Pseudo-hydrodynamic Delivery of Helper-dependent Adenoviral Vectors into Non-human Primates for Liver-directed Gene Therapy

Molecular Therapy - Tập 15 - Trang 732-740 - 2007
Nicola Brunetti-Pierri1, Gary E Stapleton2, Donna J Palmer1, Yu Zuo1, Viraj P Mane1, Milton J Finegold3, Arthur L Beaudet1, Michelle M Leland4, Charles E Mullins2, Philip Ng1
1Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas, USA.
2Department of Pediatric Cardiology, Baylor College of Medicine, Houston, Texas, USA
3Department of Pathology, Baylor College of Medicine, Houston, Texas USA
4Southwest Foundation for Biomedical Research, San Antonio, Texas, USA

Tài liệu tham khảo

Nguyen, 2004, Liver gene therapy: advances and hurdles, Gene Ther, 11, S76, 10.1038/sj.gt.3302373 Palmer, 2005, Helper-dependent adenoviral vectors for gene therapy, Hum Gene Ther, 16, 1, 10.1089/hum.2005.16.1 Kim, 2001, Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector, Proc Natl Acad Sci USA, 98, 12387, 10.1073/pnas.241506298 Toietta, 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, 10.1073/pnas.0500930102 Brunetti-Pierri, 2005, Sustained phenotypic correction of canine hemophilia B following systemic administration of helper dependent adenoviral vectors, Hum Gene Ther, 16, 811, 10.1089/hum.2005.16.811 Brunetti-Pierri, 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, 10.1089/hum.2006.17.391 Morral, 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, 10.1073/pnas.96.22.12816 Morral, 1998, High doses of a helper-dependent adenoviral vector yield supraphysiological levels of 1-antitrypsin with negligible toxicity, Hum Gene Ther, 9, 2709, 10.1089/hum.1998.9.18-2709 Tao, 2001, Sequestration of adenoviral vector by kupffer cells leads to a nonlinear dose response of transduction in liver, Mol Ther, 3, 28, 10.1006/mthe.2000.0227 Schiedner, 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, 10.1016/S1525-0016(02)00017-5 Sullivan, 1997, Liver-directed gene transfer in non-human primates, Hum Gene Ther, 8, 1195, 10.1089/hum.1997.8.10-1195 Nunes, 1999, Gene transfer into the liver of nonhuman primates with E1-deleted recombinant adenoviral vectors: safety of readministration, Hum Gene Ther, 10, 2515, 10.1089/10430349950016852 Morral, 2002, Lethal toxicity, severe endothelial injury, and a threshold effect with high doses of an adenoviral vector in baboons, Hum Gene Ther, 13, 143, 10.1089/10430340152712692 Muruve, 2004, The innate immune response to adenovirus vectors, Hum Gene Ther, 15, 1157, 10.1089/hum.2004.15.1157 Muruve, 2004, Helper-dependent adenovirus vectors elicit intact innate but attenuated adaptive host immune responses in vivo, J Virol, 78, 5966, 10.1128/JVI.78.11.5966-5972.2004 Schnell, 2001, Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors, Mol Ther, 3, 708, 10.1006/mthe.2001.0330 Raper, 2003, Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer, Mol Genet Metab, 80, 148, 10.1016/j.ymgme.2003.08.016 Muruve, 1999, Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo, Hum Gene Ther, 10, 965, 10.1089/10430349950018364 Brunetti-Pierri, 2004, Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into non human primates, Hum Gene Ther, 15, 35, 10.1089/10430340460732445 Zhang, 2001, Acute cytokine response to systemic adenoviral vectors in mice is mediated by dendritic cells and macrophages, Mol Ther, 3, 697, 10.1006/mthe.2001.0329 Liu, 1999, Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA, Gene Ther, 6, 1258, 10.1038/sj.gt.3300947 Zhang, 1999, High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA, Hum Gene Ther, 10, 1735, 10.1089/10430349950017734 Brunetti-Pierri, 2005, Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors, Mol Ther, 12, 99, 10.1016/j.ymthe.2005.03.001 Andrianaivo, 2004, Hydrodynamics-based transfection of the liver: entrance into hepatocytes of DNA that causes expression takes place very early after injection, J Gene Med, 6, 877, 10.1002/jgm.574 Zhang, 2004, Hydroporation as the mechanism of hydrodynamic delivery, Gene Ther, 11, 675, 10.1038/sj.gt.3302210 Lievens, 2004, The size of sinusoidal fenestrae is a critical determinant of hepatocyte transduction after adenoviral gene transfer, Gene Ther, 11, 1523, 10.1038/sj.gt.3302326 Meier, 2003, Adenovirus endocytosis, J Gene Med, 5, 451, 10.1002/jgm.409 Zhang, 1997, Expression of naked plasmid DNA injected into the afferent and efferent vessels of rodent and dog livers, Hum Gene Ther, 8, 1763, 10.1089/hum.1997.8.15-1763 Eastman, 2002, Development of catheter-based procedures for transducing the isolated rabbit liver with plasmid DNA, Hum Gene Ther, 13, 2065, 10.1089/10430340260395910 Parks, 1999, Effects of stuffer DNA on transgene expression from helper-dependent adenovirus vectors, J Virol, 73, 8027, 10.1128/JVI.73.10.8027-8034.1999 Sandig, 1999, Optimization of the helper-dependent adenovirus system for production and potency in vivo, Proc Natl Acad Sci USA, 97, 1002, 10.1073/pnas.97.3.1002 Schiedner, 2002, Variables affecting in vivo performance of high-capacity adenovirus vectors, J Virol, 76, 1600, 10.1128/JVI.76.4.1600-1609.2002 Berkenstadt, 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, 10.1016/S0952-8180(98)00100-7 Palmer, 2003, Improved system for helper-dependent adenoviral vector production, Mol Ther, 8, 846, 10.1016/j.ymthe.2003.08.014 Palmer, 2004, Physical and infectious titers of helper-dependent adenoviral vectors: a method of direct comparison to the adenovirus reference material, Mol Ther, 10, 792, 10.1016/j.ymthe.2004.06.1013 Croyle, 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, 10.1128/JVI.75.10.4792-4801.2001 Varnavski, 2002, Preexisting immunity to adenovirus in rhesus monkeys fails to prevent vector-induced toxicity, J Virol, 76, 5711, 10.1128/JVI.76.11.5711-5719.2002