Readministration of helper-dependent adenoviral vectors to mouse airway mediated via transient immunosuppression

Gene Therapy

Volumn 18, Issue 2, 2011, Pages 173-181

  Cao, H ^a   Yang, T ^a,b   Li, X F ^a,c   Wu, J ^a   Duan, C ^a   Coates, A L ^a,d   Hu, J ^a,d,e  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b CHINA AGRICULTURAL UNIVERSITY   (China)

^c FOURTH MILITARY MEDICAL UNIVERSITY   (China)

^d UNIVERSITY OF TORONTO   (Canada)

^e UNIVERSITY OF TORONTO   (Canada)

Author keywords

cyclophosphamide (cytoxan); cystic fibrosis; gene expression; immune responses; lung gene therapy

Indexed keywords

ADENOVIRUS VECTOR; BETA GALACTOSIDASE; CAPSID PROTEIN; CYCLOPHOSPHAMIDE; CYTOKINE; NEUTRALIZING ANTIBODY; TRANSMEMBRANE CONDUCTANCE REGULATOR; VIRUS ANTIBODY;

AIRWAY; ARTICLE; CD4+ T LYMPHOCYTE; CD8+ T LYMPHOCYTE; CELL INFILTRATION; CONTROLLED STUDY; FEMALE; GENE EXPRESSION; HUMAN; IMMUNE DEFICIENCY; IMMUNE RESPONSE; IMMUNE SYSTEM; IMMUNITY; INFLAMMATORY CELL; LEUKOCYTE COUNT; LYMPHOCYTE; MOUSE; NONHUMAN; PRIORITY JOURNAL; REPORTER GENE; TRANSGENE; VIRAL GENE THERAPY; VIRUS CAPSID;

ADENOVIRIDAE; ADMINISTRATION, INTRANASAL; ANIMALS; ANTIBODIES, VIRAL; CYCLOPHOSPHAMIDE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DEFECTIVE VIRUSES; FEMALE; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; IMMUNOSUPPRESSIVE AGENTS; MICE; MICE, INBRED C57BL; RETREATMENT;

ADENOVIRIDAE; MIRIDAE; MUS;

EID: 79951517549     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2010.125     Document Type: Article

References (48)

1. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008; 358: 2231-2239. (Pubitemid 351724452)
2. Maguire AM, Simonelli F, Pierce EA, Pugh Jr EN, Mingozzi F, Bennicelli J et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008; 358: 2240-2248. (Pubitemid 351724453)
3. Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci USA 2008; 105: 15112-15117.
4. Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL et al. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther 2009; 20: 999-1004.
5. Wilson JM, Gansbacher B, Berns KI, Bosch F, Kay MA, Naldini L et al. Good news on the clinical gene transfer front. Hum Gene Ther 2008; 19: 429-430. (Pubitemid 351748795)
6. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360: 447-458.
7. Flotte TR, Ng P, Dylla DE, McCray Jr PB, Wang G, Kolls JK et al. Viral vector-mediated and cell-based therapies for treatment of cystic fibrosis. Mol Ther 2007; 15: 229-241. (Pubitemid 46111871)
8. Pearson H. Human genetics: one gene, twenty years. Nature 2009; 460: 164-169.
9. Ferrari S, Griesenbach U, Geddes DM, Alton E. Immunological hurdles to lung gene therapy. Clin Exp Immunol 2003; 132: 1-8. (Pubitemid 36418899)
10. Wu TL, Ertl HC. Immune barriers to successful gene therapy. Tr e n d s M o l M e d 2009; 15: 32-39.
11. Chirmule N, Tazelaar J, Wilson JM. Th2-dependent B cell responses in the absence of CD40-CD40 ligand interactions. J Immunol 2000; 164: 248-255. (Pubitemid 30012997)
12. Jooss K, Yang Y, Wilson JM. Cyclophosphamide diminishes inflammation and prolongs transgene expression following delivery of adenoviral vectors to mouse liver and lung. Hum Gene Ther 1996; 7: 1555-1566. (Pubitemid 26302210)
13. Scaria A, St George JA, Gregory RJ, Noelle RJ, Wadsworth SC, Smith AE et al. Antibody to CD40 ligand inhibits both humoral and cellular immune responses to adenoviral vectors and facilitates repeated administration to mouse airway. Gene Ther 1997; 4: 611-617. (Pubitemid 27352959)
14. Zsengeller ZK, Boivin GP, Sawchuk SS, Trapnell BC, Whitsett JA, Hirsch R. Anti-T cell receptor antibody prolongs transgene expression and reduces lung inflammation after adenovirus-mediated gene transfer. Hum Gene Ther 1997; 8: 935-941. (Pubitemid 27270156)
15. Ye X, Robinson MB, Pabin C, Batshaw ML, Wilson JM. Transient depletion of CD4 lymphocyte improves efficacy of repeated administration of recombinant adeno-virus in the ornithine transcarbamylase deficient sparse fur mouse. Gene Ther 2000; 7: 1761-1767.
16. Chirmule N, Raper SE, Burkly L, Thomas D, Tazelaar J, Hughes JV et al. Readministration of adenovirus vector in nonhuman primate lungs by blockade of CD40-CD40 ligand interactions. J Virol 2000; 74: 3345-3352. (Pubitemid 30143760)
17. 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.
18. Koehler DR, Martin B, Corey M, Palmer D, Ng P, Tanswell AK et al. Readministration of helper-dependent adenovirus to mouse lung. Gene Ther 2006; 13: 773-780.
19. Croyle MA, Chirmule N, Zhang Y, Wilson JM. '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 2001; 75: 4792-4801. (Pubitemid 32381522)
20. Eto Y, Yoshioka Y, Mukai Y, Okada N, Nakagawa S. Development of PEGylated adenovirus vector with targeting ligand. Int J Pharm 2008; 354: 3-8. (Pubitemid 351400325)
21. O'Neal WK, Zhou H, Morral N, Langston C, Parks RJ, Graham FL et al. Toxicity associated with repeated administration of first-generation adenovirus vectors does not occur with a helper-dependent vector. Mol Med 2000; 6: 179-195.
22. Mian A, Guenther M, Finegold M, Ng P, Rodgers J, Lee B. Toxicity and adaptive immune response to intracellular transgenes delivered by helper-dependent vs first generation adenoviral vectors. Mol Genet Metab 2005; 84: 278-288. (Pubitemid 40202775)
23. Kim IH, Jozkowicz A, Piedra PA, Oka K, Chan L. Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci USA 2001; 98: 13282-13287. (Pubitemid 33051354)
24. 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 helper-dependent adenoviral vector expressing the very low-density lipoprotein receptor. Circulation 2001; 103: 1274-1281. (Pubitemid 32221352)
25. Croyle MA, Le HT, Linse KD, Cerullo V, Toietta G, Beaudet A et al. PEGylated helper-dependent adenoviral vectors: highly efficient vectors with an enhanced safety profile. Gene Ther 2005; 12: 579-587. (Pubitemid 40468105)
26. Iwai T, Tomita Y, Shimizu I, Zhang Q, Okano S, Minagawa R et al. Different role of cyclophosphamide-induced tolerance in heart and skin allograft tolerance. Transplant Proc 2001; 33: 173-175. (Pubitemid 32237321)
27. Okano S, Eto M, Tomita Y, Yoshizumi T, Yamada H, Minagawa R et al. Cyclophos-phamide-induced tolerance in rat orthotopic liver transplantation. Transplantation 2001; 71: 447-456. (Pubitemid 32175984)
28. Takeda A, Uchida K, Haba T, Tominaga Y, Katayama A, Kobayashi T et al. Acute humoral rejection of kidney allografts in patients with a positive flow cytometry crossmatch (FCXM). Clin Transplant 2000; 14 (Suppl 3): 15-20.
29. Dai Y, Schwarz EM, Gu D, Zhang WW, Sarvetnick N, Verma IM. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc Natl Acad Sci USA 1995; 92: 1401-1405.
30. Otake K, Ennist DL, Harrod K, Trapnell BC. Nonspecific inflammation inhibits adenovirus-mediated pulmonary gene transfer and expression independent of specific acquired immune responses. Hum Gene Ther 1998; 9: 2207-2222. (Pubitemid 28498007)
31. Muruve DA, Cotter MJ, Zaiss AK, White LR, Liu Q, Chan T et al. Helper-dependent adenovirus vectors elicit intact innate but attenuated adaptive host immune responses in vivo. J Virol 2004; 78: 5966-5972. (Pubitemid 38661684)
32. Butti E, Bergami A, Recchia A, Brambilla E, Franciotta D, Cattalini A et al. Absence of an intrathecal immune reaction to a helper-dependent adenoviral vector delivered into the cerebrospinal fluid of non-human primates. Gene Ther 2008; 15: 233-238. (Pubitemid 351150046)
33. Xiong W, Goverdhana S, Sciascia SA, Candolfi M, Zirger JM, Barcia C et al. Regulatable gutless adenovirus vectors sustain inducible transgene expression in the brain in the presence of an immune response against adenoviruses. J Virol 2006; 80: 27-37.
34. Brunetti-Pierri N, Ng P. Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors. Gene Ther 2008; 15: 553-560.
35. Seiler MP, Cerullo V, Lee B. Immune response to helper dependent adenoviral mediated liver gene therapy: challenges and prospects. Curr Gene Ther 2007; 7: 297-305. (Pubitemid 47595772)
36. Lowenstein PR, Mandel RJ, Xiong WD, Kroeger K, Castro MG. Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the cell biology of neuroimmune interactions. Curr Gene Ther 2007; 7: 347-360. (Pubitemid 47595777)
37. Muruve DA, Barnes MJ, Stillman IE, Libermann TA. Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo. Hum Gene Ther 1999; 10: 965-976. (Pubitemid 29195426)
38. Price AR, Limberis MP, Wilson JM, Diamond SL. Pulmonary delivery of adenovirus vector formulated with dexamethasone-spermine facilitates homologous vector re-administration. Gene Ther 2007; 14: 1594-1604. (Pubitemid 350056231)
39. 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 non-human primates. Hum Gene Ther 2004; 15: 35-46. (Pubitemid 38147187)
40. Zsengeller Z, Otake K, Hossain SA, Berclaz PY, Trapnell BC. Internalization of adenovirus by alveolar macrophages initiates early proinflammatory signaling during acute respiratory tract infection. J Virol 2000; 74: 9655-9667.
41. Franchimont D, Galon J, Gadina M, Visconti R, Zhou Y, Aringer M et al. Inhibition of Th1 immune response by glucocorticoids: dexamethasone selectively inhibits IL-12-induced Stat4 phosphorylation in T lymphocytes. J Immunol 2000; 164: 1768-1774. (Pubitemid 30108728)
42. Moore DJ, Markmann JF, Deng S. Avenues for immunomodulation and graft protection by gene therapy in transplantation. TransplInt 2006; 19: 435-445. (Pubitemid 43736339)
43. Bouvet M, Fang B, Ekmekcioglu S, Ji L, Bucana CD, Hamada K et al. Suppression of the immune response to an adenovirus vector and enhancement of intratumoral transgene expression by low-dose etoposide. Gene Ther 1998; 5: 189-195. (Pubitemid 28077746)
44. Koehler DR, Sajjan U, Chow YH, Martin B, Kent G, Tanswell AK et al. Protection of Cftr knockout mice from acute lung infection by a helper-dependent adenoviral vector expressing Cftr in airway epithelia. Proc Natl Acad Sci USA 2003; 100: 15364-15369. (Pubitemid 38020995)
45. Toietta G, Koehler DR, Finegold MJ, Lee B, Hu J, Beaudet AL. Reduced inflammation and improved airway expression using helper-dependent adenoviral vectors with a K18 promoter. Mol Ther 2003; 7: 649-658. (Pubitemid 36626245)
46. Wu J, Duan R, Cao H, Field D, Newnham C, Koehler DR et al. Regulation of epithelium-specific Ets-like transcription factors ESE-1 and ESE-3 in airway epithelial cells. Cell Res 2008; 18: 649-663. (Pubitemid 351793120)
47. Collins DP, Luebering BJ, Shaut DM. T-lymphocyte functionality assessed by analysis of cytokine receptor expression, intracellular cytokine expression, and femtomolar detection of cytokine secretion by quantitative flow cytometry. Cytometry 1998; 33: 249-255. (Pubitemid 28447151)
48. Livak KJ, Schmittgen TD. Analysis of relative gene expression data using real-time quantitative PCR and the 2(-Delta Delta C(T)) Method. Methods 2001; 25: 402-408. (Pubitemid 34164012)