Erratum: PEGylated helper-dependent adenoviral vector expressing human Apo A-I for gene therapy in LDLR-deficient mice (Gene Therapy (2013) 20 (1124-1130) DOI: 10.1038/gt.2013.38);PEGylated helper-dependent adenoviral vector expressing human Apo A-I for gene therapy in LDLR-deficient mice

Gene Therapy

Volumn 20, Issue 12, 2013, Pages 1124-1130

  Leggiero, E ^a,b   Astone, D ^a,b   Cerullo, V ^a,b,c   Lombardo, B ^a,b   Mazzaccara, C ^b   Labruna, G ^d   Sacchetti, L ^a,b   Salvatore, F ^a,d   Croyle, M ^e   Pastore, L ^a,b  

^a CEINGE BIOTECNOLOGIE AVANZATE   (Italy)

^b UNIVERSITY OF NAPLES FEDERICO II   (Italy)

^c UNIVERSITY OF HELSINKI   (Finland)

^d IRCCS SDN   (Italy)

^e UNIVERSITY OF TEXAS AT AUSTIN   (United States)

Author keywords

atherosclerosis; helper dependent adenoviral vectors; PEGylation

Indexed keywords

ADENOVIRUS VECTOR; APOLIPOPROTEIN A1; HIGH DENSITY LIPOPROTEIN; HIGH DENSITY LIPOPROTEIN CHOLESTEROL; INTERLEUKIN 12P40; INTERLEUKIN 12P70; INTERLEUKIN 6; LOW DENSITY LIPOPROTEIN; LOW DENSITY LIPOPROTEIN CHOLESTEROL; LOW DENSITY LIPOPROTEIN RECEPTOR; MONOCYTE CHEMOTACTIC PROTEIN 1; PEGYLATED HELPER DEPENDENT ADENOVIRAL VECTOR; TUMOR NECROSIS FACTOR ALPHA; UNCLASSIFIED DRUG;

ACUTE TOXICITY; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; AORTA ATHEROSCLEROSIS; ARTICLE; ATHEROSCLEROTIC PLAQUE; CHOLESTEROL BLOOD LEVEL; CHOLESTEROL DIET; CHOLESTEROL METABOLISM; CONTROLLED STUDY; CYTOKINE RESPONSE; DRUG MEGADOSE; FAMILIAL HYPERCHOLESTEROLEMIA; FEMALE; GENE OVEREXPRESSION; GENE THERAPY; IMMUNE RESPONSE; IN VITRO GENE TRANSFER; INFLAMMATION; LIPID DIET; LIPID METABOLISM; LIPOPROTEIN BLOOD LEVEL; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; TRANSGENE; TRIACYLGLYCEROL BLOOD LEVEL; VIRAL GENE DELIVERY SYSTEM;

ANIMALIA; MURINAE; MUS; PRIMATES;

ADENOVIRIDAE; ANIMALS; APOLIPOPROTEIN A-I; CHOLESTEROL, HDL; CHOLESTEROL, LDL; CYTOKINES; DISEASE MODELS, ANIMAL; FEMALE; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HELPER VIRUSES; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; MICE; MICE, INBRED C57BL; PLAQUE, ATHEROSCLEROTIC; POLYETHYLENE GLYCOLS; RECEPTORS, LDL;

EID: 84890122203     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2013.45     Document Type: Erratum

References (49)

1. Fitzgerald ML, Mujawar Z, Tamehiro N. ABC transporters, atherosclerosis and inflammation. Atherosclerosis 2010; 211: 361-370.
2. Yusuf S, Hawken S, Ounpuu S, Dans T, Avezum A, Lanas F et al. Effect of potentially modifiable risk factors associated with myocardial infarction in 52 countries (the INTERHEART study): Case-control study. Lancet 2004; 364: 937-952.
3. Civeira F. Guidelines for the diagnosis and management of heterozygous familial hypercholesterolemia. Atherosclerosis 2004; 173: 55-68.
4. Rashid S, Marcil M, Ruel I, Genest J. Identification of a novel human cellular HDL biosynthesis defect. Eur Heart J 2009; 30: 2204-2212.
5. Oram JF, Vaughan AM. ATP-Binding cassette cholesterol transporters and cardiovascular disease. Circ Res 2006; 99: 1031-1043.
6. Lewis GF, Rader DJ. New insights into the regulation of HDL metabolism and reverse cholesterol transport. Circ Res 2005; 96: 1221-1232.
7. Robinson JG, Bakris G, Torner J, Stone NJ, Wallace R. Is it time for a cardiovascular primary prevention trial in the elderly? Stroke 2007; 38: 441-450.
8. Assmann G, Gotto Jr AM. HDL cholesterol and protective factors in atherosclerosis. Circulation 2004; 109(23 Suppl 1): III8-14.
9. Van Craeyveld E, Gordts S, Jacobs F, De Geest B. Gene therapy to improve high-density lipoprotein metabolism and function. Curr Pharm Des 2010; 16: 1531-1544.
10. Van Lenten BJ, Wagner AC, Anantharamaiah GM, Navab M, Reddy ST, Buga GM et al. Apolipoprotein A-I mimetic peptides. Curr Atheroscler Rep 2009; 11: 52-57.
11. Harris JD, Evans V, Owen JS. ApoE gene therapy to treat hyperlipidemia and atherosclerosis. Curr Opin Mol Ther 2006; 8: 275-287.
12. De Geest B, Van Linthout S, Collen D. Sustained expression of human apo A-I following adenoviral gene transfer in mice. Gene Therapy 2001; 8: 121-127.
13. Pastore L, Belalcazar LM, Oka K, Cela R, Lee B, Chan L et al. Helper-dependent adenoviral vector-mediated long-term expression of human apolipoprotein A-I reduces atherosclerosis in apo E-deficient mice. Gene 2004; 327: 153-160.
14. Kassim SH, Wilson JM, Rader DJ. Gene therapy for dyslipidemia: A review of gene replacement and gene inhibition strategies. Clin Lipidol 2010; 5: 793-809.
15. Toietta G, Mane VP, Norona WS, Finegold MJ, Ng P, McDonagh AF et al. Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helperdependent adenoviral vector. Proc Natl Acad Sci USA 2005; 102: 3930-3935.
16. Oka K, Pastore L, Kim IH, Merched A, Nomura S, Lee HJ et al. Long-term stable correction of low-density lipoprotein receptor-deficient mice with a helperdependent adenoviral vector expressing the very low-density lipoprotein receptor. Circulation 2001; 103: 1274-1281.
17. Belalcazar LM, Merched A, Carr B, Oka K, Chen KH, Pastore L et al. Long-term stable expression of human apolipoprotein A-I mediated by helper-dependent adenovirus gene transfer inhibits atherosclerosis progression and remodels atherosclerotic plaques in a mouse model of familial hypercholesterolemia. Circulation 2003; 107: 2726-2732.
18. Cerreto M, Mehdawy B, Ombrone D, Nistico R, Ruoppolo M, Usiello A et al. Reversal of metabolic and neurological symptoms of phenylketonuric mice treated with a PAH containing helper-dependent adenoviral vector. Curr Gene Ther 2012; 12: 48-56.
19. Morral N, Parks RJ, Zhou H, Langston C, Schiedner G, Quinones J et al. High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicity. Hum Gene Ther 1998; 9: 2709-2716.
20. Schiedner G, Morral N, Parks RJ, Wu Y, Koopmans SC, Langston C et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet 1998; 18: 180-183.
21. Brunetti-Pierri N, Palmer DJ, Beaudet AL, Carey KD, Finegold M, Ng P. Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates. Hum Gene Ther 2004; 15: 35-46.
22. Muruve DA. The innate immune response to adenovirus vectors. Hum Gene Ther 2004; 15: 1157-1166.
23. Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003; 80: 148-158.
24. Appledorn DM, Patial S, McBride A, Godbehere S, Van Rooijen N, Parameswaran N et al. Adenovirus vector-induced innate inflammatory mediators, MAPK signaling, as well as adaptive immune responses are dependent upon both TLR2 and TLR9 in vivo. J Immunol 2008; 181: 2134-2144.
25. Cerullo V, Seiler MP, Mane V, Brunetti-Pierri N, Clarke C, Bertin TK et al. Toll-like receptor 9 triggers an innate immune response to helper-dependent adenoviral vectors. Mol Ther 2007; 15: 378-385.
26. Seregin SS, Appledorn DM, McBride AJ, Schuldt NJ, Aldhamen YA, Voss T et al. Transient pretreatment with glucocorticoid ablates innate toxicity of systemically delivered adenoviral vectors without reducing efficacy. Mol Ther 2009; 17: 685-696.
27. Mane VP, Toietta G, McCormack WM, Conde I, Clarke C, Palmer D et al. Modulation of TNFalpha, a determinant of acute toxicity associated with systemic delivery of first-generation and helper-dependent adenoviral vectors. Gene Therapy 2006; 13: 1272-1280.
28. Croyle MA, Le HT, Linse KD, Cerullo V, Toietta G, Beaudet A et al. PEGylated helperdependent adenoviral vectors: Highly efficient vectors with an enhanced safety profile. Gene Therapy 2005; 12: 579-587.
29. Wonganan P, Clemens CC, Brasky K, Pastore L, Croyle MA. Species differences in the pharmacology and toxicology of PEGylated helper-dependent adenovirus. Mol Pharm 2011; 8: 78-92.
30. Alemany R, Suzuki K, Curiel DT. Blood clearance rates of adenovirus type 5 in mice. J Gen Virol 2000; 81(Pt 11): 2605-2609.
31. Prill JM, Espenlaub S, Samen U, Engler T, Schmidt E, Vetrini F et al. Modifications of adenovirus hexon allow for either hepatocyte detargeting or targeting with potential evasion from Kupffer cells. Mol Ther 2011; 19: 83-92.
32. Hofherr SE, Shashkova EV, Weaver EA, Khare R, Barry MA. Modification of adenoviral vectors with polyethylene glycol modulates in vivo tissue tropism and gene expression. Mol Ther 2008; 16: 1276-1282.
33. Weaver EA, Barry MA. Effects of shielding adenoviral vectors with polyethylene glycol on vector-specific and vaccine-mediated immune responses. Hum Gene Ther 2008; 19: 1369-1382.
34. Weaver EA, Nehete PN, Buchl SS, Senac JS, Palmer D, Ng P et al. Comparison of replication-competent, first generation, and helper-dependent adenoviral vaccines. PLoS One 2009; 4: E5059.
35. Hofherr SE, Senac JS, Chen CY, Palmer DJ, Ng P, Barry MA. Short-term rescue of neonatal lethality in a mouse model of propionic acidemia by gene therapy. Hum Gene Ther 2009; 20: 169-180.
36. Weaver EA, Chen CY, May SM, Barry ME, Barry MA. Comparison of adenoviruses as oncolytics and cancer vaccines in an immunocompetent B cell lymphoma model. Hum Gene Ther 2011; 22: 1095-1100.
37. Ishibashi S, Brown MS, Goldstein JL, Gerard RD, Hammer RE, Herz J. Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirus-mediated gene delivery. J Clin Invest 1993; 92: 883-893.
38. Tangirala RK, Rubin EM, Palinski W. Quantitation of atherosclerosis in murine models: Correlation between lesions in the aortic origin and in the entire aorta, and differences in the extent of lesions between sexes in LDL receptor-deficient and apolipoprotein E-deficient mice. J Lipid Res 1995; 36: 2320-2328.
39. Benihoud K, Esselin S, Descamps D, Jullienne B, Salone B, Bobe P et al. Respective roles of TNF-alpha and IL-6 in the immune response-elicited by adenovirusmediated gene transfer in mice. Gene Therapy 2007; 14: 533-544.
40. Aldhamen YA, Seregin SS, Amalfitano A. Immune recognition of gene transfer vectors: Focus on adenovirus as a paradigm. Front Immunol 2011; 2: 40.
41. Appledorn DM, Aldhamen YA, Depas W, Seregin SS, Liu CJ, Schuldt N et al. A new adenovirus based vaccine vector expressing an Eimeria tenella derived TLR agonist improves cellular immune responses to an antigenic target. PLoS One 2010; 5: E9579.
42. Hartman ZC, Kiang A, Everett RS, Serra D, Yang XY, Clay TM et al. Adenovirus infection triggers a rapid, MyD88-regulated transcriptome response critical to acute-phase and adaptive immune responses in vivo. J Virol 2007; 81: 1796-1812.
43. Brunetti-Pierri N, Liou A, Patel P, Palmer D, Grove N, Finegold M et al. Balloon catheter delivery of helper-dependent adenoviral vector results in sustained, therapeutic hFIX expression in rhesus macaques. Mol Ther 2012; 20: 1863-1870.
44. Brunetti-Pierri N, Ng T, Iannitti DA, Palmer DJ, Beaudet AL, Finegold MJ et al. Improved hepatic transduction, reduced systemic vector dissemination, and longterm transgene expression by delivering helper-dependent adenoviral vectors into the surgically isolated liver of nonhuman primates. Hum Gene Ther 2006; 17: 391-404.
45. Brunetti-Pierri N, Clarke C, Mane V, Palmer DJ, Lanpher B, Sun Q et al. Phenotypic correction of ornithine transcarbamylase deficiency using low dose helperdependent adenoviral vectors. J Gene Med 2008; 10: 890-896.
46. Toietta G, Pastore L, Cerullo V, Finegold M, Beaudet AL, Lee B. Generation of helper-dependent adenoviral vectors by homologous recombination. Mol Ther 2002; 5: 204-210.
47. Zhou H, Pastore L, Beaudet AL. Helper-dependent adenoviral vectors. Methods Enzymol 2002; 346: 177-198.
48. Palmer D, Ng P. Improved system for helper-dependent adenoviral vector production. Mol Ther 2003; 8: 846-852.
49. Bolin LM, Zhaung A, Strychkarska-Orczyk I, Nelson E, Huang I, Malit M et al. Differential inflammatory activation of IL-6 (-/-) astrocytes. Cytokine 2005; 30: 47-55.