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. 2000 Jan;46(1):136–139. doi: 10.1136/gut.46.1.136

Gene therapy of hepatic diseases: prospects for the new millennium

K SHETTY 1, G WU 1, C WU 1
PMCID: PMC1727783  PMID: 10601070

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Selected References

These references are in PubMed. This may not be the complete list of references from this article.

  1. Amalfitano A., McVie-Wylie A. J., Hu H., Dawson T. L., Raben N., Plotz P., Chen Y. T. Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase. Proc Natl Acad Sci U S A. 1999 Aug 3;96(16):8861–8866. doi: 10.1073/pnas.96.16.8861. [DOI] [PMC free article] [PubMed] [Google Scholar]
  2. Asahina Y., Ito Y., Wu C. H., Wu G. Y. DNA ribonucleases that are active against intracellular hepatitis B viral RNA targets. Hepatology. 1998 Aug;28(2):547–554. doi: 10.1002/hep.510280236. [DOI] [PubMed] [Google Scholar]
  3. Bilbao G., Feng M., Rancourt C., Jackson W. H., Jr, Curiel D. T. Adenoviral/retroviral vector chimeras: a novel strategy to achieve high-efficiency stable transduction in vivo. FASEB J. 1997 Jul;11(8):624–634. doi: 10.1096/fasebj.11.8.9240964. [DOI] [PubMed] [Google Scholar]
  4. Farhood H., Gao X., Barsoum J., Huang L. Codelivery to mammalian cells of a transcriptional factor with cis-acting element using cationic liposomes. Anal Biochem. 1995 Feb 10;225(1):89–93. doi: 10.1006/abio.1995.1112. [DOI] [PubMed] [Google Scholar]
  5. Fraefel C., Jacoby D. R., Lage C., Hilderbrand H., Chou J. Y., Alt F. W., Breakefield X. O., Majzoub J. A. Gene transfer into hepatocytes mediated by helper virus-free HSV/AAV hybrid vectors. Mol Med. 1997 Dec;3(12):813–825. [PMC free article] [PubMed] [Google Scholar]
  6. Grossman M., Rader D. J., Muller D. W., Kolansky D. M., Kozarsky K., Clark B. J., 3rd, Stein E. A., Lupien P. J., Brewer H. B., Jr, Raper S. E. A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia. Nat Med. 1995 Nov;1(11):1148–1154. doi: 10.1038/nm1195-1148. [DOI] [PubMed] [Google Scholar]
  7. Herz J., Gerard R. D. Adenovirus-mediated transfer of low density lipoprotein receptor gene acutely accelerates cholesterol clearance in normal mice. Proc Natl Acad Sci U S A. 1993 Apr 1;90(7):2812–2816. doi: 10.1073/pnas.90.7.2812. [DOI] [PMC free article] [PubMed] [Google Scholar]
  8. Hobbs H. H., Brown M. S., Goldstein J. L. Molecular genetics of the LDL receptor gene in familial hypercholesterolemia. Hum Mutat. 1992;1(6):445–466. doi: 10.1002/humu.1380010602. [DOI] [PubMed] [Google Scholar]
  9. Huber B. E., Richards C. A., Austin E. A. Virus-directed enzyme/prodrug therapy (VDEPT). Selectively engineering drug sensitivity into tumors. Ann N Y Acad Sci. 1994 May 31;716:104–143. doi: 10.1111/j.1749-6632.1994.tb21706.x. [DOI] [PubMed] [Google Scholar]
  10. Hurford R. K., Jr, Dranoff G., Mulligan R. C., Tepper R. I. Gene therapy of metastatic cancer by in vivo retroviral gene targeting. Nat Genet. 1995 Aug;10(4):430–435. doi: 10.1038/ng0895-430. [DOI] [PubMed] [Google Scholar]
  11. Ilan Y., Jona V. K., Sengupta K., Davidson A., Horwitz M. S., Roy-Chowdhury N., Roy-Chowdhury J. Transient immunosuppression with FK506 permits long-term expression of therapeutic genes introduced into the liver using recombinant adenoviruses in the rat. Hepatology. 1997 Oct;26(4):949–956. doi: 10.1002/hep.510260422. [DOI] [PubMed] [Google Scholar]
  12. Jaffe H. A., Danel C., Longenecker G., Metzger M., Setoguchi Y., Rosenfeld M. A., Gant T. W., Thorgeirsson S. S., Stratford-Perricaudet L. D., Perricaudet M. Adenovirus-mediated in vivo gene transfer and expression in normal rat liver. Nat Genet. 1992 Aug;1(5):372–378. doi: 10.1038/ng0892-372. [DOI] [PubMed] [Google Scholar]
  13. Kalpana G. V. Retroviral vectors for liver-directed gene therapy. Semin Liver Dis. 1999;19(1):27–37. doi: 10.1055/s-2007-1007095. [DOI] [PubMed] [Google Scholar]
  14. Kaneko S., Hallenbeck P., Kotani T., Nakabayashi H., McGarrity G., Tamaoki T., Anderson W. F., Chiang Y. L. Adenovirus-mediated gene therapy of hepatocellular carcinoma using cancer-specific gene expression. Cancer Res. 1995 Nov 15;55(22):5283–5287. [PubMed] [Google Scholar]
  15. Kren B. T., Bandyopadhyay P., Steer C. J. In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides. Nat Med. 1998 Mar;4(3):285–290. doi: 10.1038/nm0398-285. [DOI] [PubMed] [Google Scholar]
  16. Kren B. T., Cole-Strauss A., Kmiec E. B., Steer C. J. Targeted nucleotide exchange in the alkaline phosphatase gene of HuH-7 cells mediated by a chimeric RNA/DNA oligonucleotide. Hepatology. 1997 Jun;25(6):1462–1468. doi: 10.1002/hep.510250626. [DOI] [PubMed] [Google Scholar]
  17. Lee M. G., Abina M. A., Haddada H., Perricaudet M. The constitutive expression of the immunomodulatory gp19k protein in E1-, E3- adenoviral vectors strongly reduces the host cytotoxic T cell response against the vector. Gene Ther. 1995 Jun;2(4):256–262. [PubMed] [Google Scholar]
  18. Offensperger W. B., Offensperger S., Walter E., Teubner K., Igloi G., Blum H. E., Gerok W. In vivo inhibition of duck hepatitis B virus replication and gene expression by phosphorothioate modified antisense oligodeoxynucleotides. EMBO J. 1993 Mar;12(3):1257–1262. doi: 10.1002/j.1460-2075.1993.tb05767.x. [DOI] [PMC free article] [PubMed] [Google Scholar]
  19. Patijn G. A., Kay M. A. Hepatic gene therapy using adeno-associated virus vectors. Semin Liver Dis. 1999;19(1):61–69. doi: 10.1055/s-2007-1007098. [DOI] [PubMed] [Google Scholar]
  20. Patijn G. A., Lieber A., Schowalter D. B., Schwall R., Kay M. A. Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice. Hepatology. 1998 Sep;28(3):707–716. doi: 10.1002/hep.510280317. [DOI] [PubMed] [Google Scholar]
  21. Raper S. E., Wilson J. M., Yudkoff M., Robinson M. B., Ye X., Batshaw M. L. Developing adenoviral-mediated in vivo gene therapy for ornithine transcarbamylase deficiency. J Inherit Metab Dis. 1998;21 (Suppl 1):119–137. doi: 10.1023/a:1005369926784. [DOI] [PubMed] [Google Scholar]
  22. Santoro S. W., Joyce G. F. A general purpose RNA-cleaving DNA enzyme. Proc Natl Acad Sci U S A. 1997 Apr 29;94(9):4262–4266. doi: 10.1073/pnas.94.9.4262. [DOI] [PMC free article] [PubMed] [Google Scholar]
  23. Smith R. M., Wu G. Y. Hepatocyte-directed gene delivery by receptor-mediated endocytosis. Semin Liver Dis. 1999;19(1):83–92. doi: 10.1055/s-2007-1007100. [DOI] [PubMed] [Google Scholar]
  24. Strayer D. S., Zern M. A. Gene delivery to the liver using simian virus 40-derived vectors. Semin Liver Dis. 1999;19(1):71–81. doi: 10.1055/s-2007-1007099. [DOI] [PubMed] [Google Scholar]
  25. Tada K., Roy-Chowdhury N., Prasad V., Kim B. H., Manchikalapudi P., Fox I. J., van Duijvendijk P., Bosma P. J., Roy-Chowdhury J. Long-term amelioration of bilirubin glucuronidation defect in Gunn rats by transplanting genetically modified immortalized autologous hepatocytes. Cell Transplant. 1998 Nov-Dec;7(6):607–616. doi: 10.1177/096368979800700611. [DOI] [PubMed] [Google Scholar]
  26. VandenDriessche T., Vanslembrouck V., Goovaerts I., Zwinnen H., Vanderhaeghen M. L., Collen D., Chuah M. K. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proc Natl Acad Sci U S A. 1999 Aug 31;96(18):10379–10384. doi: 10.1073/pnas.96.18.10379. [DOI] [PMC free article] [PubMed] [Google Scholar]
  27. Verma I. M., Somia N. Gene therapy -- promises, problems and prospects. Nature. 1997 Sep 18;389(6648):239–242. doi: 10.1038/38410. [DOI] [PubMed] [Google Scholar]
  28. Wang L., Takabe K., Bidlingmaier S. M., Ill C. R., Verma I. M. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc Natl Acad Sci U S A. 1999 Mar 30;96(7):3906–3910. doi: 10.1073/pnas.96.7.3906. [DOI] [PMC free article] [PubMed] [Google Scholar]
  29. Wilson J. M., Chowdhury N. R., Grossman M., Wajsman R., Epstein A., Mulligan R. C., Chowdhury J. R. Temporary amelioration of hyperlipidemia in low density lipoprotein receptor-deficient rabbits transplanted with genetically modified hepatocytes. Proc Natl Acad Sci U S A. 1990 Nov;87(21):8437–8441. doi: 10.1073/pnas.87.21.8437. [DOI] [PMC free article] [PubMed] [Google Scholar]
  30. Wu C. H., Wu G. Y. Targeted inhibition of hepatitis C virus-directed gene expression in human hepatoma cell lines. Gastroenterology. 1998 Jun;114(6):1304–1312. doi: 10.1016/s0016-5085(98)70437-8. [DOI] [PubMed] [Google Scholar]
  31. Wu G. Y., Wu C. H. Specific inhibition of hepatitis B viral gene expression in vitro by targeted antisense oligonucleotides. J Biol Chem. 1992 Jun 25;267(18):12436–12439. [PubMed] [Google Scholar]
  32. Yang Y., Li Q., Ertl H. C., Wilson J. M. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J Virol. 1995 Apr;69(4):2004–2015. doi: 10.1128/jvi.69.4.2004-2015.1995. [DOI] [PMC free article] [PubMed] [Google Scholar]
  33. Yang Y., Nunes F. A., Berencsi K., Gönczöl E., Engelhardt J. F., Wilson J. M. Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nat Genet. 1994 Jul;7(3):362–369. doi: 10.1038/ng0794-362. [DOI] [PubMed] [Google Scholar]
  34. Yoon K., Cole-Strauss A., Kmiec E. B. Targeted gene correction of episomal DNA in mammalian cells mediated by a chimeric RNA.DNA oligonucleotide. Proc Natl Acad Sci U S A. 1996 Mar 5;93(5):2071–2076. doi: 10.1073/pnas.93.5.2071. [DOI] [PMC free article] [PubMed] [Google Scholar]
  35. Zhang H., Hanecak R., Brown-Driver V., Azad R., Conklin B., Fox M. C., Anderson K. P. Antisense oligonucleotide inhibition of hepatitis C virus (HCV) gene expression in livers of mice infected with an HCV-vaccinia virus recombinant. Antimicrob Agents Chemother. 1999 Feb;43(2):347–353. doi: 10.1128/aac.43.2.347. [DOI] [PMC free article] [PubMed] [Google Scholar]

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