Vectors for gene therapy of skin diseases;Vektoren für die Gentherapie von Hautkrankheiten

JDDG - Journal of the German Society of Dermatology

Volumn 8, Issue 8, 2010, Pages 582-590

  Pfützner, Wolfgang ^a  

^a PHILIPPS UNIVERSITY MARBURG   (Germany)

Author keywords

adeno associated virus; adenovirus; gene therapy; genodermatosis; plasmid; retrovirus

Indexed keywords

ADENOVIRUS VECTOR; PARVOVIRUS VECTOR; PLASMID VECTOR; RETROVIRUS VECTOR;

ARTICLE; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENE VECTOR; HUMAN; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; SKIN DISEASE; VIRAL GENE DELIVERY SYSTEM;

EID: 77954854583     PISSN: 16100379     EISSN: 16100387     Source Type: Journal    
DOI: 10.1111/j.1610-0387.2010.07435.x     Document Type: Article

References (64)

1. Wolff I, Malone R, Williams P, Chong W, Ascadi G, Jani A, Felgner P. Direct gene transfer into mouse muscle in vivo. Science 1990 247 : 1465 1468.
2. Williams R, Johnston S, Riedy M, De Vit M, McElligott S, Sanford J. Introduction of foreign genes into tissues of living mice by DNA-coated micropo-jectiles. Proc Natl Acad Sci USA 1991 88 : 2726 2730.
3. Hengge U, Chan F, Foster R, Walker P, Vogel J. Cytokine gene expression in epidermis with biological effects following injection of naked DNA. Nature Genetics 1995 10 : 161 166.
4. 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.
5. Raghavachari N, Fahl W. Targeted gene delivery to skin cells in vivo: a comparative srudy of liposomes and polymers as delivery vehicles. J Pharm Sci 2002 91 : 615 622.
6. Meykadeh N, Mirmohammadsadegh A, Wang Z, Basner-Tschakarjan E, Hengge U. Topical application of plasmid DNA to mouse and human skin. J Mol Med 2005 83 : 897 903.
7. Liu L, Watabe S, Yang J, Hamajima K, Ishii N, Hagiwara E, Onari K, Xin K, Okuda K. Topical application of HIV DNA vaccine with cytokine-expression plasmids induces strong antigen-specific immune responses. Vaccine 2002 20 : 42 8.
8. Shaker D, Sloat B, Le U, Löhr C, Yanasam N, Fischer K, Cui Z, Immunization by application of DNA vaccine onto a skin area wherein the hair follicles have been induced into ana-gen-onset stage. Mol Ther 2007 15 : 2037 2043.
9. Falo L. Targeting the skin for genetic immunization. Proc Assoc Am Phys 1998 111 : 211 219.
10. Hengge U. Gene therapy progress and prospects: the skin - easily accessible, but still far away. Gene Ther 2006 13 : 1555 1563.
11. Rosenberg S, Yang J, Sherry R, Hwu P, Topalian S, Schwartzentruber D, Restifo N, Haworth L, Seipp C, Freezer L, Morton K, Mavroukakis S, White D. Inability to immunize patients with metastatic melanoma using plasmid DNA encoding the gp100 melanoma-melanocyte antigen. Hum Gene Ther 2003 14 : 709 714.
12. Weber J, Boswell W, Smith J, Hersh E, Snively J, Diaz M, Miles S, Liu X, Obrocea M, Qui Z, Bot A. Phase I trial of intranodal injection of a melan-A/MART-1 DNA plasmid vaccine in patients with stage IV melanoma. J Immunother 2008 31 : 215 223.
13. Mahvi D, Henry M, Albertini M, Weber S, Meredith K, Schalch H, Rakhmilevich A, Hank J, Sondel P. Intratumoral injection of IL-12 plas-mid-DNA - results of a phase I/Ib clinical trial. Cancer Gene Ther 2007 14 : 717 723.
14. Heinzerling L, Burg G, Dummer R, Maier T, Oberholzer P, Schultz J, Elzaouk L, Pavlovic J, Moelling K. Intratumoral injection of DNA encoding human interleukin 12 into patients with meta-static melanoma: clinical efficacy. Hum Gene Ther 2005 16 : 35 48.
15. Schneeberger A, Wagner C, Zemann A, Lührs P, Kutil R, Goos M, Stingl G, Wagner S. CpG motifs are efficient adjuvants for DNA cancer vaccines. J Invest Dermatol 2004 123 : 371 379.
16. Siegmund-Schultze N. Immunthera-pien gegen Krebs. Die klinische An-wendung rückt näher. Deutsches Ärzte-blatt 2009 106 : 2069 2073.
17. Heller L, Jaroszeski M, Coppola D, Heller R. Comparison of electrically mediated and liposome-complexed plasmid DNA delivery to the skin. Ge-net Vacc Ther 2008 6 : 16 23.
18. Hüttinger C, Hirschberger J, Jahnke A, Köstlin R, Brill T, Plank C, Küchenhoff H, Krieger S, Schillinger U. Neoadju-vantgene delivery of feline granulocyte-macrophage colony-stimulating factor using magnetofection for the treatment of feline fibrosarcomas: a phase I trial. J Gene Med 2002 10 : 665 667.
19. Daud A, De Conti R, Andrews S, Urbas P, Riker A, Sondak V, Munster P, Sullivan D, Ugen K, Messina J, Heller R. Phase I trial of interleukin-12 plasmid electroporation in patients with metatstatic melanoma. J Clin Oncol 2008 26 : 5896 5903.
20. Ortiz-Urda S, Thyagarajan B, Keene D, Lin Q, Calos M, Khavari P. fC31 inte-grase-mediated nonviral genetic correction of junctional epidermolysis bullosa. Hum Gene Ther 2003 14 : 923 928. (Pubitemid 36693800)
21. Ortiz-Urda S, Lin Q, Yant S, Keene D, Kay M, Khavari P. Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer. Gene Ther 2003 10 : 1099 1104.
22. Hackett C, Geurts A, Hackett P. Predicting preferential DNA vector insertion sites: implications for functional genomics and gene therapy. Genome Biol 2007 8 : S1 S12.
23. Hodgson C. Retrovirus-derived vectors. In : Hodgson C : Retro-Vectors for human gene therapy.
24. Verma I, Weitzman M. Gene therapy: twenty-first century medicine. Annu Rev Biochem 2005 74 : 711 738.
25. Friedmann T. The origins, evolution, and directions of human gene therapy. In : Friedmann T : The development of human gene therapy.
26. Rosenberg S, Aebersold P, Cornetta K, Kasid A, Morgan R, Moen R, Karson E, Lotze M, Yang J, Topalian S. Gene transfer into humans - immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990 323 : 570 578.
27. Edelstein M, Abedi M, Wixon J. Gene therapy clinical trials worldwide to 2007 - an update. J Gene Med 2007 9 : 833 842.
28. Mavilio F, Pellegrini G, Ferrari S, Di Nunzio F, Di Iorio E, Recchia A, Maruggi G, Ferrari G, Provasi E, Bonini C, Capurro S, Conti A, Magnoni C, Giannetti A, De Luca M. Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat Med 2006 12 : 1397 1402.
29. Naldini L, Verma I. Lentiviral vectors. In : Friedmann T : The development of human gene therapy.
30. Kühn U, Terunuma A, Pfützner W, Foster R, Vogel J. In vivo assessment of gene delivery to keratinocytes by lenti-viral vectors. J Virology 2002 76 : 1496 1504.
31. Kolodka T, Garlick J, Taichman L. 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.
32. Miller D, Adam M, Miller A. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 1990 10 : 4239 4242.
33. Romano G, Marino I, Pentimalli F, Adamo V, Giordano A. Insertional mutagenesis and development of malignancies induced by integrating gene delivery systems: implications for the design of safer gene-based interventions in patients. Drug News Perspect 2009 22 : 185 196.
34. Lauf S, Nagy K, Giordano F, Hotz-Wagenblatt A, Zeller W, Fruehauf S. Insertion of retroviral vectors in NOD/ SCID repopulating human peripheral blood progenitor cells occurs preferentially in the vicinity of transcription start regions and in introns. Mol Ther 2004 10 : 874 888.
35. Schroeder A, Shinn P, Chen H, Berry C, Ecker J, Bushman F. HIV-I integration in the humane genome favors active genes and local hotspots. Cell 2002 110 : 521 529.
36. McCormack M, Rabbits T. Activation of the T-cell oncogene LMO2 after gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2004 350 : 913 922. (Pubitemid 38252539)
37. Ott M, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins N, Copeland N, Lüthi U, Hassan M, Trasher A, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granuloma-tous disease by gene therapy, augmented by insertinal activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006 5 : 401 409.
38. Porteus M, Connelly J, Pruett S. A look to future directions in gene therapy research for monogenic diseases. PLoS Genetics 2006 2 : 1285 1292.
39. Choate K, Medalie D, Morgan J, Khavari P. Corrective gene transfer in the human skin disorder lamellar icht-hyosis. Nat Med 1996 2 : 1263 1267.
40. Freiberg R, Choate K, Deng H, Alperin E, Shapiro L, Khavari P. A model of corrective gene transfer in X-linked ichthyo-sis. Hum Mol Genet 1997 6 : 933 937.
41. Robbins P, Lin Q, Goodnough J, Tian H, Chen X, Khavari P. In vivo restoration of laminin 5 beta 3 expression and function in junctional epidermolysis bullosa. Proc Natl Acad Sci USA 2001 98 : 5193 5198.
42. Woodley D, Keene D, Atha T, Huang Y, Ram R, Kasahara N, Chen M. Intra-dermal injection of lentiviral vectors corrects regenerated human dystrophic epidermolysis bullosa skin tissue in vivo. Mol Ther 2004 10 : 318 326.
43. Baek S, Lin Q, Robbins P, Fan H Khavari P. Sustainable systemic delivery via a single injection of lentivirus into human skin tissue. Hum Gen Ther 2001 12 : 1551 1558.
44. White S, Page S, Margaritis P, Brownlee G. Long-term expression of human clotting factor IX from retrovirally transduced primary human keratinocytes in vivo. Hum Gene Ther 1998 9 : 1187 1195.
45. Therrien JP, Kim SM, Terunuma A, Qin Y, Tock CL, Pfützner W, Ohyama M, Schnermann J, Vogel JC. A gene therapy approach for long-term normalization of blood pressure in hypertensive mice by ANP-secreting human skin grafts. Proc Natl Acad Sci U S A. 2010 Jan 19 107 : 1178 1183.
46. Scheidemann F, Loeser M, Kromminga A, Therrien J, Vogel J, Pfutzner W. The skin as a biofactory for secretion of erythropoietin: potential of genetically modified keratinocytes and fibroblasts. Exp Dermatol 2008 17 : 481 488.
47. Christensen R, Güttler F, Jensen T. Comparison of epidermal keratinocytes and dermal fibroblasts as potential target cells for somatic gene therapy of phenylketonuria. Mol Gen Met 2002 76 : 313 318.
48. Zufferey R, Dull R, Mandel R, Bukovsky A, Quiroz D, Naldini L, Trono D. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virology 1998 72 : 9873 9880.
49. Siprashvili Z, Khavari P. Lentivectors for regulated and reversible cutaneous gene delivery. Mol Ther 2004 9 : 93 100.
50. Pfützner W. Retroviral bicistronic vectors. Drug News Pers 2008 21 : 473 480.
51. Scheidemann F, Therrien J, Pfützner W. Selectable bicistronic vectors in skin gene therapy. Arch Derm Res 2008 300 : 415 423.
52. Pfützner W, Terunuma A, Tock C, Snead E, Kolodka T, Gottesman M, Taichman I, Vogel J. Topical selection of keratinocytes transduced with the multidrug resistance gene (MDR1) can sustain and enhance transgene expression in-vivo. Proc Natl Acad Sci U S A. 2002 99 : 13096 13101.
53. Samulski R, Sally M, Muzyczka N. Adebo-associated viral vectors. In : Friedmann T : The development of human gene therapy.
54. Braun-Falco M, Doenecke A, Smola H, Hallek M. Efficient gene transfer into human keratinocytes with recombinant adeno-associated virus vectors. Gene Ther 1999 6 : 432 441.
55. Hengge U, Mirmohammasadegh A. Adeno-associated virus expresses trans-genes in hair follicles and epidermis. Mol Ther 2000 2 : 188 194.
56. Nakai H, Wu X, Fuess S, Storm T, Munroe D, Montini E, Burgess S, Grompe M, Kay M. Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. J Virol 2005 79 : 3606 3614.
57. Donsante A, Miller D, Li Y, Vogler C, Brunt E, Russell D, Sands M. AAV vector integration sites in mouse hepa-tocellular carcinoma. Science 2007 317 : 477.
58. Haug S, Braun-Falco M. Restoration of fatty aldehyde dehydrogenase deficiency in Sjögren-Larsson syndrome. Gene Ther 2006 13 : 1021 1026.
59. Sun L, Li J, Xiao X. Overcoming adenoassociated virus vector size limitation through viral DNA heterodime-rization. Nat Med 2000 6 : 599 602.
60. Crombleholme T. Adenoviral-mediated gene transfer in wound healing. Wound Rep Reg 2000 8 : 460 472.
61. Douglas J. Adenoviral vectors for gene therapy. Mol Biotechnol 2007 36 : 71 80.
62. Palmer D, Ng P. Helper-dependent adenoviral vectors for gene therapy. Hum Gene Ther 2005 16 : 1 16.
63. Margolis D, Morris L, Papadopoulos M, Weinberg L, Filip J, Lang S, Vaikunth S, Cromblehome T. Phase I study of H5.020CMV.PDGF-beta to treat venous leg ulcer disease. Mol Ther 2009 17 : 1822 1829.
64. Yoo G, Moon J, Le Blanc M, Lonardo F, Kim H, Hanna E, Tsue T, Valentino J, Ensley J, Wolf G. A phase 2 trial of surgery with perioperative INGN 201 (Ad5CMV-p53) gene therapy followed by chemoradiotherapy for advanced, resectable squamous cell carcinoma of the oral cavity, oropharynx, hypopharynx, and larynx. Arch Otolaryngol Head Neck Surg 2009 135 : 869 874.