Strategies for long-term gene expression in the skin to treat metabolic disorders

Expert Opinion on Biological Therapy

Volumn 4, Issue 5, 2004, Pages 677-682

  Jensen, Thomas G ^a  

^a JOHN F KENNEDY INSTITUTE   (Denmark)

Author keywords

Bioreactor; Gene therapy; Metabolic diseases; Metabolic sink; Vectors

Indexed keywords

VIRUS VECTOR;

GENE EXPRESSION; GENE TRANSFER; GENETIC ENGINEERING; GENETIC MANIPULATION; HUMAN; KERATINOCYTE; METABOLIC DISORDER; METABOLITE; NONHUMAN; PROTEIN SECRETION; REVIEW; SOMATIC GENE THERAPY;

ANIMALS; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HUMANS; METABOLIC DISEASES; SKIN; VIRUSES;

EID: 2442680216     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.4.5.677     Document Type: Review

References (55)

1. 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. (1995) 10:161-166.
2. GLASSPOOL-MALONE J, SOMIARI S, DRABICK JJ, MALONE RW: Efficient nonviral cutaneous transfection. Mol. Ther. (2000) 2:140-146.
3. WILLIAMS RS, JOHNSTON SA, RIEDY M, DEVIT MJ, MCELLIGOTT SG, SANFORD JC: Introduction of foreign genes into tissues of living mice by DNA-coated microprojectiles. Proc. Natl. Acad. Sci. USA (1991) 88:2726-2730.
4. JOHNSTON SA, TANG DC: The use of microparticle injection to introduce genes into animal cells in vitro and in vivo. Genet. Eng. (1993) 15:225-236.
5. FAN H, LIN Q, MORRISSEY GR, KHAVARI PA: Immunization via hair follicles by topical application of naked DNA to normal skin. Nat. Biotechnol. (1999) 17:870-872.
6. TANG DC, SHI Z, CURIEL DT: Vaccination onto bare skin. Nature (1997) 388:729-730.
7. DOMASHENKO A, GUPTA S, COTSARELIS G: Efficient delivery of transgenes to human hair follicle progenitor cells using topical lipoplex. Nat. Biotechnol. (2000) 18:420-423.
8. ALEXANDER MY, AKHURST RJ: Liposome-medicated gene transfer and expression via the skin. Hum. Mol. Genet. (1995) 4:2279-2285.
9. ROBBINS PB, OLIVER SF, SHEU SM et al.: Peptide delivery to tissues via reversibly linked protein transduction sequences. Biotechniques (2002) 33:190-192, 194.
10. WHITE PJ, GRAY AC, FOGARTY RD et al.: C-5 propyne-modified oligonucleotides penetrate the epidermis in psoriatic and not normal human skin after topical application, J. Invest. Dermatol. (2002) 118:1003-1007.
11. MEHTA RC, STECKER KK, COOPER SR et al.: Intercellular adhesion molecule-1 suppression in skin by topical delivery of anti-sense oligonucleotides. J. Invest. Dermatol. (2000) 115:805-812.
12. SIPRASHVILI Z, SCHOLL FA, OLIVER SF et al.: Gene transfer via reversible plasmid condensation with cysteine-flanked, internally spaced arginine-rich peptides. Hum. Gene Ther. (2003) 14:1225-1233. Promising non-viral gene transfer method for in vivo applications.
13. BRANDEN LJ, SMITH CI: Bioplex technology: novel synthetic gene delivery system based on peptides anchored to nucleic acids. Methods Enzymol. (2002) 346:106-124.
14. YANT SR, MEUSE L, CHIU W et al.: Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat. Genet. (2000) 25:35-41. Gene transfer system leading to efficient integration in the genome.
15. ORTIZ-URDA S, LIN Q, YANT SR et al.: Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer. Gene Ther. (2003) 10:1099-1104.
16. ORTIZ-URDA S, THYAGARAJAN B, KEENE DR et al.: Stable nonviral genetic correction of inherited human skin disease. Nat. Med. (2002) 8:1166-1170. Efficient site-specific integration in the genome of human keratinocytes.
17. GARLICK JA, KATZ AB, FENJVES ES, TAICHMAN LB: Retrovirus-mediated transduction of cultured epidermal keratinocytes. J. Invest. Dermatol. (1991) 97:824-829. Pioneering article about epidermal gene therapy.
18. DENG H, CHOATE KA, LIN Q, KHAVARI PA: High-efficiency gene transfer and pharmacologic selection of genetically engineered human keratinocytes. Biotechniques (1998) 25:274-280.
19. JENSEN UB, PETERSEN MS, LUND TB, JENSEN TG, BOLUND L: Transgene expression in human epidermal keratinocytes: cell cycle arrest of productively transfected cells. Exp. Dermatol. (2000) 9:298-310.
20. KOLODKA TM, GARLICK JA, TAICHMAN LB: Evidence for keratinocyte stem cells in vitro: long term engraftment and persistence of transgene expression from retrovirus-transduced keratinocytes. Proc. Natl. Acad. Sci. USA (1998) 95:4356-4361.
21. WHITE SJ, PAGE SM, MARGARITIS P, BROWNLEE GG: Long-term expression of human clotting Factor IX from retrovirally transduced primary human keratinocytes in vivo. Hum. Gene Ther. (1998) 9:1187-1195.
22. DENG H, LIN Q, KHAVARI PA: Sustainable cutaneous gene delivery. Nat. Biotechnol. (1997) 15:1388-1391.
23. LEVY L, BROAD S, ZHU AJ et al.: Optimised retroviral infection of human epidermal keratinocytes: long-term expression of transduced integrin gene following grafting on to SCID mice. Gene Ther. (1998) 5:913-922.
24. GHAZIZADEH S, HARRINGTON R, TAICHMAN L: In vivo transduction of mouse epidermis with recombinant retroviral vectors: implications for cutaneous gene therapy. Gene Ther. (1999) 6:1267-1275.
25. GHAZIZADEH S, KALISH RS, TAICHMAN L: Immune-mediated loss of transgene expression in skin: implications for cutaneous gene therapy. Mol. Ther. (2003) 7:294-295.
26. BAEK SC, LIN Q, ROBBINS PB, FAN H, KHAVARI PA: Sustainable systemic delivery via a single injection of lentivirus into human skin tissue. Hum. Gene Ther. (2001) 12:1551-1558.
27. HENGGE UR, MIRMOHAMMADSADEGH A: Adeno-associated virus expresses transgenes in hair follicles and epidermis. Mol. Ther. (2000) 2:188-194.
28. DONAHUE BA, MCARTHUR JG, SPRATT SK et al.: Selective uptake and sustained expression of AAV vectors following subcutaneous delivery. J. Gene Med. (1999) 1:31-42.
29. JENSEN TG, JENSEN UB, BOLUND L: Production of retroviral vectors in primary human keratinocytes after DNA-mediated gene transfer leads to prolonged gene expression. Acta Derm. Venereol. (2003) 83:83-87.
30. WATT FM: Epidermal stem cells as targets for gene transfer. Hum. Gene Ther. (2000) 11:2261-2266.
31. PFUTZNER W, HENGGE UR, JOARI MA, FOSTER RA, VOGEL JC: Selection of keratinocytes transduced with the multidrug resistance gene in an in vitro skin model presents a strategy for enhancing gene expression in vivo. Hum. Gene Ther. (1999) 10:2811-2821.
32. PFUTZNER W, TERUNUMA A, TOCK CL et al.: Topical colchicine selection of keratinocytes transduced with the multidrug resistance gene (MDR1) can sustain and enhance transgene expression in vivo. Proc. Natl. Acad. Sci. USA (2002) 99:13096-13101.
33. FAKHARZADEH SS, ZHANG Y, SARKAR R, KAZAZIAN HH JR: Correction of the coagulation defect in hemophilia A mice through Factor VIII expression in skin. Blood (2000) 95:2799-2805. Important article showing that proteins from the skin can correct a disease phenotype.
34. LARCHER F, DEL RIO M, SERRANO F et al.: A cutaneous gene therapy approach to human leptin deficiencies: correction of the murine ob/ob phenotype using leptin-targeted keratinocyte grafts. FASEB J. (2001) 15:1529-1538.
35. TAICHMAN LB: Epithelial gene therapy. In: The Keratinocyte Handbook. Leigh IM, Lane B, Watt F (Eds), Cambridge University Press, Cambridge, UK (1994):543-551.
36. PALMER TD, HOCK RA, OSBORNE WR, MILLER AD: Efficient retrovirus-mediated transfer and expression of a human adenosine deaminase gene in diploid skin fibroblasts from an adenosine deaminase-deficient human. Proc. Natl. Acad. Sci. USA (1987) 84:1055-1059.
37. FENJVES ES, SCHWARTZ PM, BLAESE RM, TAICHMAN LB: Keratinocyte gene therapy for adenosine deaminase deficiency: a model approach for inherited metabolic disorders. Hum. Gene Ther. (1997) 8:911-917.
38. KAISER-KUPFER MI, DE MONASTERIO F, VALLE D, WALSER M, BRUSILOW S: Visual results of a long-term trial of a low-arginine diet in gyrate atrophy of choroid and retina. Ophthalmology (1981) 88:307-310.
39. KAISER-KUPFER MI, CARUSO RC, VALLE D: Gyrate atrophy of the choroid and retina. Long-term reduction of ornithine slows retinal degeneration. Arch. Ophthalmol. (1991) 109:1539-1548.
40. JENSEN TG, SULLIVAN DM, MORGAN RA et al.: Retrovirus-mediated gene transfer of ornithine-delta-aminotransferase into keratinocytes from gyrate atrophy patients. Hum. Gene Ther. (1997) 8:2125-2132.
41. SULLIVAN DM, JENSEN TG, TAICHMAN LB, CSAKY KG: Ornithine-delta-aminotransferase expression and ornithine metabolism in cultured epidermal keratinocytes: toward metabolic sink therapy for gyrate atrophy. Gene Ther. (1997) 4:1036-1044.
42. CHRISTENSEN R, KOLVRAA S, BLAESE RM, JENSEN TG: Development of a skin-based metabolic sink for phenylalanine by overexpression of phenylalanine hydroxylase and GTP cyclohydrolase in primary human keratinocytes. Gene Ther. (2000) 7:1971-1978.
43. CHRISTENSEN R, GUTTLER F, JENSEN TG: Comparison of epidermal keratinocytes and dermal fibroblasts as potential target cells for somatic gene therapy of phenylketonuria. Mol. Genet. Metab. (2002) 76:313-318.
44. CHRISTENSEN R, KOLVRAA S, JENSEN TG: Manipulation of the phenylalanine metabolism in human keratinocytes. Submitted.
45. 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. (1994) 102:415-421.
46. FENJVES ES, GORDON DA, PERSHING LK, WILLIAMS DL, TAICHMAN LB: Systemic distribution of apolipoprotein E secreted by grafts of epidermal keratinocytes: implications for epidermal function and gene therapy. Proc. Natl. Acad. Sci. USA (1989) 86:8803-8807.
47. KRUEGER GG, MORGAN JR, JORGENSEN CM et al.: Genetically modified skin to treat disease: potential and limitations. J. Invest. Dermatol. (1994) 103:76S-84S.
48. GERRARD AJ, HUDSON DL, BROWNLEE GG, WATT FM: Towards gene therapy for haemophilia B using primary human keratinocytes. Nat. Genet. (1993) 3:180-183.
49. JENSEN UB, JENSEN TG, JENSEN PK et al.: Gene transfer into cultured human epidermis and its transplantation onto immunodeficient mice: an experimental model for somatic gene therapy. J. Invest. Dermatol. (1994) 103:391-394.
50. DA COSTA TH, WILLIAMSON DH, WARD A et al.: High plasma insulin-like growth factor-II and low lipid content in transgenic mice: measurements of lipid metabolism. J. Endocrinol. (1994) 143:433-439.
51. MENG X, SAWAMURA D, INA S et al.: Keratinocyte gene therapy: cytokine gene expression in local keratinocytes and in circulation by introducing cytokine genes into skin. Exp. Dermatol. (2002) 11:456-461.
52. SUN WH, BURKHOLDER JK, SUN J et al.: In vivo cytokine gene transfer by gene gun reduces tumor growth in mice. Proc. Natl. Acad. Sci. USA (1995) 92:2889-2893.
53. MENG X, SAWAMURA D, TAMAI K et al.: Keratinocyte gene therapy for systemic diseases. Circulating interleukin 10 released from gene-transferred keratinocytes inhibits contact hypersensitivity at distant areas of the skin. J. Clin. Invest. (1998) 101:1462-1467.
54. CHENG J, TURKSEN K, YU QC et al.: Cachexia and graft-vs.-host-disease-type skin changes in keratin promoter-driven TNF alpha transgenic mice. Genes Dev. (1992) 6:1444-1456.
55. RIO MD, LARCHER F, MEANA A et al.: Nonviral transfer of genes to pig primary keratinocytes. Induction of angiogenesis by composite grafts of modified keratinocytes overexpressing VEGF driven by a keratin promoter. Gene Ther. (1999) 6:1734-1741.