Long-term reduction of serum bilirubin levels in gunn rats by retroviral gene transfer in vivo

Liver Transplantation and Surgery

Volumn 4, Issue 1, 1998, Pages 78-88

  Tada, K ^a   Chowdhury, N R ^a   Neufeld, D ^a   Bosma, P J ^a   Heard, M ^a   Prasad, V R ^a   Chowdhury, J R ^a  

^a ALBERT EINSTEIN COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE; BILIRUBIN; BILIRUBIN GLUCURONIDE; GLUCURONOSYLTRANSFERASE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BILIRUBIN BINDING; BILIRUBIN BLOOD LEVEL; CONTROLLED STUDY; CRIGLER NAJJAR SYNDROME; ENZYME ACTIVITY; ESCHERICHIA COLI; FEMALE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HYPERBILIRUBINEMIA; IMMUNOBLOTTING; IMMUNOFLUORESCENCE TEST; MALE; NONHUMAN; PARTIAL HEPATECTOMY; PRIORITY JOURNAL; RAT; RETROVIRUS; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; REVERSED PHASE HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; TRANSGENE; VIRUS RECOMBINANT;

EID: 0031973035     PISSN: 10743022     EISSN: None     Source Type: Journal    
DOI: 10.1002/lt.500040111     Document Type: Article

References (44)

1. 1 Roy Chowdhury J, Wolkoff AW, Roy Chowdhury N, Arias IM. Hereditary jaundice and disorders of bilirubin metabolism. In: Scriver, CR Beaudet, AL Sly, WS Valle, D eds. The metabolic and molecular bases of inherited diseaseed 7 New York: McGraw‐Hill. 1995 ; 2161–2210.
2. 2 Ritter JK, Crawford JM, Owens IS. Cloning of two human liver bilirubin‐UDP‐glucuronosyltransferase cDNAs with expression in COS‐1 cells. J Biol Chem 1991 ; 266: 1043–1047. MEDLINE
3. 3 Bosma PJ, Seppen J, Goldhoorn, Bakker C, Oude Elferink RPJ, Roy Chowdhury J, Roy Chowdhury N, Jansen PLM. Bilirubin UDP glucuronosyltransferase‐1 is the only relevant bilirubin glucuronidating isoform in man. J Biol Chem 1994 ; 269: 17960–17964. MEDLINE
4. 4 Jansen PLM, Bosma PJ, Roy Chowdhury J. Molecular biology of bilirubin metabolism. Prog Liver Dis 1995 ; 13: 125–150. MEDLINE
5. 5 Crigler JF, Najjar VA. Congenital familial non‐hemolytic jaundice with kernicterus. Pediatrics 1952 ; 10: 169–180.
6. 6 Roy Chowdhury N, Kondapalli R, Roy Chowdhury J. The Gunn rat: an animal model for inherited deficiency of bilirubin glucuronidation. In: Cornelius, CE eds. Animal models in liver research. New York: Academic. 1993 ; 150–175.
7. 7 Arias IM, Gartner LM, Cohen M, Benezzer J, Levi AJ. Chronic nonhemolytic unconjugated hyperbilirubinemia with glucuronyl transferase deficiency: clinical biochemical, pharmacological and genetic evidence for heterogeneity. Am J Med 1969 ; 47: 395–402. MEDLINE
8. 8 Seppen J, Bosma PJ, Roy Chowdhury J, Roy Chowdhury N, Jansen PLM, Oude Elferink RPJ. Discrimination between Crigler Najjar syndrome type I and II by expression of mutant bilirubin‐UDP‐glucuronosyltransferase. J Clin Invest 1994 ; 94: 2385–2391. MEDLINE
9. 9 Emi Y, Ikushiro S, Iyanagi T. Drug‐responsive and tissue‐specific alternative expression of multiple first exons in rat UDP‐glucuronosyltransferase family 1 (UGT1) gene complex. J Biochem 1995 ; 117: 392–399. MEDLINE
10. 10 Roy Chowdhury J, Huang TJ, Kasari K, Lederstein M, Arias IM, Roy Chowdhury N. Molecular basis for the lack of bilirubin‐specific and 3‐methylcholanthrene‐inducible UDP‐glucuronosyltransferase activities in Gunn rats: the two isoforms are encoded by distinct mRNA species that share an identical single‐base deletion. J Biol Chem 1991 ; 266: 18294–18298. MEDLINE
11. 11 ElAwady M, Roy Chowdhury J, Kesari K, van Es HHG, Lederstein M, Arias IM, Roy Chowdhury N. Mechanism of the lack of induction of UDP‐glucuronosyltransferase activity by 3‐methylcholanthrene in Gunn rats. J Biol Chem 1990 ; 265: 10752–10758. MEDLINE
12. 12 Roy Chowdhury J, van Es HHG, Roy Chowdhury N. Gunn rat. An animal model for deficiency of bilirubin conjugation. In: Tavaloni, N Berk, PD eds. Hepatic transport and bile secretion: physiology and pathophysiology. New York: Raven. 1993 ; 713–719.
13. 13 Demetriou AA, Whiting JF, Feldman D, Levenson SM, Roy Chowdhury N, Moscioni AD, Kram M, Roy Chowdhury J. Replacement of liver function in rats by transplantation of microcarrier‐attached hepatocytes. Science 1986 ; 233: 1190–1192. MEDLINE
14. 14 Danos O, Mulligan RC. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc Natl Acad Sci USA 1988 ; 85: 6460–6464. MEDLINE
15. 15 Dranoff G, Jaffee E, Lazenby A, Golumbek P, Levitsky H, Brose K, Jackson V, Hamada H, Pardoll D, Mulligan RC. Vaccinaton with irradiated tumor cells engineered to secrete murine granulocyte macrophage colony stimulating factor stimulates potent, specific, and long‐lasting anti‐tumor immunity. Proc Natl Acad Sci USA 1993 ; 90: 3539–3543. MEDLINE
16. 16 Fox IR, Roy Chowdhury N, Gupta S, Kondapalli R, Schilsky ML, Stockert RJ, Roy Chowdhury J. Conditional immortalization of Gunn rat hepatocytes: an ex vivo model for evaluating methods for bilirubin‐UDP‐glucuronosyltransferase gene transfer. Hepatology 1995 ; 21: 837–846. MEDLINE
17. 17 Seppen J, Tada K, Hellwig S, Bakker CTM, Prasad VR, Roy Chowdhury N, Roy Chowdhury JR, Bosma PJ, Oude Elferink RPJ. Bilirubin glucuronidation by intact Gunn rat fibroblasts expressing bilirubin UDP‐glucuronosyltransferase. Biochem J 1996 ; 314: 477–483. MEDLINE
18. 18 Bestwick RK, Kozak SL, Kabat D. Overcoming interference to retroviral superinfection results in amplified expression and transmission of cloned genes. Proc Natl Acad Sci USA 1988 ; 85: 5404–5408. MEDLINE
19. 19 Kozak SL, Kabat D. Ping‐pong amplification of a retroviral vector achieves high‐level gene expression: human growth hormone production. J Virol 1990 ; 64: 3500–3508. MEDLINE
20. 20 Wan AM, Doyle MV, Mark DF. Quantitation of mRNA by the polymerase chain reaction. Proc Natl Acad Sci USA 1989 ; 86: 9717–9721. MEDLINE
21. 21 Goff SP, Traktman P, Baltimore D. Isolation and properties of Moloney murine leukemia virus mutants: use of a rapid assay for release of virion reverse transcriptase. J Virol 1981 ; 38: 239–248. MEDLINE
22. 22 Higgins GM, Anderson RM. Experimental pathology of the liver 1. Restoration of the liver of the white rat following partial surgical removal. Arch Pathol 1931 ; 12: 186–202.
23. 23 Ferry N, Duplessis O, Houssin D, Danos O, Heard JM. Retroviral‐mediated gene transfer into hepatocytes in vivo. Proc Natl Acad Sci USA 1990 ; 88: 8377–8381.
24. 24 Takahashi M, Ilan Y, Roy Chowdhury N, Guida J, Horwitz MS, Roy Chowdhury J. 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: 26534–26536.
25. 25 Peters WHM, Allebes WA, Jansen PLM, Poeis LG, Capel PJA. Characterization and tissue specificity of a monoclonal antibody against human uridine 5′‐diphosphate‐glucuronosyltransferase. Gastroenterology 1987 ; 93: 162–169. MEDLINE
26. 26 Towbin H, Staehlin T, Gordon J. Electrophoretic transfer of proteins from polyacrylamide gels to nitrocellulose sheets: procedure and some applications. Proc Natl Acad Sci USA 1979 ; 76: 4350–4354. MEDLINE
27. 27 Roy Chowdhury J, Roy Chowdhury N, Gartner U, Wolkoff AW, Arias IM. Bilirubin diglucuronide formation in intact rats and in isolated Gunn rat liver. J Clin Invest 1982 ; 69: 595–603. MEDLINE
28. 28 Jendrassik L, Grof P. Vereinfachte Photometrische Methoden zur Bestimmung des Blutbilirubins. Biochim Z 1938 ; 297: 81–89.
29. 29 Roy Chowdhury J, Novikoff PM, Roy Chowdhury N, Novikoff AB. Distribution of uridine diphosphoglucuronate glucuronosyl transferase in rat tissues. Proc Natl Acad Sci USA 1985 ; 82: 2990–2994. MEDLINE
30. 30 Roy Chowdhury N, Gross F, Moscioni AD, Kram M, Arias IM, Roy Chowdhury J. Isolation and purification of multiple normal and functionally defective forms of UDP‐glucuronosyltransferase from livers of inbred Gunn rats. J Clin Invest 1987 ; 79: 327–334. MEDLINE
31. 31 Bosma PJ, Roy Chowdhury J, Huang TJ, Lahiri P, Oude Elferink RPJ, van Es HHG, Lederstein M, Whitington PF, Jansen PLM, Roy Chowdhury N. Mechanism of inherited deficiencies of multiple UDP‐glucuronosyltransferase isoforms in two patients with Crigler‐Najjar syndrome, type I. FASEB J 1992 ; 6: 2859–2863. MEDLINE
32. 32 Bosma PJ, Roy Chowdhury N, Goldhoorn BG, Hofker MH, Oude Elferink RPJ, Jansen PLM, Roy Chowdhury J. Sequence of exons and the flanking regions of human bilirubin‐UDP‐glucuronosyltransferase gene complex and identification of a genetic mutation in a patient with Crigler‐Najjar syndrome, type I. Hepatology 1992 ; 15: 941–947. MEDLINE
33. 33 Ritter JK, Yeatman MT, Ferriera P, Owens IS. Identification of a genetic alteration in the code for bilirubin UDP‐glucuronosyltransferase in the UGT‐1 gene complex of a Crigler‐Najjar syndrome, type 1 patient. J Clin Invest 1992 ; 90: 150–155. MEDLINE
34. 34 Muenchau DD, Freeman SM, Cornetta K, Zwiebel JA, Anderson WF. Analysis of retroviral packaging lines for generation of replication‐competent virus. Virology 1990 ; 176: 262–265. MEDLINE
35. 35 Kay MA, Baley P, Rothenberg S, Leland F, Fleming L, Ponder KP, Liu T, Finegold M, Darlington G, Pokorny W, Woo SLC. Expression of human a 1 ‐antitrypsin in dogs after autologous transplantation of retroviral transducted hepatocytes. Proc Natl Acad Sci USA 1992 ; 8: 89–93.
36. 36 Koeberl DD, Halbert CL, Krumm A, Miller AD. Sequences within the coding regions of clotting factor VIII and CFTR block transcriptional elongation. Hum Gene Ther 1995 ; 6: 469–479. MEDLINE
37. 37 Bralet MP, Branchereau S, Brechot C, Ferry N. Cell lineage study in the liver using retroviral mediated gene transfer. Am J Pathol 1994 ; 144: 896–905. MEDLINE
38. 38 Branchereau S, Calise D, Ferry N. Factors influencing retroviral‐mediated gene transfer into hepatocytes in vivo. Hum Gene Ther 1994 ; 5: 803–808. MEDLINE
39. 39 Vemuru RP, Davidson A, Aragona E, Burk RD, Roy Chowdhury J, Gupta S. Immune tolerance to a defined heterologous antigen following intrasplenic hepatocyte transplantation: implications for human gene therapy. FASEB J 1992 ; 6: 2836–2842. MEDLINE
40. 40 Wilson JM, Wu GY, Wu CH, Grossman M, Roy Chowdhury N, Roy Chowdhury J. Hepatocyte‐directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in LDL‐receptor‐deficient rabbits. J Biol Chem 1992 ; 267: 963–967. MEDLINE
41. 41 Roy Chowdhury N, Wu CH, Wu GY, Yerneni PC, Bommineni VR, Roy Chowdhury J. Fate of DNA targeted to the liver by asialoglycoprotein receptor‐mediated endocytosis in vivo: prolonged persistence in cytoplasmic vesicles after partial hepatectomy. J Biol Chem 1993 ; 268: 11265–11271. MEDLINE
42. 42 Bommineni VR, Roy Chowdhury N, Wu GY, Wu CH, Franki N, Hays RM, Roy Chowdhury J. Depolymerization of hepatocellular microtubules after partial hepatectomy. J Biol Chem 1994 ; 269: 25200–25205. MEDLINE
43. 43 Ilan Y, Attavar P, Takahashi M, Davidson A, Horwitz M, Guida J, Roy Chowdhury N, Roy Chowdhury J. Induction of central tolerance by intrathymic inoculation of adenoviral antigens into the host thymus permits long‐term gene therapy in Gunn rats. J Clin Invest 1996 ; 98: 2640–2647. MEDLINE
44. 44 Ilan Y, Prakash R, Davidson A, Jona V, Droguett G, Horwitz MS, Roy Chowdhury N, Roy Chowdhury J. Oral tolerization to adenoviral antigens permits long‐term gene expression using recombinant adenoviral vectors. J Clin Invest 1997 ; 99: 1098–1106. MEDLINE