The Liver as a Target for Gene Therapy

Concepts in Gene Therapy

Volumn , Issue , 2020, Pages 267-281

  Cichon, Günter ^a   Strauss, Michael ^a,b  

^a HUMBOLDT UNIVERSITY   (Germany)

^b DANISH CANCER SOCIETY RESEARCH CENTER   (Denmark)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 85134287629     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1515/9783110808636-014     Document Type: Chapter

References (58)

1. Miller, A. D. (1992). Retroviral vectors. Curr. Top. Microbiol. Immunol. 158, 1-24.
2. Naviaux, R. K. and Verma, I. M. (1992). Retroviral vectors for persistent expression in vivo. Curr. Opin. Biotechnol. 3, 540-547.
3. Gilboa, E. (1990). Retroviral gene transfer: applications to human therapy. Prog. Clin. Biol. Res. 352, 301-311.
4. Russell, D. W., Berger, M. S., and Miller, A. D. (1995). The effects of human serum and cerebrospinal fluid on retroviral vectors and packaging cell lines.. Hum. Gene Ther. 6, 635-641.
5. Bukrinsky, M. I., Haggerty, S., Dempsey, M. P., Sharova, N., Adzhubel, A., Spitz, L., Lewis, P., Goldfarb, D., Emerman, M., and Stevenson, M. (1993). A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells [see comments]. Nature 365, 666-669.
6. Chowdhury, J. R., Grossman, M., Gupta, S., Chowdhury, N. R., Baker, J. R., Jr., and Wilson, J. M. (1991). Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLRdeficient rabbits. Science 254, 1802-1805.
7. Kay, M. A., Rothenberg, S., Landen, C. N., Bellinger, D. A., Leland, F., Toman, C., Finegold, M., Thompson, A. R., Read, M. S., Brinkhous, K. M., and et al. (1993). In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs [see comments]. Science 262, 117-119.
8. Grossman, M., Raper, S. E., Kozarsky, K., Stein, E. A., Engelhardt, J. F., Muller, D., Lupien, P. J., and Wilson, J. M. (1994). Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia [see comments], Nat. Genet. 6, 335-341.
9. Wilson, J. M., Grossman, M., Raper, S. E., Baker, J. R., Jr., Newton, R. S., and Thoene, J. G. (1992). Ex vivo gene therapy of familial hypercholesterolemia. Hum. Gene Ther. 3, 179-222.
10. 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., and et al. (1995). A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia [see comments]. Nat. Med. 1, 1148-1154.
11. Lieber, A., Peeters, M. J., Gown, A., Perkins, J., and Kay, M. A. (1995). A modified urokinase plasminogen activator induces liver regeneration without bleeding. Hum. Gene Ther. 6, 1029-1037.
12. Krougliak, V. and Graham, F. L. (1995). Development of cell lines capable of completing El, E4, and Protein IX defective adenovirus type 5 mutants. Human Gene Therapy 6, 1575-1586.
13. Wang, Q., Jia, X.-C., and Finer, M. H. (1995). A packaging cell line for propagation of recombinant adenovirus vectors containing two lethal gene-region deletions. Gene Therapy 2, 775-783.
14. Kremer, E. J. and Perricaudet, M. (1995). Adenovirus and adeno-associated virus mediated gene transfer. Brit. Med. Bul. 51(1), 31-44.
15. Smith, T. A., Mehaffey, M. G., Kayda, D. B., Saunders, J. M., Yei, S., Trapnell, B. C., McClelland, A., and Kaleko, M. (1993). Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice. Nat. Genet. 5, 397-402.
16. Herz, J. and Gerard, R. D. (1993). Adenovirus-mediated transfer of low density lipoprotein receptor gene acutely accelerates cholesterol clearance in normal mice. Proc. Natl. Acad. Sci. U.S.A. 90, 2812-2816.
17. Mathias, P., Wickham, T., Moore, M., and Nemerow, G. (1994). Multiple adenovirus serotypes use alpha v integrins for infection. J. Virol. 68, 6811-6814.
18. Yang, Y., Nunes, F. A., Berencsi, K., Gonczol, E., Engelhardt, J. F., and Wilson, J. M. (1994b). Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nat. Genet. 7, 362-369.
19. Barr, D., Tubb, J., Ferguson, D., Scaria, A., Lieber, A., Wilson, C., Perkins, J., and Kay, M. A. (1995). Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains. Gene Ther. 2, 151-155.
20. Li, J., Fang, B., Eisensmith, R. C., Li, X. H., Nasonkin, I., Lin Lee, Y. C., Mims, M. P., Hughes, A., Montgomery, C. D., Roberts, J. D., and et al. (1995). In vivo gene therapy for hyperlipidemia: phenotypic correction in Watanabe rabbits by hepatic delivery of the rabbit LDL receptor gene. J. Clin. Invest. 95, 768-773.
21. Fang, B., Eisensmith, R. C., Wang, H., Kay, M. A., Cross, R. E., Landen, C. N., Gordon, G., Bellinger, D. A., Read, M. S., Hu, P. C., and et al. (1995). Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression. Hum. Gene Ther. 6, 1039-1044.
22. Ishibashi, S., Brown, M. S., Goldstein, J. L., Gerard, R. D., Hammer, R. E., and Herz, J. (1993). Hypercholesterolemia in low density lipoprotein receptor knockout mice and its reversal by adenovirusmediated gene delivery [see comments]. J. Clin. Invest. 92, 883-893.
23. Yang, Y., Erti, H. C., and Wilson, J. M. (1994a). MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with El-deleted recombinant adenoviruses. Immunity. 1, 433-442.
24. Yeh, P., Dedieu, J. F., Orsini, C., Vigne, E., Denefle, P., and Perricaudet, M. (1996) Efficient dual transcomplementation of adenovirus El and E4 regions from a 293-derived cell line expressing a minimal E4 functional unit. J. Virol. 70(1), 559-65.
25. Weitzman, M. D., Kyostio, S. R., Kotin, R. M., and Owens, R. A. (1994). Adeno-associated virus (AAV) Rep proteins mediate complex formation between AAV DNA and its integration site in human DNA. Proc. Natl. Acad. Sci. U.S.A. 91, 5808-5812.
26. McCarty, D. M., Ryan, J. H., Zolotukhin, S., Zhou, X., and Muzyczka, N. (1994b). Interaction of the adeno-associated virus Rep protein with a sequence within the A palindrome of the viral terminal repeat. J. Virol. 68, 4998-5006.
27. McCarty, D. M., Pereira, D. J., Zolotukhin, I., Zhou, X., Ryan, J. H., and Muzyczka, N. (1994a). Identification of linear DNA sequences that specifically bind the adeno-associated virus Rep protein. J. Virol. 68, 4988-4997.
28. Flotte, T. R. (1992). Gene expression from adeno-associated virus vectors in airway epithelial cells. Am. J. Respir. Cell. Mol. Biol. 7, 349-356.
29. Flotte, T. R., Afione, S. A., Conrad, C., McGrath, S. A., Solow, R., Oka, H., Zeitlin, P. L., Guggino, W. B., and Carter, B. J. (1993). Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. U.S.A. 90, 10613-10617.
30. Miyanohara, A., Johnson, P. A., Elam, R. L., Dai, Y., Witztum, J. L., Verma, I. M., and Friedmann, T. (1992). Direct gene transfer to the liver with herpes simplex virus type 1 vectors: transient production of physiologically relevant levels of circulating factor IX. New Biol. 4, 238-246.
31. Fong, Y. M., Federoff, H. J., Brownlee, M., Blumberg, D., Blumgart, L. H., and Brennan, M. F. (1995). Rapid and efficient gene transfer in human hepatocytes by herpes viral vectors. Hepatology 22, 723-729.
32. Roemer, K., Johnson, P. A., and Friedmann, T. (1992). Recombination between a herpes simplex virus type 1 vector deleted for immediate early gene 3 and the infected cell genome. J. Gen. Virol. 73, 1553-1558.
33. Wagner, E., Cotten, M., Foisner, R., and Birnstiel, M. L. (1991). Transferrin-polycation-DNA complexes: the effect of polycations on the structure of the complex and DNA delivery to cells. Proc. Natl. Acad. Sci. U.S.A. 88, 4255-4259.
34. Lozier, J. N., Thompson, A. R., Hu, P. C., Read, M., Brinkhous, K. M., High, K. A., and Curiel, D. T. (1994). Efficient transfection of primary cells in a canine hemophilia B model using adenoviruspolylysine-DNA complexes. Hum. Gene Ther. 5, 313-322.
35. Wu, G. Y. and Wu, C. H. (1988). Receptor-mediated gene delivery and expression in vivo. J. Biol. Chem. 263, 14621-14624.
36. Tietz, P. S., Yamazaki, K., and LaRusso, N. F. (1990). Time-dependent effects of chloroquine on pH of hepatocyte lysosomes. Biochem. Pharmacol. 40, 1419-1421.
37. Seth, P. (1994). Adenovirus-dependent release of choline from plasma membrane vesicles at an acidic pH is mediated by the penton base protein. J. Virol. 68, 1204-1206.
38. Wagner, E., Plank, C., Zatloukal, K., Cotten, M., and Birnstiel, M. L. (1992a). Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine-DNA complexes: toward a synthetic virus-like gene-transfer vehicle. Proc. Natl. Acad. Sci. U.S.A. 89, 7934-7938.
39. Wagner, E., Zatloukal, K., Cotten, M., Kirlappos, H., Mechtler, K., Curiel, D. T., and Birnstiel, M. L. (1992b). Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc. Natl. Acad. Sci. U.S.A. 89, 6099-6103.
40. Kaneda, Y., Iwai, K., and Uchida, T. (1989). Increased expression of DNA cointroduced with nuclear protein in adult rat liver. Science 243, 375-378.
41. Hafenrichter, D. G., Ponder, K. P., Rettinger, S. D., Kennedy, S. C., Wu, X., Saylors, R. S., and Flye, M. W. (1994). Liver-directed gene therapy: evaluation of liver specific promoter elements. J. Surg. Res. 56, 510-517.
42. Adams, T. E. (1995). Differential expression of growth hormone receptor messenger RNA from a second promoter. Mol. Cell Endocrinol. 108, 23-33.
43. Boquet, D., Vaulont, S., Tremp, G., Ripoche, M. A., Daegelen, D., Jami, J., Kahn, A., and Raymondjean, M. (1992). DNase-I hypersensitivity analysis of the L-type pyruvate kinase gene in rats and transgenic mice. Eur. J. Biochem. 207, 13-21.
44. Miao, C. H., Leytus, S. P., Chung, D. W., and Davie, E. W. (1992). Liver-specific expression of the gene coding for human factor X, a blood coagulation factor. J. Biol. Chem. 267, 7395-7401.
45. Loser, L., Sandig, V., Kirillova, I., Strauss, M. (1996). Evaluation of HBV promoters for use in hepatic gene therapy. Biol. Chem. Hoppe-Seyler, 377, 187-193.
46. Lutfalla, G., Armbruster, L., Dequin, S., and Bertolotti, R. (1989). Construction of an EBNAproducing line of well-differentiated human hepatoma cells and of appropriate Epstein-Barr virusbased shuttle vectors. Gene 76, 27-39.
47. Wilson, J. and Levine, A. (1993). Curr. Top. Immunol. Microbiol.
48. Thierry, A. R., Lunardi Iskandar, Y., Bryant, J. L., Rabinovich, P., Gallo, R. C., and Mahan, L. C. (1995). Systemic gene therapy: biodistribution and long-term expression of a transgene in mice. Proc. Natl. Acad. Sci. U.S.A. 92, 9742-9746.
49. Kay, M. A., Holterman, A. X., Meuse, L., Gown, A., Ochs, H. D., Linsley, P. S., and Wilson, C. B. (1995). Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration. Nat. Genet. 11, 191-197.
50. Volk, H. D., Siegling, A., Platzer, C., Brock, J., Emmrich, F., and Lehmann, M. (1994). Tolerance induction by anti-CD4 MAb treatment in a rat kidney transplantation model. Transplant. Proc. 26, 3155-3156.
51. Scully, R., Qin, S., Cobbold, S., and Waldmann, H. (1994). Mechanisms in CD4 antibody-mediated transplantation tolerance: kinetics of induction, antigen dependency and role of regulatory T cells. Eur. J. Immunol. 24, 2383-2392.
52. Kolls, J. K., Lei, D., Odom, G., Nelson, S., Summer, W. R., Gerber, M. A., Shellito, J. E. (1996). Use of transient CD4 lymphocyte depletion to prolong transgene expression of El-deleted adenoviral vectors. Hum. Gene. Ther. 7, 489-497.
53. Overturf, K., Aldhalimy, M., Tanguay, R., Brantly, M., Ou, C. N., Finegold, M., and Grompe, M. (1996). Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I. Nat. Genet. 12, 266-273.
54. Sandig, V., Löser, P., Lieber, A., Kay, M. A., and Strauss, M. (1996). HBV-derived promoter directed liver-specific expression of an adenovirally transduced LDL receptor gene. Gene Therapy 3, 1002-1009.
55. Hofmann, C., Sandig, V., Jennings, G., Rudolph, M., Schlag, P., and Strauss, M. (1995). Efficient gene transfer into human hepatocytes by baculovirus vectors. Proc. Natl. Acad. Sci. U.S.A 92, 10099-10103.
56. Sandig, V., Hofmann, C., Steinert, S., Jennings, G., Schlag, P., and Strauss, M. (1996). Gene transfer into human liver cells and tissue by baculoviral vectors. Human Gene Therapy, 1937-1945.
57. Sandig, V. and Strauss, M. (1996). Liver-directed gene transfer: prospects for therapy. J. Molec. Med. 74, 205-212.
58. Strauss, M. (1996). Gene transfer on its long path to clinical application. J. Molec. Med. 74, 167-169.