SEARCH

SEARCH BY CITATION

References

  • 1
    Ritter JK, Chen F, Sheen YY, Tran HM, Kimura S, Yeatman MT, et al. A novel complex locus UGT encodes human bilirubin phenol and other UDP-glucuronosyltransferase isozymes with identical carboxyl termini. J Biol Chem 1992; 267: 32573261.
  • 2
    King CD, Rios GR, Green MD, Tephly TR. UDP-Glucuronosyltransferases. Curr Drug Metab 2000; 1: 143161.
  • 3
    Gunn CK. Hereditary acholuric jaundice in a new mutant strain of rat. J Hered 1939; 29: 137139.
  • 4
    Iyanagi T, Watanabe T, Uchiyama Y. The 3-methylcholanthrene-inducible UDP-glucuronosyltransferase deficiency in the hyperbilirubinemic rat (Gunn rat) is caused by a -1 frameshift mutation. J Biol Chem 1989; 264: 2130221307.
  • 5
    Roy-Chowdhury J, Huang TJ, Kesari 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: 1829418298.
  • 6
    Sauter BV, Parashar B, Chowdhury NR, Kadakol A, Ilan Y, Singh H, et al. A replication-deficient rSV40 mediates liver-directed gene transfer and a long-term amelioration of jaundice in gunn rats. Gastroenterology 2000; 119: 13481357.
  • 7
    Tada K, Chowdhury NR, Neufeld D, Bosma PJ, Heard M, Prasad VR, et al. Long-term reduction of serum bilirubin levels in Gunn rats by retroviral gene transfer in vivo. Liver Transpl Surg 1998; 4: 7888.
  • 8
    Wilke M, Bijma A, Timmers-Reker AJ, Scholte BJ, Sinaasappel M. Complementation of the genetic defect in Gunn rat hepatocytes in vitro by highly efficient gene transfer with cationic liposomes. Gene Ther 1997; 4: 13051312.
  • 9
    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: 388396.
  • 10
    Seppen J, van der Rijt R, 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: 593599.
  • 11
    Askari FK, Hitomi Y, Mao M, Wilson JM. Complete correction of hyperbilirubinemia in the Gunn rat model of Crigler-Najjar syndrome type I following transient in vivo adenovirus- mediated expression of human bilirubin UDP-glucuronosyltransferase. Gene Ther 1996; 3: 381388.
  • 12
    Thummala NR, Ghosh SS, Lee SW, Reddy B, Davidson A, Horwitz MS, et al. A non-immunogenic adenoviral vector, coexpressing CTLA4Ig and bilirubin-uridine-diphosphoglucuronateglucuronosyltransferase permits long-term, repeatable transgene expression in the Gunn rat model of Crigler-Najjar syndrome. Gene Ther 2002; 9: 981990.
  • 13
    Ilan Y, Attavar P, Takahashi M, Davidson A, Horwitz MS, Guida J, et al. 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: 26402647.
  • 14
    Ilan Y, Jona VK, Sengupta K, Davidson A, Horwitz MS, Roy-Chowdhury N, et al. Transient immunosuppression with FK506 permits long-term expression of therapeutic genes introduced into the liver using recombinant adenoviruses in the rat. HEPATOLOGY 1997; 26: 949956.
  • 15
    Ilan Y, Prakash R, Davidson A, Jona, Droguett G, Horwitz MS, et al. Oral tolerization to adenoviral antigens permits long-term gene expression using recombinant adenoviral vectors. J Clin Invest 1997; 99: 1098106.
  • 16
    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 helper-dependent adenoviral vector. Proc Natl Acad Sci U S A 2005; 102: 39303935.Epub 2005;Mar 7.
  • 17
    Ponder KP, Melniczek JR, Xu L, Weil MA, O'Malley TM, O'Donnell PA, et al. Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs. Proc Natl Acad Sci U S A 2002; 99: 1310213107.
  • 18
    Xu L, Gao C, Sands MS, Cai SR, Nichols TC, Bellinger DA, et al. Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B. Blood 2003; 101: 39243932.
  • 19
    Seppen J, Bosma PJ, Goldhoorn BG, Bakker CTM, Chowdhury JR, Chowdhury NR, et al. Discrimination between Crigler-Najjar type I and II by expression of mutant bilirubin uridine diphosphate-glucuronosyltransferase. J Clin Invest 1994; 94: 23852391.
  • 20
    Ory DS, Neugeboren BA, Mulligan RC. A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes. Proc Natl Acad Sci U S A 1996; 93: 1140011406.
  • 21
    Muraca M, Blanckaert N. Liquid-chromatographic assay and identification of mono- and diester conjugates of bilirubin in normal serum. Clin Chem 1983; 29: 17671771.
  • 22
    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: 162169.
  • 23
    Bellodi-Privato M, Le Meur G, Aubert D, Mendes-Madera A, Pichard V, Rolling F, et al. AAV gene transfer to the retina does not protect retrovirally transduced hepatocytes from the immune response. J Mol Med 2004; 82: 403410.
  • 24
    Bralet MP, Branchereau S, Brechot C, Ferry N. Cell lineage study in the liver using retroviral mediated gene transfer: evidence against the streaming of hepatocytes in normal liver. Am J Pathol 1994; 144: 896905.
  • 25
    Ilan Y, Roy-Chowdhury N, Prakash R, Jona V, Attavar P, Guha C, et al. Massive repopulation of rat liver by transplantation of hepatocytes into specific lobes of the liver and ligation of portal vein branches to other lobes. Transplantation 1997; 64: 813.
  • 26
    Johnson-Delaney C. Exotic Animal Companion Medicine Handbook for Veterinarians. Lake Worth, FL: Zoological Education Network, 1996.
  • 27
    Van Steenbergen W, Fevery J. Effects of uridine diphosphate glucuronosyltransferase activity on the maximal secretion rate of bilirubin conjugates in the rat. Gastroenterology 1990; 99: 488499.
  • 28
    Kitten O, Cosset FL, Ferry N Highly efficient retrovirus-mediated gene transfer into rat hepatocytes in vivo. Hum Gene Ther 1997; 8: 14911494.
  • 29
    Fraser R, Le Couteur DG, Warren A, Cogger VC, Bertolino P, Smith M. The liver sieve and gene therapy. Blood 2003; 101: 3338; author reply 3338–3339.
  • 30
    Fox IJ, Chowdhury JR, Kaufman SS, Goertzen TC, Chowdhury NR, Warkentin PI, et al. Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation. N Engl J Med 1998; 338: 14221426.
  • 31
    Challita PM, Kohn DB. Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc Natl Acad Sci U S A 1994; 91: 25672571.
  • 32
    Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 415419.