Immune response to lentiviral bilirubin UDP-glucuronosyltransferase gene transfer in fetal and neonatal rats

Gene Therapy

Volumn 13, Issue 8, 2006, Pages 672-677

  Seppen, J ^a   van Til, N P ^a   van der Rijt, R ^a   Hiralall, J K ^a   Kunne, C ^a   Oude Elferink, R P J ^a  

^a ACADEMIC MEDICAL CENTER   (Netherlands)

Author keywords

Bilirubin; Crigler Najjar; Gunn rat; In utero; Lentiviral vectors

Indexed keywords

BILIRUBIN GLUCURONIDE; GLUCURONOSYLTRANSFERASE 1A1; GREEN FLUORESCENT PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY RESPONSE; ARTICLE; BILIRUBIN BLOOD LEVEL; CONTROLLED STUDY; CRIGLER NAJJAR SYNDROME; FETUS; GENE EXPRESSION; GENE TRANSFER; HYPERBILIRUBINEMIA; IMMUNOLOGICAL TOLERANCE; LENTIVIRUS VECTOR; LONG TERM CARE; NEWBORN; NONHUMAN; PRIORITY JOURNAL; RAT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALS; ANIMALS, NEWBORN; ANTIBODIES; CRIGLER-NAJJAR SYNDROME; FETUS; GENE THERAPY; GENETIC VECTORS; GLUCURONOSYLTRANSFERASE; GREEN FLUORESCENT PROTEINS; IMMUNE TOLERANCE; INJECTIONS; LENTIVIRUS; LIVER; RATS; RATS, GUNN; TRANSDUCTION, GENETIC;

ANIMALIA; LENTIVIRUS; RATTUS NORVEGICUS;

EID: 33645779725     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302681     Document Type: Article

References (38)

1. Nguyen TH, Ferry N. Liver gene therapy: Advances and hurdles. Gene Therapy 2004; 11: S76-S84.
2. Wang CH, Liu DW, Tsao YP, Xiao X, Chen SL. Can genes transduced by adeno-associated virus vectors elicit or evade an immune response? Arch Virol 2004; 149: 1-15.
3. Billingham RE, Brent L, Medawar PB. Actively aquired tolerance of foreign cells. Nature 1953; 172: 603-606.
4. Nossal GJ. Cellular mechanisms of immunologic tolerance. Annu Rev Immunol 1983; 1: 33-62.
5. Zanjani ED, Anderson WF. Review: Medicine - Prospects for in utero human gene therapy. Science 1999; 285: 2084-2088.
6. Coutelle C, Themis M, Waddington S, Gregory L, Nivsarkar M, Buckley S et al. The hopes and fears of in utero gene therapy for genetic disease - a review. Placenta 2003; 24: S114-S121.
7. Waddington SN, Kramer MG, Hernandez-Alcoceba R, Buckley SM, Themis M, Coutelle C et al. In utero gene therapy: Current challenges and perspectives. Mol Ther 2005; 11: 661-676.
8. Waddington SN, Buckley SM, Nivsarkar M, Jezzard S, Schneider H, Dahse T et al. In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor. Blood 2003; 101: 1359-1366.
9. Waddington SN, Nivsarkar MS, Mistry AR, Buckley SM, Kemball-Cook G, Mosley KL et al. Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Blood 2004; 104: 2714-2721.
10. Ostrow JD, Mukerjee P, Tiribelli C. Structure and binding of unconjugated bilirubin: Relevance for physiological and pathophysiological function. J Lipid Res 1994; 35: 1715-1737.
11. Tukey RH, Strassburg CP. Human UDP-glucuronosyltransferases: Metabolism, expression, and disease. Annu Rev Pharmacol Toxicol 2000; 40: 581-616.
12. Jansen PL. Diagnosis and management of Crigler-Najjar syndrome. Eur J Pediatr 1999; 158 (Suppl 2): S89-S94.
13. Ostrow JD, Pascolo L, Tiribelli C. Mechanisms of bilirubin neurotoxicity. Hepatology 2002; 35: 1277-1280.
14. Chowdhury JR, Kondapalli R, Chowdhury NR. Gunn rat: A model for inherited deficiency of bilirubin glucuronidation. Adv Vet Sci Comp Med 1993; 37: 149-173.
15. Aubert D, Menoret S, Chiari E, Pichard V, Durand S, Tesson L et al. Cytotoxic immune response blunts long-term transgene expression after efficient retroviral-mediated hepatic gene transfer in rat. Mol Ther 2002; 5: 388-396.
16. Ailles LE, Naldini L. HIV-1-derived lentiviral vectors. Curr Top Microbiol Immunol 2002; 261: 31-52.
17. Galimi F, Verma IM. Opportunities for the use of lentiviral vectors in human gene therapy. Curr Top Microbiol Immunol 2002; 261: 245-254.
18. Trono D. Lentiviral vectors: Turning a deadly foe into a therapeutic agent. Gene Therapy 2000; 7: 20-23.
19. Seppen J, van der RR, Looije N, van Til NP, Lamers WH, Oude Elferink RP. Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer. Mol Ther 2003; 8: 593-599.
20. van Til NP, Markusic DM, van der Rijt R, Kunne C, Hiralall JK, Vreeling H et al. Kupffer cells and not liver sinusoidal endothelial cells prevent lentiviral transduction of hepatocytes. Mol Ther 2005; 11: 26-34.
21. Takahashi M, Ilan Y, Chowdhury NR, Guida J, Horwitz M, Chowdhury JR. Long term correction of bilirubin-UDP-glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period. J Biol Chem 1996; 271: 26536-26542.
22. Stripecke R, Villacres MD, Skelton DC, Satake N, Halene S, Kohn DB. Immune response to green fluorescent protein: Implications for gene therapy. Gene Therapy 1999; 6: 1305-1312.
23. Inoue H, Ohsawa I, Murakami T, Kimura A, Hakamata Y, Sato Y et al. Development of new inbred transgenic strains of rats with LacZ or GFP. Biochem Biophys Res Commun 2005; 329: 288-295.
24. Morris JC, Conerly M, Thomasson B, Storek J, Riddell SR, Kiem HP. Induction of cytotoxic T-lymphocyte responses to enhanced green and yellow fluorescent proteins after myeloablative conditioning. Blood 2004; 103: 492-499.
25. Kimura S, Eldridge JH, Michalek SM, Morisaki I, Hamada S, McGhee JR. Immunoregulation in the rat: Ontogeny of B cell responses to types 1, 2, and T-dependent antigens. J Immunol 1985; 134: 2839-2846.
26. Ladics GS, Smith C, Bunn TL, Dietert RR, Anderson PK, Wiescinski CM et al. Characterization of an approach to developmental immunotoxicology assessment in the rat using SRBC as the antigen. Toxicol Methods 2000; 10: 283-311.
27. Watts AM, Stanley JR, Shearer MH, Hefty PS, Kennedy RC. Fetal immunization of baboons induces a fetal-specific antibody response. Nat Med 1999; 5: 427-430.
28. Marchant A, Appay V, Van Der SM, Dulphy N, Liesnard C, Kidd M et al. Mature CD8(+) T lymphocyte response to viral infection during fetal life. J Clin Invest 2003; 111: 1747-1755.
29. Jerebtsova M, Batshaw ML, Ye X. Humoral immune response to recombinant adenovirus and adeno-associated virus after in utero administration of viral vectors in mice. Pediatr Res 2002; 52: 95-104.
30. Manns MP, Obermayer-Straub P. Cytochromes P450 and uridine triphosphate-glucuronosyltransferases: Model autoantigens to study drug-induced, virus-induced, and autoimmune liver disease. Hepatology 1997; 26: 1054-1066.
31. Clarke DJ, Keen JN, Burchell B. Isolation and characterisation of a new hepatic bilirubin UDP-glucuronosyltransferase. Absence from Gunn rat liver. FEBS Lett 1992; 299: 183-186.
32. Magdalou J, Antoine B, Ratanasavanh D, Siest G. Phenobarbital induction of cytochrome P-450 and UDP-glucuronosyltransferase in rabbit liver plasma membranes. Enzyme 1982; 28: 41-47.
33. Kootstra NA, Matsumura R, Verma IM. Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther 2003; 7: 623-631.
34. VandenDriessche T, Thorrez L, Naldini L, Follenzi A, Moons L, Berneman Z et al. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood 2002; 100: 813-822.
35. Follenzi A, Battaglia M, Lombardo A, Annoni A, Roncarolo MG, Naldini L. Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes longterm expression of human antihemophilic factor IX in mice. Blood 2004; 103: 3700-3709.
36. Nguyen TH, Bellodi-Privato M, Aubert D, Pichard V, Myara A, Trono D et al. Therapeutic lentivius-mediated neonatal in vivo gene therapy in hyperbilirubinemic Gunn rats. Mol Ther 2005 (in press).
37. Peters WH, Allebes WA, Jansen PL, Poels LG, Capel PJ. Characterization and tissue specificity of a monoclonal antibody against human uridine 5′-diphosphate-glucuronosyltransferase. Gastroenterology 1987; 93: 162-169.
38. Seppen J, Tada K, Ottenhoff R, Sengupta K, Chowdhury NR, Chowdhury JR et al. Transplantation of Gunn rats with autologous fibroblasts expressing bilirubin UDP-glucuronosyltransferase: Correction of genetic deficiency and tumor formation. Hum Gene Ther 1997; 8: 27-36.