targeted microbubble destruction and polyethylenimine," J. Huazhong Univ. Sci. Technolog. Med. Sci., vol. 28, no. 5, pp. 613-617, 2008.