Gene therapy for tissue repair: Approaches and prospects

British Journal of Plastic Surgery

Volumn 50, Issue 7, 1997, Pages 491-500

  Eming, S A ^a,c   Morgan, J R ^a   Berger, A ^b,d  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

^b LEIBNIZ UNIVERSITY HANNOVER   (Germany)

^c UNIVERSITY HOSPITAL OF COLOGNE   (Germany)

^d Zentrum Inn Med und Dermatologie   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

GROWTH FACTOR; LIPOSOME; PLATELET DERIVED GROWTH FACTOR AA;

ANIMAL CELL; ARTICLE; BLOOD VESSEL; CARTILAGE; CELL MIGRATION; CELL PROLIFERATION; CONTROLLED STUDY; EPIDERMIS CELL; EXTRACELLULAR MATRIX; GENE THERAPY; GENE TRANSFER; HUMAN; HUMAN CELL; KERATINOCYTE; MOLECULAR BIOLOGY; MOLECULAR GENETICS; NONHUMAN; PRIORITY JOURNAL; RETROVIRUS; SKIN GRAFT; SOMATIC CELL; TISSUE REPAIR; VIRUS RECOMBINANT; WOUND HEALING;

EID: 0030731716     PISSN: 00071226     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0007-1226(97)91297-2     Document Type: Article

References (93)

1. Clark RAF. Cutaneous tissue repair: Basic biologic considerations. I. Am Acad Dermatol 1985; 13: 701-25.
2. Falanga V, Eaglstein WH. The trap hypothesis of venous ulceration. Lancet 1993; 341: 1006-8.
3. Higley HR, Ksander GA, Gerhardt CO, Falanga V. Extravasation of macromolecules and possible trapping of transforming growth factor-β in venous ulceration. Br J Dermatol 1995; 132: 79-85.
4. Weckroth M, Vaheri A, Lauharanta J, Sorsa T, Konttinen YT. Matrix metalloproteinases, gelatinase and collagenase, in chronic leg ulcers. J Invest Dermatol 1996; 106: 1119-24.
5. Wysocki AB, Staiano-Coico L, Grinnell F. Wound fluid from chronic leg ulcers contains elevated levels of metalloproteinases MMP-2 and MMP-9. J Invest Dermatol 1993; 101: 64-8.
6. Nemeth AJ. Keloids and hypertrophic scars. J Dermatol Surg Oncol 1993; 19: 738-46.
7. Lawrence WT, Diegelmann RF. Growth factors in wound healing. Clin Dermatol 1994; 12: 157-69.
8. Anderson KD, Thompson JA, DiPitro JM, Montgomory KT, Reid LM, Anderson WF. Gene expression in implanted rat hepatocytes following retroviral-mediated gene transfer. Somatic Cell and Molecular Genetics 1989; 15: 215-27.
9. Kay MA, Rothenberg S, Landen CN, et al. In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient dogs. Science 1993; 262: 117-9.
10. Kozarsky KF, Jooss K, Donahee M, Strauss JF, Wilson JM. Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL. Nat Genet 1996; 13: 54-62.
11. Wilson JM. Round two for liver gene therapy. Nat Genet 1996; 12: 232-3.
12. Zabner J, Couture LA, Gregory RJ, Graham SM, Smith AE, Welsh MJ. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 1993; 75: 207-16.
13. Hyde SC, Gill DR, Higgins CF, et al. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 1993; 362: 250-5.
14. Stribling R, Brunette E, Liggitt D, Gaensler K, Debs R. Aerosol gene delivery in vivo. Proc Natl Acad Sci 1992; 89: 11277-81.
15. Vile RG, Hart IR. In vitro and in vivo targeting of gene expression to melanoma cells. Cancer Res 1993; 53: 962-7.
16. Bonnekoh B, Greenhalgh DA, Bundman DS, et al. Adenoviral-mediated herpes simplex virus-thymidine kinase transfer in vivo for treatment of experimental human melanoma. J Invest Dermatol 1996; 106: 1163-8.
17. Nabel GJ, Nabel EG, Yang ZY, et al. Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity, and lack of toxicity in humans. Proc Natl Acad Sci 1993; 90: 11307-11.
18. Schadendorf D, Sun Y, Möller H et al. IL-7 gene transfer into autologous melanoma cells for vaccination - a pilot study in patients with advanced melanoma. J Invest Dermatol 1996; 107: 454.
19. Bandara G, Mueller GM, Galea-Lauri J et al. Intraarticular expression of biologically active interleukin 1-receptor-antagonist protein by ex vivo gene transfer. Proc Natl Acad Sci 1993; 90: 10764-8.
20. Hung GL, Galea-Lauri J, Mueller GM et al. Suppression of intra-articular responses to IL-1 by transfer of the IL-1 receptor antagonist to synovium. Gene Therapy 1994; 1: 64-9.
21. St. Louis D, Verma IM. An alternative approach to somatic cell gene therapy. Proc Natl Acad Sci 1988; 85: 3150-4.
22. Palmer TD, Rosman GJ, Osborne WR, Miller AD. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc Natl Acad Sci 1991; 88: 1330-4.
23. Scharfmann R, Axelrod JH, Verma IM. Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants. Proc Natl Acad Sci 1991; 88: 4626-30.
24. Moullier P, Bohl D, Heard JM, Danos O. Correction of the lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts. Nat Gene 1993; 4: 154-9.
25. Petersen MJ, Kaplan J, Jorgensen CM et al. Sustained production of human transferrin by transduced fibroblasts implanted into athymic mice: a model for somatic gene therapy. J Invest Dermatol 1995; 104: 171-6.
26. Krueger GG, Morgan JR, Jorgensen CM et al. Genetically modified skin to treat disease: potential and limitations. J Invest Dermatol 1994; 103: 73S-84S.
27. Palmer TD, Hock RA, Osborne WRA, 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 1987; 84: 1055-9.
28. Baragi VM, Renkiewicz RR, Jordan H, Bonadio J, Hartman JW, Roessler BJ. Transplantation of transduced chondrocytes protects articular cartilage from interleukin 1 induced extracellular matrix degradation. J Clin Invest 1995; 96: 2454-60.
29. Dhawan J, Pan LC, Pavlath GK, Travis MA, Lanctot AM, Blau HM. Systematic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 1991; 254: 1509-12.
30. Barr E, Leiden JM. Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 1991; 254: 1507-9.
31. Ohno T, Gordon D, San H et al. Gene therapy for vascular smooth muscle cell proliferation after arterial injury. Science 1994; 265: 781-4.
32. Zwiebel JA, Freeman SM, Kantoff PW, Cornetta K, Ryan US, Anderson F. High-level recombinant gene expression in rabbit endothelial cells transduced by retroviral vectors. Science 1989; 243: 220-2.
33. Wilson JM, Birinyi LK, Salomon RN, Libby PL, Callow AD, Mulligan RC. Implantation of vascular grafts lined with genetically modified endothelial cells. Science 1989; 244: 1344-6.
34. Dichek DA, Neville RF, Zwiebel JA, Freeman SM, Leon MB, Anderson WF. Seeding of intravascular stents with genetically engineered endothelial cells. Circulation 1989; 80: 1347-53.
35. Steg PG, Feldman LJ, Scoazec JY et al. Arterial gene transfer to rabbit endothelial and smooth muscle cells using percutaneous delivery of an adenoviral vector. Circulation 1994; 90: 1648-56.
36. Newman KD, Dunn PF, Owens JW et al. Adenovirus-mediated gene transfer into normal rabbit arteries results in prolonged vascular cell activation, inflammation, and neointimal hyperplasia. J Clin Invest 1995; 96: 2955-65.
37. Morgan JR, Barrandon Y, Green H, Mulligan RC. Expression of an exogenous growth hormone gene by transplantable human epidermal cells. Science 1987; 237: 1476-9.
38. Garlick JA, Katz AB, Fenjves ES, Taichman LB. Retrovirus-mediated transduction of cultured epidermal keratinocytes. J Invest Dermatol 1991; 97: 824-9.
39. Fenjves ES, Smith J, Zaradic S, Taichman LB. Systemic delivery of secreted protein by grafts of epidermal keratinocytes: prospects for keratinocyte gene therapy. Hum Gen Therp 1994; 5: 1241-8.
40. Li L, Hoffman RM. The feasibility of targeted selective gene therapy of the hair follicle. Nat Genet 1995; 1: 705-6.
41. Hengge UR, Chan EF, Foster RA, Walker PS, Vogel JC. Cytokines gene expression in epidermis with biological effects following injection of naked DNA. Nat Genet 1995; 10: 161-6.
42. Eming SA, Lee J, Snow RG, Tompkins RG, Yarmush ML, Morgan JR. Genetically modified human epidermis over-expressing PDGF-A directs the development of a cellular and vascular connective tissue stroma when transplanted to athymic mice: implications for the use of genetically modified keratinocytes to modulate dermal regeneratiom. J Invest Dermatol 1995; 105: 756-63.
43. Eming SA, Snow RG, Yarmush ML, Morgan JR. Targeted expression of IGF-1 to human keratinocytes: modification of the autocrine control of keratinocyte proliferation. J Invest Dermatol 1996; 106: 113-20.
44. Fenjves ES, Yao SN, Kurachi K, Taichman LB. Loss of expression of a retrovirus-transduced gene in human keratinocytes. J Invest Dermatol 1996; 106: 576-8.
45. Vogt PM, Thompson S, Andree C et al. Genetically modified keratinocytes transplanted to wounds reconstitute the epidermis. Proc Natl Acad Sci USA 1994; 91: 9307-11.
46. Carreau M, Quilliet X, Eveno E et al. Functional retroviral vector for gene therapy of xeroderma pigmentosum group D patients. Hum Gen Ther 1995; 6: 1307-15.
47. Andree C, Swain WF, Page CP et al. In vivo transfer and expression of a human epidermal growth factor gene accelerates wound repair. Proc Natl Acad Sci 1994; 91: 12188-92.
48. Benn SI, Whitsitt JS, Broadley KN et al. Enhancement of wound healing in rat skin following particle bombardment with cDNAs encoding TGF-β1. J Clin Invest (in press).
49. Chen C, Okayama H. High-efficient transformation of mammalian cells by plasmid DNA. Mol Cell Biol 1987; 7: 2745-52.
50. Feigner PL, Ringold GM. Cationic liposome-mediated transfection. Nature 1989; 337: 387-8.
51. Trubetskoy VS, Torchilin VP, Kennel S, Huang L. Cationic liposomes enhance targeted delivery and expression of exogenous DNA mediated by N-terminal modified poly(L-lysine)-antibody conjugate in mouse lung endothelial cells. Biochem Biophys Acta 1992; 1131: 311-3.
52. Nabel EG, Plautz G, Nabel GJ. Site-specific gene expression in vivo by direct gene transfer into arterial wall. Science 1990; 249: 1285-8.
53. Takeshita S, Gal D, Leclerc GJ et al. Increased gene expression after liposome-mediated arterial gene transfer associated with intimai smooth muscle cell proliferation. In vitro and in vivo findings in a rabbit model of vascular injury. J Clin Invest 1994; 93: 652-61.
54. Tang D, De Vit M, Johnston S. Genetic immunization is a simple method for eliciting an immune response. Nature 1992; 356: 152-4.
55. 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: 2726-30.
56. 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 1993; 990: 4455-9.
57. 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: 2889-93.
58. Yang NS. Gene transfer into mammalian somatic cells in vivo. Crit Rev Biotechnol 1992; 12: 335-56.
59. Weiss R, Teich N, Varmus H. Molecular biology of tumor viruses. In: RNA Tumor Viruses, 2nd edn. Cold Spring Harbor Laboratory Press, Cold Spring Harbor, New York, 1982.
60. Roemer K, Friedmann T. Concepts and strategies for human gene therapy. Eur J Biochem 1992; 208: 211-25.
61. Mulligan RC. The basic science of gene therapy. Science 1993; 260: 926-32.
62. Naldini L, Blömer U, Gallay P et al. In vivo delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-7.
63. 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 USA 1994; 91: 2567-71.
64. Cornetta K, Morgan RA, Anderson WF. Safety issues related to retroviral-mediated gene transfer in humans. Human Gen Ther 1991; 2: 5-14.
65. Danos O, Mulligan R. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc Natl Acad Sci USA 1988; 85: 6460-4.
66. Horwitz MS. Adenoviruses. In: Fields BN, Knipe DM, et al. (eds). Virology, 2nd edn. New York: Raven Press, 1990: 1723-41.
67. Wilson JM. Adenovirus as gene-delivery vehicles. N Engl J Med 1996; 334: 1185-7.
68. Fisher KJ, Choi H, Burda J, Chen SJ, Wison JM. Recombinant adenovirus deleted of all genes for gene therapy of cystic fibrosis. Virology 1996; 217: 11-22.
69. Wang Q, Finer MH. Second-generation adenovirus vectors. Nat Med 1996; 2: 714-16.
70. Eming SA, Morgan JR. Methods for use of genetically-modified keratinocytes in gene therapy. In: Methods in Molecular Biology, Gene Therapy Protocols (in press).
71. Gerrard AJ, Hudson DL, Brownlee GG, Watt FM. Towards gene therapy for haemophilia B using primary human keratinocytes. Nat Genet 1993; 3: 180-3.
72. Lee JI, Taichman LB. Transient expression of a transfected gene in cultured epidermal keratinocytes: implications for future studies. J Invest Dermatol 1989; 92: 267-71.
73. Fenjves ES, Smith J, Zaradic S, Taichman LB. Systemic delivery of secreted protein by grafts of epidermal keratinocytes: Prospects for keratinocyte gene therapy. Hum Gene Ther 1994; 5: 1241-8.
74. Hoeffler WK, Wang CK, Matsui C et al. Phenotypic reversion of junctional epidermolysis bullosa keratinocytes by introduction of a therapeutic laminin 5 chain gene. J Invest Dermatol 1995; 104: 597.
75. Terasaki K, Kanzaki T, Oku T, Shinagawa A. Recombinant human tissue inhibitor of metalloproteinase-2 (rh-TIMP-2) accelerated wound healing. J Invest Dermatol 1996; 107: 517.
76. Qin L, Chavin KD, Ding Y, Woodward JE, Favaro JP, Lin J, Bromberg JS. Gene transfer for transplantation: prolongation of allograft survival with TGF-β1. Ann Surg 1994; 220: 508-8.
77. Amadio PC. Tendon and Ligament. In: Cohen IK, Diegelmann RF, Lindblad WJ (eds). Wound Healing, 1992: 384-95.
78. DesRosiers EA, Yahia L, Rivard CH. Proliferative and matrix synthesis response of canine anterior cruciate ligament fibroblasts submitted to combined growth factors. J Orthop Res 1996; 14: 200-8.
79. Howell TH, Martuscelli G, Oringer J. Polypeptide growth factors for periodontal regeneration. Curr Opin Periodontol 1996; 3: 149-56.
80. Nakamura N, Horibe S, Matsumoto N et al. Transient introduction of a foreign gene into healing rat patellar ligament. J Clin Invest 1996; 97: 226-31.
81. Williams SK, Jarrell BE. Tissue-engineered vascular grafts. Nat Med 1996; 2: 32-4.
82. Ziegler T, Nerem RM. Tissue engineering a blood vessel: Regulation of vascular biology by mechanical stresses. J Cell Biochem 1994; 56: 204-9.
83. Williams SK. Endothelial cell transplantation. Cell Transplant 1995; 4: 401-10.
84. Dichek DA, Anderson J, Kelly AB, Hanson SR, Harker LA. Enhanced in vivo antithrombotic effects of endothelial cells expressing recombinant plasminogen activators transduced with retroviral vectors. Circulation 1996; 93: 301-9.
85. Zoldhelyi P, McNatt J, Xu XM et al. Prevention of arterial thrombosis by adenovirus-mediated transfer of cyclooxygnase gene. Circulation 1996; 93: 10-17.
86. Muhlhauser J, Pili R, Merrill MJ et al. In vivo angiogenesis induced by recombinant adenovirus vectors coding either for secreted or nonsecreted forms of cidic fibroblast growth factor. Hum Gene Ther 1995; 6: 1457-65.
87. Mulhauser J, Merrill MJ, Pili R et al. VEGF165 expressed by a replication-deficient recombinant adenovirus vector induces angiogenesis in vivo. Circ Res 1995; 77: 1077-86.
88. Brittberg M, Lindahl A, Nilsson A, Ohlsson C, Isaksson O, Peterson L. Treatment of deep cartilage defects in the knee with autologous chondrocyte transplantation. N Engl J Med 1994; 331: 889-95.
89. Freed LE, Marquis JC, Nohria A, Emmanual J, Mikos AG, Langer R. Neocartilage formation in vitro and in vivo using cells cultured on synthetic biodegradable polymers. J Biomed Mater Res 1993; 27: 11-23.
90. Wornom IL, Buchman SR. Bone and Cartilaginous Tissue. In: Cohen IK, Diegelmann RF, Lindblad WJ (eds). Wound Healing, 1992: 356-83.
91. Shida J, Jingushi S, Izumi T, Iwaki A, Sugioka Y. bFGF stimulates articular cartilage enlargement in young rats in vivo. J Orthop Res 1996; 14: 265-72.
92. Fujisate T, Sajiki T, Liu O, Ikada Y. Effects of bFGF on cartilage regeneration in chondrocyte-seeded collagen sponge scaffold. Biomaterials 1996; 17: 155-62.
93. Baragi VM, Renkiewicz RR, Jordan H, Bonadio J, Hartman JW. Transplantation of transduced chondrocytes protects articular cartilage form interleukin 1-induced extracellular matrix degradation. J Clin Invest 1995; 96: 2454-60.