An ex vivo keratinocyte model for gene therapy of hemophilia B

Journal of Investigative Dermatology

Volumn 109, Issue 2, 1997, Pages 139-145

  Page, Sean M ^a   Brownlee, George G ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

Epidermis; Factor IX; Retroviral vector; Skin grafting

Indexed keywords

BLOOD CLOTTING FACTOR 9; COMPLEMENTARY DNA; KERATIN; SILICONE; VIRUS VECTOR;

ANIMAL EXPERIMENT; ARTICLE; CELL TRANSPLANTATION; CHIMERA; CYTOMEGALOVIRUS; ENZYME LINKED IMMUNOSORBENT ASSAY; GENE; GENE THERAPY; GENE TRANSFER; GRAFT SURVIVAL; HEMOPHILIA B; HUMAN; HUMAN CELL; KERATINOCYTE; MODEL; MOUSE; NONHUMAN; PRIORITY JOURNAL; RETROVIRUS; WART VIRUS;

EID: 0030832442     PISSN: 0022202X     EISSN: None     Source Type: Journal    
DOI: 10.1111/1523-1747.ep12319194     Document Type: Article

References (62)

1. Alexander MY, Akhurst RJ: Liposome-mediated gene transfer and expression via the skin. Hum Mol Genet 4:2279-2285, 1995
2. Alexander MY, Bidichandani SI, Cousins FM, Robinson CJ, Duffie E, Akhurst RJ: Circulating human factor IX produced in keratin promoter-transgenic mice: a feasibility study tor gene therapy of hemophilia B. Hum Mol Genet 4:993-999, 1995
3. Anson DS, Austen DEG, Brownlee GG: Expression of active human clotting factor IX from recombinant DNA clones in mammalian cells. Nature 315:683-685, 1985
4. Bonnekoh B, Greenhalgh DA, Bundman DS, Eckhardt JN, Longley MA, Chen SH, Woo SL, Roop DR: Inhibition of melanoma growth by adenoviral-mediated HSV thymidine kinase gene transfer in vivo. J Invest Dermatol 104:313-317, 1995
5. Boukamp P, Breitkreutz D, Stark H-J, Fusenig NE: Mesenchyme-mediated and endogenous regulation of growth and differentiation of human skin keratinocytes derived from different body sites. Differentiation 44:150-161, 1990
6. Boukamp P, Rupniak HTR, Fusenig NE: Environmental modulation of the expression of differentiation and malignancy in six human squamous cell carcinoma cell lines. Cancer Res 45:5582-5592, 1985
7. Boyce ST, Supp AP, Harriger MD, Pickens WL, Wickett RR, Hoath SB: Surface electrical capacitance as a noninvasive index of epidermal harrier in cultured skin substitutes in athymic mice. J Invest Dermatol 107:82-87, 1996
8. Brownlee GG: Prospects for gene therapy of haemophilia A and B. Br Med Bull 51:91-105, 1995
9. Byrne C, Fuchs E: Probing keratinocyte and differentiation specificity of the human K5 promoter in vitro and in transgenic mice. Mol Cell Biol 13:3176-319, 1993
10. Challita P-M, 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 USA 91:2567-2571, 1994
11. Chen S-T, Iida A, Guo L, Friedmann T, Yee J-K: Generation of packaging cell lines for pseudotyped retroviral vectors of the G protein of vesicular stomatis virus by using a modified tetracycline inducible system. Proc Natl Acad Sci USA 93:10057-10062, 1996
12. Cheng L, Ziegelhoffer PR, Yang NS: In vivo promoter activity and transgene expression in mammalian somatic tissues evaluated by using particle bombardment. Proc Natl Acad Sci USA 90:4455-4459, 1993
13. Choate KA, Medalie DA, Morgan JR, Khavari PA: Corrective gene transfer in the human skin disorder lamellar ichthyosis. Nat Med 2:1263-1267, 1996
14. Chowdhury JR, Grossman M, Gupta S, Chowdhury NR, Baker JR Jr, Wilson JM: Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. Science 254:1802-1805, 1991
15. Couture LA, Mullen CA, Morgan RA: Retroviral vectors containing chimeric promoter/enhancer elements exhibit cell-type specific gene expression. Hum Gene Ther 5:667-677, 1994
16. Cripe TP, Haugen TH, Turk JP, Tabatabai F, Schmid PG, Dürst M, Gissmann L, Roman A, Turek LP: Transcriptional regulation of the human papilloma virus-16 E6-E7 promoter by a keratinocyte-dependent enhancer, and by viral E2 transactivator and repressor gene products: implications for cervical carcinogenesis. EMBO J 6:3745-3753, 1987
17. Dai Y, Roman M, Naviaux RK, Verma IM: Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo. Proc Natl Acad Sci USA 89:10892-10895, 1992
18. Dwarki VJ, Belloni P, Nijjar T, Smith J, Couto L, Rabier M, Clift S, Berns A, Cohen LK: Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci USA 92:1023-1027, 1995
19. Fenjves ES, Yao S-N, Kurachi K, Taichman LB: Loss of expression of a retrovirus-transduced gene in human keratinocytes. J Invest Dermatol 106:576-578, 1996
20. Ferry N, Duplessis O, Houssin D, Danos O, Heard J-M: Retroviral-mediated gene transfer into hepatocytes in vivo. Proc Natl Acad Sci USA 88:8377-8381, 1991
21. Flowers MED, Stockschläder MAR, Schuening FG, Niederwieser D, Hackman R, Miller DA, Storb R: Long-term transplantation of canine keratinocytes made resistant to G418 through retro virus-mediated gene transfer. Proc Natl Acad Sci USA 87:2349-2353, 1990
22. Gallico GG, O'Connor NE, Compton CC, Kehinde O, Green H: Permanent coverage of large burn wounds with autologous cultured human epithelium. N Engl J Med 311:448-451, 1984
23. Gerrard AJ, Austen DEG, Brownlee GG: Subcutaneous injection of factor IX for the treatment of haemophilia B. Br J Haematol 81:610-613, 1992
24. Gerrard AJ, Austen DEJG, Brownlee GG: Recombinant factor IX secreted by transduced human keratinocytes is biologically active. Br J Haematol 95:561-563, 1996
25. Gerrard AJ, Hudson DL, Brownlee GG, Watt FM: Towards gene therapy for haemophilia B using normal human keratinocytes. Nat Genet 3:180-183, 1993
26. Green H, Kehinde O, Thomas J: Growth of cultured human epidermal cells into multiple epithelia suitable for grafting. Proc Natl Acad Sci USA 76:5665-5668, 1979
27. Greenhalgh DA, Rothnagel JA, Roop DR: Epidermis: an attractive target tissue for gene therapy. J Invest Dermatol 103:63S-69S, 1994
28. Hafenrichter DG, Wu X, Rettinger SD, Kennedy SC, Flye MW, Ponder KP: Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes. Blood 84:3394-3404, 1994
29. Heartlein MW, Roman VA, Jiang J-L, Sellers JW, Zuliani AM, Treco DA, Seiden RF: Long-term production and delivery of human growth hormone in vivo. Proc Natl Acad Sci USA 91:10967-10971, 1994
30. Hedner U, Davie EW: Introduction to hemostasis and the vitamin K-dependent coagulation factors. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds.). The Metabolic Basis of Inherited Disease. 6th ed. McGraw Hill Publishers, New York, pp 2107-2134, 1989
31. Hengge UR, Chan EF, Foster RA, Walker PS, Vogel JC: Cytokine gene expression in epidermis with biological effects following injection of naked DNA. Nat Genet 10:161-166, 1995
32. Jensen UB, Jensen TG, Jensen PKA, Rygaard J, Hansen BS, Fogh J, Kølvraa S, Bolund L: Gene transfer into cultured human epidermis and its transplantation onto immunodeficient mice: an experimental model for somatic gene therapy. J Invest Dermatol 103:391-394, 1994
33. Jiang CK, Epstein HS, Tomic M, Freedberg IM, Blumenberg M: Epithelial-specific keratin gene expression: identification of a 300 base-pair controlling segment. Nucleic Adds Res 18:247-253, 1990
34. Jones PH, Watt FM: Separation of human epidermal stem cells from transit amplifying cells on the basis of differences in integrin function and expression. Cell 73:713-724, 1993
35. Katsumi A, Emi N, Abe A, Hasegawa Y, Ito M, Saito H: Humoral and cellular immunity to an encoded protein induced by direct DNA injection. Hum Gene Ther 5:1335-1339, 1994
36. 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 89:89-93, 1992
37. Kozak M: Structural features in eukaryotic mRNAs that modulate the initiation of translation. J Biol Chem 266:19867-19870, 1991
38. Leask A, Byrne C, Fuchs E: Transcription factor AP2 and its role in epidermal-specific gene expression. Proc Natl Acad Sci USA 88:7948-7952, 1991
39. Leask A, Rosenberg M, Vassar R, Fuchs E: Regulation of a human epidermal keratin gene: sequences and nuclear factors involved in keratinocyte-specific transcription. Gents Dev 4:1985-1998, 1990
40. Li L, Hoffman RM: The feasibility of targeted selective gene therapy of the hair follicle. Nat Med 1:705-706, 1995
41. Mann R, Mulligan RC, Baltimore D: Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus. Cell 33:153-159, 1983
42. Miller AD, Buttimore C: Redesign of retrovirus packaging cell lines to avoid recombination leading to helper virus production. Mol Cell Biol 6:2895-2902, 1986
43. Miller AD, Miller DG, Garcia JV, Lynch CM: Use of retroviral vectors for gene transfer and expression. Methods Enzymol 217:581-599, 1993
44. Miller AD, Rosman GJ: Improved retroviral vectors for gene transfer and expression. BioTechniques 7:980-987, 1989
45. Morgan JR, Barrandon Y, Green H, Mulligan RC: Expression of an exogenous growth hormone gene by transplantable human epidermal cells. Science 237: 1476-1479, 1987
46. Moullier P, Bohl D, Heard J-M, Danos O: Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically-modified skin fibroblasts. Nat Genet 4:154-159, 1993
47. Ohtsuki M, Tomic-Canic M, Freedberg IM, Blumenberg M: Nuclear proteins involved in transcription of the human K5 keratin gene. J Invest Dermatol 99:206-215, 1992
48. 1-antitrypsin in vivo. Hum Gene Ther 7:637-645,1996
49. Palmer TD, Rosman GJ, Osborne WRA, Miller AD: Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc Natl Acad Sci USA 88:1330-1334, 1991
50. Palmer TD, Thompson AR, Miller AD: Production of human factor IX in animals by genetically modified skin fibroblasts: potential gene therapy for hemophilia B. Blond 73:438-445, 1989
51. Rees DJG, Jones IM, Handford PA, Walter SJ, Esnouf MP, Smith KJ, Brownlee GG: The role of β-hydroxyaspartate and adjacent carboxylate residues in the first EGF domain of human factor IX. EMBO J 7:2053-2061, 1988
52. Rettinger SD, Kennedy SC, Wu X, Saylors RL, Hafenrichter DG, Flye MW, Ponder KP: Liver-directed gene therapy: quantitative evaluation of promoter elements by using in vivo retroviral transduction. Proc Natl Acad Sci USA 91:1460-1464, 1994
53. Rheinwald JG: Methods for clonal growth and serial cultivation of normal human epidermal keratinocytes and mesothelial cells. In: Baserga R, (ed.). Cell Growth and Division: A Practical Approach. IRL Press, Oxford, U.K., pp 81-94, 1989
54. Rheinwald JG, Green H: Serial cultivation of strains of human epidermal keratinocytes: the formation of keratinizing colonies from single cells. Cell 6:331-344, 1975
55. Scharfmann R, Axelrod JH, Verma IM: Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants. Proc Natl Acad Sci USA 88:4626-4630, 1991
56. Seedorf K, Krämmer G, Durst M, Suhai S, Röwekamp WG: Human papilloma virus type 16 DNA sequence. Virology 145:181-185, 1985
57. Setoguchi Y, Jaffe HA, Danel C, Crystal RG: Ex vivo and in vivo gene transfer to the skin using replication-deficient recombinant adenovirus vectors. J Invest Dermatol 102:415-421, 1994
58. Stockschläder MAR, Schuening FG, Graham TC, Storb R: Transplantation of retrovirus-transduced canine keratinocytes expressing the β-galactosidase gene. Gene Ther 1:317-322, 1994
59. Stockschläder MAR, Storb R, Osborne WRA, Miller AD: L-histidinol provides effective selection of retrovirus-vector-transduced keratinocytes without impairing their proliferative potential. Hum Gene Ther 2:33-39, 1991
60. Wu X, Holschen J, Kennedy SC, Ponder KP: Retroviral vector sequences may interact with some internal promoters and influence expression. Hum Gene Ther 7:159-171, 1996
61. Yao S-N, Wilson JM, Nabel EG, Kurachi S, Hachiya HL, Kurachi K: Expression of human factor IX in rat capillary endothelial cells: towards somatic gene therapy for hemophilia B. Proc Natl Acad Sci USA 88:8101-8105, 1991
62. Yoshioka A, Giddings JC, Thomas JE, Fujimura Y, Bloom AL: Immunoassays of factor IX antigen using monoclonal antibodies. Br J Haematol 59:265-275, 1985