Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates

Molecular Therapy

Volumn 17, Issue 2, 2009, Pages 327-333

  Brunetti Pierri, Nicola ^a   Stapleton, Gary E ^a,c   Law, Mark ^a,d   Breinholt, John ^a,e   Palmer, Donna J ^a   Zuo, Yu ^a   Grove, Nathan C ^a   Finegold, Milton J ^a   Rice, Karen ^b   Beaudet, Arthur L ^a   Mullins, Charles E ^a   Ng, Philip ^a  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

^b SOUTHWEST NATIONAL PRIMATE RESEARCH CENTER   (United States)

^c JOHNS HOPKINS ALL CHILDREN S HOSPITAL   (United States)

^d UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^e INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ALANINE AMINOTRANSFERASE; ALPHA FETOPROTEIN; ASPARTATE AMINOTRANSFERASE; INTERLEUKIN 6;

ANIMAL TISSUE; ARTICLE; BALLOON OCCLUSION; CONTROLLED STUDY; GENE DOSAGE; GENE EXPRESSION; HISTOPATHOLOGY; LIVER TOXICITY; MALE; MINIMALLY INVASIVE PROCEDURE; NONHUMAN; TARGET ORGAN; TOXICITY TESTING; TRANSGENE; VEIN OCCLUSION; VIRAL GENE DELIVERY SYSTEM; ANIMAL; CATHETERIZATION; CYTOLOGY; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; LIVER; METABOLISM; METHODOLOGY; PAPIO;

PRIMATES;

ANIMALS; CATHETERIZATION; GENE THERAPY; GENETIC VECTORS; LIVER; MALE; PAPIO; TRANSDUCTION, GENETIC; TRANSGENES;

EID: 63649138339     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2008.257     Document Type: Article

References (25)

1. Brunetti-Pierri, N and Ng, P (2006). Progress towards the clinical application of helper-dependent adenoviral vectors for liver and lung gene therapy. Curr Opin Mol Ther 8: 446-454.
2. Kim, IH, Jozkowicz, A, Piedra, PA, Oka, K and Chan, L (2001). Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci USA 98 13282-13287.
3. Toietta, G, Mane, VP, Norona, WS, Finegold, MJ, Ng, P, McDonagh, AF et al. (2005). Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci USA 102: 3930-3935.
4. Brunetti-Pierri, N, Nichols, TC, McCorquodale, S, Merricks, E, Palmer, DJ, Beaudet, AL et al. (2005). Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector. Hum Gene Ther 16: 811-820.
5. Brunetti-Pierri, N, Ng, T, Iannitti, DA, Palmer, DJ, Beaudet, AL, Finegold, MJ et al. (2006). Improved hepatic transduction, reduced systemic vector dissemination, and long-term transgene expression by delivering helper-dependent adenoviral vectors into the surgically isolated liver of nonhuman primates. Hum Gene Ther 17: 391-404.
6. Morral, N, O'Neal, W, Rice, K, Leland, M, Kaplan, J, Piedra, PA et al. (1999). Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc Natl Acad Sci USA 96: 12816-12821.
7. Brunetti-Pierri, N, Stapleton, GE, Palmer, DJ, Zuo, Y, Mane, VP, Finegold, MJ et al. (2007). Pseudo-hydrodynamic delivery of helper-dependent adenoviral vectors into non-human primates for liver-directed gene therapy. Mol Ther 15: 732-740.
8. Tao, N, Gao, GP, Parr, M, Johnston, J, Baradet, T, Wilson, JM et al. (2001). Sequestration of adenoviral vector by Kupffer cells leads to a nonlinear dose response of transduction in liver. Mol Ther 3 28-35.
9. Schiedner, G, Hertel, S, Johnston, M, Dries, V, van Rooijen, N, Kochanek, S (2003). Selective depletion or blockade of Kupffer cells leads to enhanced and prolonged hepatic transgene expression using high-capacity adenoviral vectors. Mol Ther 7: 35-43.
10. Sullivan, DE, Dash, S, Du, H, Hiramatsu, N, Aydin, F, Kolls, J et al. (1997). Liver-directed gene transfer in non-human primates. Hum Gene Ther 8: 1195-1206.
11. Nunes, FA, Furth, EE, Wilson, JM and Raper, SE (1999). Gene transfer into the liver of nonhuman primates with E1-deleted recombinant adenoviral vectors: Safety of readministration. Hum Gene Ther 10: 2515-2526.
12. Morral, N, O'Neal, WK, Rice, K, Leland, MM, Piedra, PA, Aguilar-Cordova, E et al. (2002). Lethal toxicity, severe endothelial injury, and a threshold effect with high doses of an adenoviral vector in baboons. Hum Gene Ther 13: 143-154.
13. Raper, SE, Yudkoff, M, Chirmule, N, Gao, GP, Nunes, F, Haskal, ZJ et al. (2002). A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency. Hum Gene Ther 13: 163-175.
14. Brunetti-Pierri, N, Palmer, DJ, Beaudet, AL, Carey, KD, Finegold, M and Ng, P (2004). Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates. Hum Gene Ther 15: 35-46.
15. Varnavski, AN, Zhang, Y, Schnell, M, Tazelaar, J, Louboutin, JP, Yu, QC et al. (2002). Preexisting immunity to adenovirus in rhesus monkeys fails to prevent vector-induced toxicity. J Virol 76 5711-5719.
16. Muruve, DA, Cotter, MJ, Zaiss, AK, White, LR, Liu, Q, Chan, T et al. (2004). Helper-dependent adenovirus vectors elicit intact innate but attenuated adaptive host immune responses in vivo. J Virol 78 5966-5972.
17. Schnell, MA, Zhang, Y, Tazelaar, J, Gao, GP, Yu, QC, Qian, R et al. (2001). Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors. Mol Ther 3 708-722.
18. Raper, SE, Chirmule, N, Lee, FS, Wivel, NA, Bagg, A, Gao, GP et al. (2003). Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 80: 148-158.
19. Muruve, DA, Barnes, MJ, Stillman, IE and Libermann, TA (1999). Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo. Hum Gene Ther 10: 965-976.
20. Zhang, Y, Chirmule, N, Gao, GP, Qian, R, Croyle, M, Joshi, B et al. (2001). Acute cytokine response to systemic adenoviral vectors in mice is mediated by dendritic cells and macrophages. Mol Ther 3 697-707.
21. O'Neal, WK, Zhou, H, Morral, N, Aguilar-Cordova, E, Pestaner, J, Langston, C et al. (1998). Toxicological comparison of E2a-deleted and first-generation adenoviral vectors expressing alpha1-antitrypsin after systemic delivery. Hum Gene Ther 9: 1587-1598.
22. Berkenstadt, H, Ben-Ari, G and Perel, A (1998). Hemodynamic changes during a new procedure for regional chemotherapy involving occlusion of the thoracic aorta and inferior vena cava. J Clin Anesth 10: 636-640.
23. Palmer, D and Ng, P (2003). Improved system for helper-dependent adenoviral vector production. Mol Ther 8: 846-852.
24. Palmer, DJ and Ng, P (2004). Physical and infectious titers of helper-dependent adenoviral vectors: A method of direct comparison to the adenovirus reference material. Mol Ther 10: 792-798.
25. Croyle, MA, Chirmule, N, Zhang, Y and Wilson, JM (2001). "Stealth" adenoviruses blunt cell-mediated and humoral immune responses against the virus and allow for significant gene expression upon readministration in the lung. J Virol 75: 4792-4801.