Advances in skin gene therapy

Expert Opinion on Investigational Drugs

Volumn 9, Issue 9, 2000, Pages 2069-2083

  Pfutzner, W ^a   Vogel, J C ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

Author keywords

DNA vaccination; Epidermis; Genetic skin diseases; Skin gene therapy

Indexed keywords

ANTIGESTAGEN; COLCHICINE; DNA; GAMMA INTERFERON; GOLD; OLIGONUCLEOTIDE; RAPAMYCIN; TETRACYCLINE; VIRUS DNA; VIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; CELL TYPE; DENDRITIC CELL; DNA MODIFICATION; DRUG ACTIVITY; EPIDERMIS; GENE EXPRESSION; GENE EXPRESSION REGULATION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; HUMAN; HUMAN CELL; HUMAN TISSUE; IN VITRO STUDY; IN VIVO STUDY; INFECTION; KERATINOCYTE; MELANOCYTE; METABOLIC DISORDER; MOUSE; NONHUMAN; REVIEW; SKIN; SKIN DISEASE; STEM CELL; TRANSGENE; TUMOR;

EID: 0033863369     PISSN: 13543784     EISSN: None     Source Type: Journal    
DOI: 10.1517/13543784.9.9.2069     Document Type: Review

References (115)

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2. Prolonged occlusion in the treatment of psoriasis: A clinical and immunohistologic study
3. Human gene therapy comes of age
4. The basic science of gene therapy
5. Human gene therapy
6. Keratinocyte gene therapy
7. Epithelial gene therapy
8. Epidermis: An attractive target tissue for gene therapy
9. Therapeutic gene delivery to the skin
10. Corrective gene transfer in the human skin disorder lamellar ichthyosis
11. Corrective gene transfer of keratinocytes from patients with junctional epidermolysis bullosa restores assembly of hemidesmosomes in reconstructed epithelia
12. BP180 gene delivery in junctional epidermolysis bullosa
13. Evidence for an epidermal cytokine network
14. Epidermal signal transduction and transcription factor activation in activated keratinocytes
15. Towards gene therapy for haemophilia B using primary human keratinocytes
16. Genetically modified human keratinocytes overexpressing PDGF-A enhance the performance of a composite skin graft
17. Retrovirus mediated gene transfer of ornithine-δ-aminotransferase into keratinocytes from gyrate atrophy patients
18. Localized in vivo genotypic and phenotypic correction of the albino mutation in skin by RNA-DNA oligonucleotide
19. Adenovirus-mediated expression of melanoma antigen gp75 as immunotherapy for metastatic melanoma
20. Immune response in human melanoma after transfer of an allogeneic class I major histocompatibility complex gene with DNA-liposome complexes
21. Genetic immunization with glycoprotein 63 cDNA results in a helper T cell Type 1 immune response and protection in a murine model of leishmaniasis
22. IL-12-engineered dendritic cells serve as effective tumor vaccine adjuvants in vivo
23. Immunization through dermal delivery of protein-encoding DNA: A role for migratory dendritic cells
24. Probing keratinocyte and differentiation specificity of the human K5 promoter in vitro and in transgenic mice
25. Transgenic studies with a keratin promoter-driven growth hormone transgene: Prospects for gene therapy
26. Tissue- and stratum-specific expression of the human involucrin promoter in transgenic mice
27. Regulated gene expression systems
28. Progress in transcriptionally targeted and regulatable vectors for gene therapy
29. Transduction of a preselected population of human epidermal stem cells: Consequences for gene therapy
30. Introduction of foreign genes into tissues of living mice by DNA-coated microprojectiles
31. Efficient delivery of transgenes to human hair follicle progenitor cells using topical lipoplex
32. Liposome-mediated gene transfer and expression via the skin
33. Immunization via hair follicles by topical application of naked DNA to normal skin
34. In vivo electrically mediated protein and gene transfer in murine melanoma
35. In vivo gene introduction into keratinocytes using jet injection
36. In vivo gene transfer to skin and wound microseeding
37. Puncture-mediated gene transfer to the skin
38. Expression of naked DNA in human, pig and mouse skin
39. Cytokine gene expression in epidermis with biological effects following injection of naked DNA
40. Direct cutaneous gene delivery in a human genetic skin disease
41. Liposome-mediated gene transfer into human basal cell carcinoma
42. Targeting the skin for genetic immunization
43. DNA-based immunization by in vivo transfection of dendritic cells
44. Intradermal gene immunization: The possible role of DNA uptake in the induction of cellular immunity to viruses
45. Preferential induction of a Th1 immune response and inhibition of specific IgE antibody formation by plasmid DNA immunization
46. DNA immunization: Induction of higher avidity antibody and effect of route on T cell cytotoxicity
47. Immunostimulatory DNA sequences necessary for effective intradermal gene immunization
48. Immunostimulatory oligodeoxynucleotides containing the CpG motif are effective as immune adjuvants in tumor antigen immunization
49. Immunostimulatory oligodeoxynucleotides promote protective immunity and provide systemic therapy for leishmaniasis via IL-12 and IFN-γ-dependent mechanisms
50. Correction of the mutation responsible for sickle cell anemia by an RNA-DNA oligonucleotide
51. In vivo site-directed mutagenesis of the Factor IX gene by chimeric RNA/DNA oligonucleotides
52. A model for keratinocyte gene therapy: Preclinical and therapeutic considerations
53. In vivo gene therapy with interleukin-12 inhibits primary vascular tumor growth and induces apoptosis in a mouse model
54. In vivo transfer and expression of a human epidermal growth factor gene accelerates wound repair
55. Gene gun-based nucleic acid immunization. Elicitation of humoral and cytotoxic T lymphocyte responses following epidermal delivery of nanogram quantities of DNA
56. Different T helper cell types and antibody isotypes generated by saline and gene gun DNA immunization
57. Perifollicular transgenic expression of human interleukin-1 receptor antagonist protein following topical application of novel liposome-plasmid DNA formulations in vivo
58. Viral vectors for gene therapy
59. Virus vector design in gene therapy
60. Topical application of viral vectors for epidermal gene transfer
61. Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs
62. Cellular immune responses of healthy individuals to intradermal administration of an E1-E3- adenovirus gene transfer vector
63. Vaccination onto bare skin
64. Ex vivo and in vivo adenovirus-mediated gene therapy strategies induce a systematic anti-tumor immune defense in the B16 melanoma model
65. A phase I study of intralesional administration of an adenovirus vector expressing the HSV-1 thymidine kinase gene (AdV.RSV-TK) in combination with escalating doses of ganciclovir in patients with cutaneous metastatic malignant melanoma
66. Use of retroviral vectors for gene transfer and expression
67. Retroviral vectors. From laboratory tools to molecular medicine
68. In vivo transduction of mouse epidermis with recombinant retroviral vectors: Implications for cutaneous gene therapy
69. Lentiviral vectors and gene therapy
70. Reconstruction of human epidermis on de-epidermized dermis: Expression of differentiation-specific protein markers and lipid composition
71. Effect of TGF-β1 on re-epithelialization of human keratinocytes in vitro: An organotypic model
72. High-titer packaging cells producing recombinant retroviruses resistant to human serum
73. Evidence for Keratinocyte stem cells in vitro: Long term engraftment and persistence of transgene expression from retrovirus-transduced keratinocytes
74. A model of corrective gene transfer in X-linked ichthyosis
75. Functional retroviral vector for gene therapy of xeroderma pigmentosum group D patients
76. Retrovirus-mediated DNA repair gene transfer into xeroderma pigmentosum cells: Perspectives for a gene therapy
77. Long-term expression of human clotting Factor IX from retrovirally transduced primary human keratinocytes in vivo
78. Expression of an exogenous growth hormone gene by transplantable human epidermal cells
79. Systemic delivery of secreted protein by grafts of epidermal keratinocytes: Prospects for keratinocyte gene therapy
80. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors
81. Sustainability of keratinocyte gene transfer and cell survival in vivo
82. Optimised retroviral infection of human epidermal keratinocytes: Long-term expression of transduced integrin gene following grafting on to SCID mice
83. Loss of expression of a retrovirus-transduced gene in human keratinocytes
84. Sustainable cutaneous gene delivery
85. Loss of retroviral gene expression in bone marrow reconstituted mice correlates with down-regulation of gene expression in long-term culture initiating cells
86. Liver-directed gene therapy: Prospects and problems
87. Gene silencing as a threat to the success of gene therapy
88. Adeno-associated viral vectors for gene transfer and gene therapy
89. Stable gene transfer and expression of human blood coagulation Factor IX after intramuscular injection of recombinant adeno-associated virus
90. Recombinant adeno-associated viral vectors mediate long-term transgene expression in muscle
91. Efficient gene transfer into human keratinocytes with recombinant adeno-associated virus vectors
92. Targeting of retroviral vectors for gene therapy
93. In vitro and in vivo targeting of gene expression to melanoma cells
94. Targeted therapy for malignant melanoma
95. A safe, effective in vivo gene therapy for melanoma using tyrosine promoter-driven cytosine deaminase gene
96. Systemic gene therapy of murine melanoma using tissue specific expression of the HSVtk gene involves an immune component
97. Circulating human Factor IX produced in keratin-promoter transgenic mice: A feasibility study for gene therapy of haemophilia B
98. Skin hyperkeratosis and papilloma formation in transgenic mice expressing a ras oncogene from a suprabasal keratin promoter
99. Augmentation of melanoma-specific gene expression using a tandem melanocyte-specific enhancer results in increased cytotoxicity of the purine nucleoside phosporylase gene in melanoma
100. Selection of keratinocytes transduced with the multidrug resistance gene in an in vitro skin model presents a strategy for enhancing gene expression in vivo
101. Epigenetics: Regulation through repression
102. A new family of murine retroviral vectors with extended multiple cloning sites for gene insertion
103. Improved gene expression upon transfer of the adenosine deaminase minigene outside the transcriptional unit of a retroviral vector
104. Existence of slow-cycling limbal epithelial basal cells that can be preferentially stimulated to proliferate: Implications on epithelial stem cells
105. Stem cell patterning and fate in human epidermis
106. Epidermal turnover time - A re-examination
107. Selection and extended growth of murine epidermal stem cells in culture
108. A 5'-upstream region of abovine keratine 6 gene confers tissue-specific expression and hyperproliferation-related induction in transgenic mice
109. Use of monospecific antisera and cRNA probes to localize the major changes in keratin expression during normal and abnormal epidermal differentiation
110. Changes in keratinocyte maturation during wound healing
111. Altered epidermal cell growth control in vivo by inducible expression of transforming growth factor β1 in the skin of transgenic mice
112. Development of gene-switch transgenic mice that inducibly express transforming growth factor β1 in the epidermis
113. Characterization of an inducible, epidermal-specific knockout-system: Differential expression of lacZ in different cre reporter mouse strains
114. A humanized system for pharmacologic control of gene expression
115. Inducible gene expression and protein translocation using nontoxic ligands identified by a mammalian three-hybrid screen