Dose response in rodents and nonhuman primates after hydrodynamic limb vein delivery of naked plasmid DNA

Human Gene Therapy

Volumn 22, Issue 7, 2011, Pages 889-903

  Wooddell, Christine I ^a   Hegge, Julia O ^a   Zhang, Guofeng ^b   Sebestyén, Magdolna G ^a   Noble, Mark ^a   Griffin, Jacob B ^a   Pfannes, Loretta V ^a   Herweijer, Hans ^a   Hagstrom, James E ^a   Braun, Serge ^c   Huss, Thierry ^d   Wolff, Jon A ^b  

^a Roche Madison   (United States)

^b UNIVERSITY OF WISCONSIN MADISON   (United States)

^c Association Francaise Contre les Myopathies   (France)

^d TRANSGENE S A   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ALKALINE PHOSPHATASE PLACENTA ISOENZYME; BETA GALACTOSIDASE; CREATINE KINASE; DESMIN; LUCIFERASE; NAKED DNA; PLASMID DNA;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; CYTOMEGALOVIRUS; DOSE RESPONSE; DRUG DOSE ESCALATION; DRUG EFFICACY; FEMALE; GASTROCNEMIUS MUSCLE; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENETIC TRANSFECTION; HYDRODYNAMICS BASED TRANSFECTION; KINETICS; LEG MUSCLE; MALE; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIMATE; PROMOTER REGION; RAT; REPORTER GENE; RHESUS MONKEY; RODENT; SKELETAL MUSCLE; SOLEUS MUSCLE; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; BETA-GALACTOSIDASE; CREATINE KINASE, MM FORM; CYTOMEGALOVIRUS; DNA; EXTREMITIES; FEMALE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC VECTORS; HYDRODYNAMICS; KINETICS; LAC OPERON; LUCIFERASES; MACACA MULATTA; MALE; MICE; MICE, INBRED C57BL; MICE, INBRED ICR; MUSCLE, SKELETAL; PLASMIDS; PROMOTER REGIONS, GENETIC; RATS; RATS, SPRAGUE-DAWLEY; TRANSFECTION; VEINS;

CYTOMEGALOVIRUS; MACACA MULATTA; MUS; PRIMATES; RATTUS; RODENTIA;

EID: 79960374215     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2010.160     Document Type: Article

References (35)

1. Baskar, J.F., Smith, P.P., Nilaver, G., et al. (1996). The enhancer domain of the human cytomegalovirus major immediate-early promoter determines cell type-specific expression in trans-genic mice. J. Virol. 70, 3207-3214. (Pubitemid 26121939)
2. Braun, S. (2008). Muscular gene transfer using nonviral vectors. Curr. Gene Ther. 8, 391-405.
3. Budker, V., Zhang, G., Danko, I., et al. (1998). The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther. 5, 272-276. (Pubitemid 28077756)
4. Danialou, G., Comtois, A.S., Matecki, S., et al. (2005). Optimization of regional intraarterial naked DNA-mediated trans-gene delivery to skeletal muscles in a large animal model. Mol. Ther. 11, 257-266.
5. Danko, I., Fritz, J.D., Latendresse, J.S., et al. (1993). Dystrophin expression improves myofiber survival in mdx muscle following intramuscular plasmid DNA injection. Hum. Mol. Genet. 2, 2055-2061. (Pubitemid 24003398)
6. Danko, I., Williams, P., Herweijer, H., et al. (1997). High expression of naked plasmid DNA in muscles of young rodents. Hum. Mol. Genet. 6, 1435-1443. (Pubitemid 27397018)
7. Duan, D. (2008). Myodys, a full-length dystrophin plasmid vector for Duchenne and Becker muscular dystrophy gene therapy. Curr. Opin. Mol. Ther. 10, 86-94. (Pubitemid 351170599)
8. Gillis, J.M. (1999). Understanding dystrophinopathies: An inventory of the structural and functional consequences of the absence of dystrophin in muscles of the mdx mouse. J. Muscle Res. Cell Motil. 20, 605-625. (Pubitemid 30068167)
9. Guasconi, V., and Puri, P.L. (2009). Chromatin: The interface between extrinsic cues and the epigenetic regulation of muscle regeneration. Trends Cell Biol. 19, 286-294.
10. Hagstrom, J.E., Hegge, J., Zhang, G., et al. (2004). A facile non-viral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Mol. Ther. 10, 386-398. (Pubitemid 39117883)
11. Hegge, J.O., Wooddell, C.I., Zhang, G., et al. (2010). Evaluation of hydrodynamic limb vein injections in non-human primates. Hum. Gene Ther. 21, 1-14.
12. Herweijer, H., Zhang, G., Subbotin, V.M., et al. (2001). Time course of gene expression after plasmid DNA gene transfer to the liver. J. Gene Med. 3, 280-291. (Pubitemid 33684776)
13. Jiao, S., Williams, P., Berg, R.K., et al. (1992). Direct gene transfer into nonhuman primate myofibers in vivo. Hum. Gene Ther. 3, 21-33.
14. Kamimura, K., Zhang, G., and Liu, D. (2010). Image-guided, intravascular hydrodynamic gene delivery to skeletal muscle in pigs. Mol. Ther. 18, 93-100.
15. Le, T.P., Coonan, K.M., Hedstrom, R.C., et al. (2000). Safety, tolerability and humoral immune responses after intramuscular administration of a malaria DNA vaccine to healthy adult volunteers. Vaccine 18, 1893-1901. (Pubitemid 30122852)
16. Li, B., Carey, M., and Workman, J.L. (2007). The role of chro-matin during transcription. Cell 128, 707-719.
17. Liu, F., Song, Y., and Liu, D. (1999). Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. 6, 1258-1266. (Pubitemid 29359019)
18. Mendell, J.R., Campbell, K., Rodino-Klapac, L., et al. (2010). Dystrophin immunity in Duchenne's muscular dystrophy. N. Engl. J. Med. 363, 1429-1437.
19. Miao, C.H., Ohashi, K., Patijn, G.A., et al. (2000). Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol. Ther. 1, 522-532.
20. Miyoshi, K., Kawai, H., Iwasa, M., et al. (1986). Autosomal recessive distal muscular dystrophy as a new type of progressive muscular dystrophy: Seventeen cases in eight families including an autopsied case. Brain 109, 31-54. (Pubitemid 16125183)
21. Okayama, H., and Berg, P. (1983). A cDNA cloning vector that permits expression of cDNA inserts in mammalian cells. Mol. Cell. Biol. 3, 280-289. (Pubitemid 13122390)
22. Riu, E., Chen, Z.Y., Xu, H., et al. (2007). Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo. Mol. Ther. 15, 1348-1355. (Pubitemid 46965331)
23. Romero, N.B., Braun, S., Benveniste, O., et al. (2004). Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy. Hum. Gene Ther. 15, 1065-1076. (Pubitemid 39552288)
24. Satkauskas, S., Bureau, M.F., Mahfoudi, A., and Mir, L.M. (2001). Slow accumulation of plasmid in muscle cells: Supporting evidence for a mechanism of DNA uptake by receptor-mediated endocytosis. Mol. Ther. 4, 317-323. (Pubitemid 33040516)
25. Sebestyen, M.G., Hegge, J.O., Noble, M.A., et al. (2007). Progress toward a nonviral gene therapy protocol for the treatment of anemia. Hum. Gene Ther. 18, 269-285. (Pubitemid 46626465)
26. Toumi, H., Hegge, J., Subbotin, V., et al. (2006). Rapid intravas-cular injection into limb skeletal muscle: A damage assessment study. Mol. Ther. 13, 229-236. (Pubitemid 41763502)
27. Vigen, K.K., Hegge, J.O., Zhang, G., et al. (2007). Magnetic resonance imaging-monitored plasmid DNA delivery in primate limb muscle. Hum. Gene Ther. 18, 257-268. (Pubitemid 46626464)
28. Wolff, J.A., Ludtke, J.J., Acsadi, G., et al. (1992). Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. 1, 363-369.
29. Wooddell, C.I., Reppen, T., Wolff, J.A., and Herweijer, H. (2008). Sustained liver-specific transgene expression from the albumin promoter in mice following hydrodynamic plasmid DNA delivery. J. Gene Med. 10, 551-563. (Pubitemid 351753524)
30. Wooddell, C.I., Subbotin, V.M., Sebestyén, M.G., et al. (2011). Muscle damage following delivery of naked plasmid DNA into skeletal muscles is batch-dependent. Hum. Gene Ther. 22, 225-235.
31. Workman, J.L. (2006). Nucleosome displacement in transcription. Genes Dev. 20, 2009-2017. (Pubitemid 44157452)
32. Zhang, G., Budker, V., and Wolff, J.A. (1999). High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum. Gene Ther. 10, 1735-1737. (Pubitemid 29328217)
33. Zhang, G., Budker, V., Williams, P., et al. (2001). Efficient expression of naked DNA delivered intraarterially to limb muscles of nonhuman primates. Hum. Gene Ther. 12, 427-438. (Pubitemid 32186654)
34. Zhang, G., Ludtke, J.J., Thioudellet, C., et al. (2004). Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of Duchenne muscular dystrophy. Hum. Gene Ther. 15, 770-782. (Pubitemid 39095249)
35. Zhang, G., Wooddell, C.I., Hegge, J.O., et al. (2010). Functional efficacy of dystrophin expression from plasmids delivered to mdx mice by hydrodynamic limb vein injection. Hum. Gene Ther. 21, 221-237.