Gene therapy for skin diseases

Cold Spring Harbor Perspectives in Medicine

Volumn 4, Issue 4, 2014, Pages

  Gorell, Emily ^a   Nguyen, Ngon ^a   Lane, Alfred ^a   Siprashvili, Zurab ^a  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

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

ARTICLE; CONGENITAL PACHYONYCHIA; EPIDERMOLYSIS BULLOSA; EPIDERMOLYSIS BULLOSA DYSTROPHICA; EPIDERMOLYSIS BULLOSA SIMPLEX; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE MUTATION; GENE THERAPY; GENE TRANSFER; GENETIC MANIPULATION; GENETIC TRANSDUCTION; HUMAN; INFLAMMATION; LAMELLAR ICHTHYOSIS; MELANOMA; SJOEGREN LARSSON SYNDROME; SKIN DISEASE; SKIN PERMEABILITY; WOUND HEALING; XERODERMA PIGMENTOSUM;

EID: 84897401101     PISSN: None     EISSN: 21571422     Source Type: Journal    
DOI: 10.1101/cshperspect.a015149     Document Type: Article

References (113)

1. Akiyama M., Sugiyama-Nakagiri Y, Sakai K, McMillan JR, Goto M., Arita K, Tsuji-Abe Y, Tabata N, Matsuoka K, Sasaki R., et al. 2005. Mutations in lipid transporter ABCA12 in harlequin ichthyosis and functional recovery by corrective gene transfer. J Clin Invest 115: 1777.
2. Al Aboud K, Al Hawsawi K, Ramesh V. 2003. Kindler syndrome: Two additional features. Dermatol Online J 9: 20.
3. Almaani N., Nagy N, Liu L, Dopping-Hepenstal PJ, Lai-Cheong J. E, Clements SE, Techanukul T, Tanaka A, Mellerio JE, McGrath JA. 2010. Revertant mosaicism in recessive dystrophic epidermolysis bullosa. J Invest Dermatol 130: 1937-1940.
4. Barrientos S., Stojadinovic O, Golinko MS, Brem H, Tomic-Canic M. 2008. Perspective article: Growth factors and cytokines in wound healing. Wound Repair Regen 16: 585-601.
5. Bell E., Ehrlich HP, Buttle DJ, Nakatsuji T. 1981. Living tissue formed in vitro and accepted as skin-equivalent tissue of full thickness. Science 211: 1052-1054.
6. Bowden P.E. 2011. Gene therapy for keratin genodermatoses: Striving forward but obstacles persist. J Invest Dermatol 131: 1403-1405.
7. Boztug K., Schmidt M, Schwarzer A, Banerjee PP, Díez IA, Dewey R. A, Böhm M, Nowrouzi A, Ball CR, Glimm H, et al. 2010. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med 363: 1918-1927.
8. Braun-Falco M, Doenecke A, Smola H, Hallek M. 1999. Efficient gene transfer into human keratinocytes with recombinant adeno-associated virus vectors. Gene Ther 6: 432-441.
9. Brem H., Kodra A, Golinko M, Entero H, Stojadinovic O, Wang V., Sheahan C, Weinberg A, Woo SLC, Ehrlich HP, et al. 2009. Mechanism of sustained release of vascular endothelial growth factor in accelerating experimental diabetic healing. J Invest Dermatol 129: 2275-2287.
10. Burke J. F, Yannas IV, Quinby WC, Bondoc CC, Jung WK. 1981. Successful use of a physiologically acceptable artificial skin in the treatment of extensive burn injury. Ann Surg 194: 413-428.
11. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, et al. 2000. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669-672.
12. Chamcheu J. C, Wood GS, Siddiqui IA, Syed DN, Adhami VM, Teng JM, Mukhtar H. 2012. Progress towards genetic and pharmacological therapies for keratin genodermatoses: Current perspective and future promise. Exp Dermatol 21: 481-489.
13. Chen Z. 2003. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 8: 495-500.
14. Chen M., Kasahara N, Keene DR, Chan L, Hoeffler WK, Finlay D., Barcova M, Cannon PM, Mazurek C, Woodley DT. 2002. Restoration of type VII collagen expression and function in dystrophic epidermolysis bullosa. Nat Genet 32: 670-675.
15. Chesnoy S., Lee P-Y, Huang L. 2003. Intradermal injection of transforming growth factor-b1 gene enhances wound healing in genetically diabetic mice. Pharm Res 20: 345-350.
16. Choate K. A, Medalie DA, Morgan JR, Khavari PA. 1996. Corrective gene transfer in the human skin disorder lamellar ichthyosis. Nat Med 2: 1263-1267.
17. Choate K. A, Lu Y, Zhou J, Choi M, Elias PM, Farhi A, Nelson-Williams C., Crumrine D, Williams ML, Nopper AJ, et al. 2010. Mitotic recombination in patients with ichthyosis causes reversion of dominant mutations in KRT10. Science 330: 94-97.
18. Cleaver J., Lam E, Revet I. 2009. Disorders of nucleotide excision repair: The genetic and molecular basis of heterogeneity. Nat Rev Genet 10: 756-768.
19. Cooray S., Howe SJ, Thrasher AJ. 2012. Retrovirus and lentivirus vector design and methods of cell conditioning. Methods Enzymol 507: 29-57.
20. Dallinger G., Puttaraju M, Mitchell LG, Yancey KB, Yee C, Klausegger A., Hintner H, Bauer JW. 2003. Development of spliceosome-mediated RNA trans-splicing (SMaRT) for the correction of inherited skin diseases. Exp Dermatol 12: 37-46.
21. Dang N., Murrell DF. 2008. Mutation analysis and characterization of COL7A1 mutations in dystrophic epidermolysis bullosa. Exp Dermatol 17: 553-568.
22. Darquet A. M, Cameron B, Wils P, Scherman D, Crouzet J. 1997. A new DNAvehicle for nonviral gene delivery: Supercoiled minicircle. Gene Ther 4: 1341-1349.
23. Dean H. J, Chen D. 2004. Epidermal powder immunization against influenza. Vaccine 23: 681-686.
24. de GruijlTD, van deVen R. 2012. Chapter six-Adenovirusbased immunotherapy of cancer: Promises to keep. In Advances in cancer research (ed. Curiel DT, Fisher PB), Vol. 115, pp. 147-220. Academic, San Diego, 2012.
25. De Luca M, Pellegrini G, Mavilio F. 2009. Gene therapy of inherited skin adhesion disorders: A critical overview. Br J Dermatol 161: 19-24.
26. Di W.-L, Larcher F, Semenova E, Talbot GE, Harper JI, Del Rio M., Thrasher AJ, Qasim W. 2011. Ex-vivo gene therapy restores LEKTI activity and corrects the architecture of netherton syndrome-derived skin grafts. Mol Ther 19: 408-416.
27. Ditto A. J, Shah PN, Yun YH. 2009. Non-viral gene delivery using nanoparticles. Exp Opin Drug Deliv 6: 1149-1160.
28. Dormond E., Kamen AA. 2011. Manufacturing of adenovirus vectors: Production and purification of helper dependent adenovirus. Methods Mol Biol 737: 139-156.
29. Dull T., Zufferey R, Kelly M, Mandel RJ, Nguyen M, TronoD, Naldini L. 1998. Athird-generation lentivirus vector with a conditional packaging system. J Virol 72: 8463-8471.
30. Dummer R., Rochlitz C, Velu T, Acres B, Limacher J-M, Bleuzen P., Lacoste G, Slos P, Romero P, Urosevic M. 2008. Intralesional adenovirus-mediated interleukin-2 gene transfer for advanced solid cancers and melanoma. Mol Ther 16: 985-994.
31. Edelstein M. L, Abedi MR, Wixon J. 2007. Gene therapy clinical trials worldwide to 2007-An update. J Gene Med 9: 833-842.
32. Endo M., Zoltick PW, Radu A, Qiujie J, Matsui C, Marinkovich PM, McGrath J, Tamai K, Uitto J, Flake AW. 2012. Early intra-amniotic gene transfer using lentiviral vector improves skin blistering phenotype in a murine model of Herlitz junctional epidermolysis bullosa. Gene Ther 19: 561-569.
33. Fan H., Lin Q, Morrissey GR, Khavari PA. 1999. Immunization via hair follicles by topical application of nakedDNA to normal skin. Nat Biotechnol 17: 870-872.
34. Fine J. D, Eady RA, Bauer EA, Briggaman RA, Bruckner-Tuderman L, Christiano A, Heagerty A, Hintner H, Jonkman MF, McGrath J, et al. 2000. Revised classification system for inherited epidermolysis bullosa: Report of the Second International ConsensusMeeting on Diagnosis and Classification of Epidermolysis Bullosa. JAmAcad Dermatol 42: 1051-1066.
35. Fontana R., Bregni M, Cipponi A, Raccosta L, Rainelli C, Maggioni D., Lunghi F, Ciceri F, Mukenge S, Doglioni C, et al. 2009. Peripheral blood lymphocytes genetically modified to express the self/tumor antigen MAGE-A3 induce antitumor immune responses in cancer patients. Blood 113: 1651-1660.
36. Gache Y., Baldeschi C, Del Rio M, Gagnoux-Palacios L, Larcher F., Lacour JP, Meneguzzi G. 2004. Construction of skin equivalents for gene therapy of recessive dystrophic epidermolysis bullosa. Hum Gene Ther 15: 921-933.
37. Gache Y., Pin D, Gagnoux-Palacios L, Carozzo C, Meneguzzi G. 2011. Correction of dog dystrophic epidermolysis bullosa by transplantation of genetically modified epidermal autografts. J Invest Dermatol 131: 2069-2078.
38. Gagnoux-Palacios L, Hervouet C, Spirito F, Roques S, Mezzina M, Danos O, Meneguzzi G. 2005. Assessment of optimal transduction of primary human skin keratinocytes by viral vectors. J Gene Med 7: 1178-1186.
39. Galeano M., Deodato B, Altavilla D, Cucinotta D, Arsic N, Marini H., Torre V, Giacca M, Squadrito F. 2003. Adenoassociated viral vector-mediated human vascular endothelial growth factor gene transfer stimulates angiogenesis and wound healing in the genetically diabetic mouse. Diabetologia 46: 546-555.
40. Ginn S. L, Alexander IE, Edelstein ML, Abedi MR, Wixon J. 2013. Gene therapy clinical trials worldwide to 2012-An update. J Gene Med 15: 65-77.
41. Gorell E. S, Tichy AL, Lane AT. 2011. Successful investigational new drug preparation without reinventing the wheel. J Invest Dermatol 131: 996-998.
42. Goto M., Sawamura D, Ito K, Abe M, Nishie W, Sakai K, Shibaki A., Akiyama M, Shimizu H. 2006a. Fibroblasts show more potential as target cells than keratinocytes in COL7A1 gene therapy of dystrophic epidermolysis bullosa. J Invest Dermatol 126: 766-772.
43. Goto M., Sawamura D, Nishie W, Sakai K, McMillan JR, Akiyama M., Shimizu H. 2006b. Targeted skipping of a single exon harboring a premature termination codon mutation: Implications and potential for gene correction therapy for selective dystrophic epidermolysis bullosa patients. J Invest Dermatol 126: 2614-2620.
44. Green H., Kehinde O, Thomas J. 1979. Growth of cultured human epidermal cells into multiple epithelia suitable for grafting. Proc Natl Acad Sci 76: 5665-5668.
45. Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP, Thrasher AJ, Wulffraat N, Sorensen R, Dupuis-Girod S, et al. 2002. Sustained correction of Xlinked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 346: 1185-1193.
46. Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E., Clappier E, Caccavelli L, Delabesse E, Beldjord K, et al. 2008. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118: 3132-3142.
47. Has C., Sparta' G, KiritsiD, Weibel L, Moeller A, Vega-Warner V, Waters A, He Y, Anikster Y, Esser P, et al. 2012. Integrin a3 mutations with kidney, lung, and skin disease. N Engl J Med 366: 1508-1514.
48. Haug S., Braun Falco M. 2005. Adeno-associated virus vectors are able to restore fatty aldehyde dehydrogenase-deficiency. Implications for gene therapy in Sjogren-Larsson syndrome. Arch Dermatol Res 296: 568-572.
49. Hengge U. R, Mirmohammadsadegh A. 2000. Adeno-associated virus expresses transgenes in hair follicles and epidermis. Mol Ther 2: 188-194.
50. Hengge U. R, Chan EF, Foster RA, Walker PS, Vogel JC. 1995. Cytokine gene expression in epidermis with biological effects following injection of naked DNA. Nat Genet 10: 161-166.
51. Hirsch T., von Peter S, Dubin G, Mittler D, Jacobsen F, Lehnhardt M, Eriksson E, Steinau H-U, Steinstraesser L. 2006. Adenoviral gene delivery to primary human cutaneous cells and burn wounds. Mol Med 12: 199-207.
52. Hirsch T., Spielmann M, Velander P, Zuhaili B, Bleiziffer O, Fossum M., Steinstraesser L, Yao F, Eriksson E. 2008. Insulin-like growth factor-1 gene therapy and cell transplantation in diabetic wounds. J Gene Med 10: 1247-1252.
53. Howe S. J, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M., Kempski H, Brugman MH, Pike-Overzet K, Chatters S. J, de Ridder D, et al. 2008. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 118: 3143-3150.
54. Johnson L. A, Morgan RA, Dudley ME, Cassard L, Yang JC, Hughes M. S, Kammula US, Royal RE, Sherry RM, Wunderlich JR, et al. 2009. Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen. Blood 114: 535-546.
55. Kardos T. J, Rabussay DP. 2012. Contactless magneto-permeabilization for intracellular plasmid DNA delivery invivo. Hum Vaccin Immunother 8: 1707-1713.
56. Kay M.A. 2011. State-of-the-art gene-based therapies: The road ahead. Nat Rev Genet 12: 316-328.
57. Khavari P. A, Rollman O, Vahlquist A. 2002. Cutaneous gene transfer for skin and systemic diseases. J Intern Med 252: 1-10.
58. Kim Y.-C, Prausnitz MR. 2011. Enabling skin vaccination using new delivery technologies. Drug Deliv Transl Res 1: 7-12.
59. Ko J., Jun H, Chung H, Yoon C, Kim T, Kwon M, Lee S, Jung S, Kim M, Park J. 2011. Comparison of EGF with VEGF non-viral gene therapy for cutaneous wound healing of streptozotocin diabetic mice. Diabetes Metab J 35: 226-235.
60. Kommareddy S., Baudner BC, Oh S, Kwon SY, Singh M, O'Hagan DT. 2012. Dissolvable microneedle patches for the delivery of cell-culture-derived influenza vaccine antigens. J Pharm Sci 101: 1021-1027.
61. Korge B. P, Krieg T. 1996. The molecular basis for inherited bullous diseases. J Mol Med 74: 59-70.
62. Kwon M. J, An S, Choi S, Nam K, Jung HS, Yoon CS, Ko JH, Jun H. J, Kim TK, Jung SJ, et al. 2012. Effective healing of diabetic skin wounds by using nonviral gene therapy based on minicircle vascular endothelial growth factor DNA and a cationic dendrimer. J GeneMed 14: 272-278.
63. Lai-Cheong JE, McGrath JA, Uitto J. 2011. Revertant mosaicism in skin: Natural gene therapy. Trends Mol Med 17: 140-148.
64. Larcher F., Dellambra E, Rico L, Bondanza S, Murillas R, Cattoglio C., Mavilio F, Jorcano JL, Zambruno G, Del Rio M. 2007. Long-term engraftment of single genetically modified human epidermal holoclones enables safety pre-assessment of cutaneous gene therapy. Mol Ther 15: 1670-1676.
65. Leachman S., Hickerson R, Schwartz M, Bullough E, Hutcherson S, Boucher K, Hansen CD, Eliason M, Srivatsa GS, Kornbrust D., et al. 2010. First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder. Mol Ther 18: 442-446.
66. Mali P., Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE, Norville J. E, Church GM. 2013. RNA-Guided Human Genome Engineering via Cas9. Science 339: 823-826.
67. Marchetto M. CN, Muotri A, Burns D, Friedberg E, Menck CFM. 2004. Gene transduction in skin cells: Preventing cancer in xeroderma pigmentosum mice. Proc Natl Acad Sci 101: 17759-17764.
68. Margolis D., Morris L, Papadopoulos M, Weinberg L, Filip J, Lang S., Vaikunth S, Crombleholme T. 2009. Phase I study of H5.020CMV. PDGF-b to treat venous leg ulcer disease. Mol Ther 17: 1822-1829.
69. Marinkovich M.P. 1993. The molecular genetics of basement membrane diseases. Arch Dermatol 129: 1557-1565.
70. Marinkovich M.P. 2012. Inherited epidermolysis bullosa. In Fitzpatrick's dermatology in general medicine (ed. Goldsmith LA, et al.), pp. 649-665. McGraw Hill, New York.
71. Mavilio F., Pellegrini G, Ferrari S, Di Nunzio F, Di Iorio E, Recchia A., Maruggi G, Ferrari G, Provasi E, Bonini C, et al. 2006. Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat Med 12: 1397-1402.
72. Miller A.D. 1992. Retroviral vectors. Curr Topic Microbiol Immunol 158: 1-24.
73. Miller D. L, Pislaru SV, Greenleaf JE. 2002. Sonoporation: MechanicalDNAdelivery by ultrasonic cavitation. Somat Cell Mol Genet 27: 115-134.
74. Mohammed A. J, AlAwaidy S, Bawikar S, Kurup PJ, Elamir E, Shaban M. MA, Sharif SM, van der Avoort HGAM, Pallansch MA, Malankar P, et al. 2010. Fractional doses of inactivated poliovirus vaccine in Oman. N Engl J Med 362: 2351-2359.
75. Moran N. 2012. First gene therapy nears landmark European market authorization. Nat Biotechnol 30: 807-809.
76. Morgan R. A, Dudley ME, Wunderlich JR, Hughes MS, Yang JC, Sherry RM, Royal RE, Topalian SL, Kammula US, Restifo N. P, et al. 2006. Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 314: 126-129.
77. Murauer E. M, Gache Y, Gratz IK, Klausegger A, Muss W, Gruber C., Meneguzzi G, Hintner H, Bauer JW. 2011. Functional correction of type VII collagen expression in dystrophic epidermolysis bullosa. J Invest Dermatol 131: 74-83.
78. O'Reilly M, Shipp A, Rosenthal E, Jambou R, Shih T, Montgomery M, Gargiulo L, Patterson A, Corrigan-Curay J. 2012. NIH oversight of human gene transfer research involving retroviral, lentiviral, and adeno-associated virus vectors and the role of the NIH recombinant DNA advisory committee. Methods Enzymol 507: 313-335.
79. Ortiz-Urda S, Thyagarajan B, Keene DR, Lin Q, Fang M, Calos M. P, Khavari PA. 2002. Stable nonviral genetic correction of inherited human skin disease. Nat Med 8: 1166-1170.
80. Ortiz-Urda S, Lin Q, Green C, Keene D, Marinkovich MP, Khavari P. 2003. Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue. J Clin Invest 111: 251-255.
81. Osborn M. J, Starker CG, McElroy AN, Webber BR, Riddle MJ, Xia L, DeFeo AP, Gabriel R, Schmidt M, Von Kalle C, et al. 2013. TALEN-based gene correction for epidermolysis bullosa. Mol Ther 21: 1151-1159.
82. Pabo C. O, Peisach E, Grant RA. 2001. Design and selection of novel CYS2HIS2 zinc finger proteins. Ann Rev Biochem 70: 313-340.
83. Paller A.S. 1996. The genetic basis of hereditary blistering disorders. Curr Opin Pediatr 8: 367-371.
84. Park H.-J, Lee J, Kim M-J, Kang T, Jeong Y, Um S, Cho S-W. 2012. Sonic hedgehog intradermal gene therapy using a biodegradable poly(b-amino esters) nanoparticle to enhance wound healing. Biomaterials 33: 9148-9156.
85. Pasmooij A. MG, Jonkman MF. 2012. First symposium on natural gene therapy of the skin. Exp Dermatol 21: 236-239.
86. Pasmooij A. MG, Garcia M, Escamez MJ, Nijenhuis AM, Azon A., Cuadrado-Corrales N, Jonkman MF, Del Rio M. 2010. Revertant mosaicism due to a second-site mutation in COL7A1 in a patient with recessive dystrophic epidermolysis bullosa. J Invest Dermatol 130: 2407-2411.
87. Pasmooij A. MG, Nijenhuis M, Brander R, Jonkman MF. 2012. Natural gene therapy may occur in all patients with generalized non-Herlitz junctional epidermolysis bullosa with COL17A1 mutations. J Invest Dermatol 132: 1374-1383.
88. Pearton M., Saller V, Coulman SA, Gateley C, Anstey AV, Zarnitsyn V., Birchall JC. 2012. Microneedle delivery of plasmid DNA to living human skin: Formulation coating, skin insertion and gene expression. J Control Release 160: 561-569.
89. Petek L., Fleckman P, Miller D. 2010. Efficient KRT14 targeting and functional characterization of transplanted human keratinocytes for the treatment of epidermolysis bullosa simplex. Mol Ther 18: 1624-1632.
90. Porteus M. H, Baltimore D. 2003. Chimeric nucleases stimulate gene targeting in human cells. Science 300: 763.
91. Raghavachari N., Fahl WE. 2002. Targeted gene delivery to skin cells in vivo: A comparative study of liposomes and polymers as delivery vehicles. J Pharm Sci 91: 615-622.
92. Regauer S., Seiler GR, Barrandon Y, Easley KW, Compton CC. 1990. Epithelial origin of cutaneous anchoring fibrils. J Cell Biol 111: 2109-2115.
93. Rheinwald J. G, Green H. 1975. Serial cultivation of strains of human epidermal keratinocytes: The formation of keratinizing colonies from single cells. Cell 6: 331-343.
94. Robbins P. F, Morgan RA, Feldman SA, Yang JC, Sherry RM, Dudley M. E, Wunderlich JR, Nahvi AV, Helman LJ, Mackall CL, et al. 2011. Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NYESO-1. J Clin Oncol 29: 917-924.
95. Roedl D., Oji V, Buters JTM, Behrendt H, Braun-Falco M. 2011. rAAV2-mediated restoration of LEKTI in LEKTIdeficient cells fromNetherton patients. J Dermatol Sci 61: 194-198.
96. Siprashvili Z., Nguyen NT, BezchinskyMY, MarinkovichMP, Lane A. T, Khavari PA. 2010. Long-term type VII collagen restoration to human epidermolysis bullosa skin tissue. Human Gene Ther 21: 1299-1310.
97. Smith F. JD, Morley SM, McLean WHI. 2004. Novel mechanism of revertant mosaicism in dowling-meara epidermolysis bullosa simplex. J Investig Dermatol 122: 73-77.
98. Titomirov A. V, Sukharev S, Kistanova E. 1991. In vivo electroporation and stable transformation of skin cells of newborn mice by plasmid DNA. Biochim Biophys Acta 1088: 131-134.
99. Tolmachov O.E. 2011. Building mosaics of therapeutic plasmid gene vectors. Curr Gene Ther 11: 466-478.
100. Uitto J., Pulkkinen L. 1996. Molecular complexity of the cutaneous basement membrane zone. Mol Biol Rep 23: 35-46.
101. Uitto J., Richard G. 2005. Progress in epidermolysis bullosa: From eponyms to molecular genetic classification. Clin Dermatol 23: 33-40.
102. Uitto J., Pulkkinen L, Christiano AM. 1994. Molecular basis of the dystrophic and junctional form of epidermolysis bullosa: mutations in the type VII collagen and kalinin (laminin-5) genes. J Invest Dermatol 103: 39S-45S.
103. van den Akker PC, Nijenhuis M, Meijer G, Hofstra RMW, Jonkman M., Pasmooij AMG. 2012. Natural gene therapy in dystrophic epidermolysis bullosa. Arch Dermatol 148: 213-216.
104. Vranckx J. J, Hoeller D, Velander PEM, Theopold CFP, Petrie N, Takedo A, Eriksson E, Feng Y. 2007. Cell suspension cultures of allogenic keratinocytes are efficient carriers for ex vivo gene transfer and accelerate the healing of full-thickness skin wounds by overexpression of human epidermal growth factor. Wound Repair Regen 15: 657-664.
105. Wally V., Brunner M, Lettner T, Wagner M, Koller U, Trost A, Murauer E. M, Hainzl S, Hintner H, Bauer JW. 2010. K14 mRNA reprogramming for dominant epidermolysis bullosa simplex. Hum Mol Genet 19: 4715-4725.
106. Wang H., Yang H, Shivalila Chikdu S, Dawlaty Meelad M, Cheng Albert W, Zhang F, Jaenisch R. 2013. One-step generation of mice carrying mutations in multiple genes by CRISPR/Cas-mediated genome engineering. Cell 153: 910-918.
107. Warrick E., Garcia M, Chagnoleau C, ChevallierO, Bergoglio V, Sartori D, Mavilio F, Angulo JF, Avril M-F, Sarasin A, et al. 2012. Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells. Mol Ther 20: 798-807.
108. Williams R. S, Johnston SA, Riedy M, DeVit MJ, McElligott SG, Sanford JC. 1991. Introduction of foreign genes into tissues of living mice by DNA-coated microprojectiles. Proc Natl Acad Sci 88: 2726-2730.
109. Woodley D. T, Krueger GG, Jorgensen CM, Fairley JA, Atha T, Huang Y., Chan L, Keene DR, Chen M. 2003. Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone. J Invest Dermatol 121: 1021-1028.
110. Woodley D. T, Keene DR, Atha T, Huang Y, Lipman K, Li W, Chen M. 2004a. Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa. Nat Med 10: 693-695.
111. Woodley D. T, Keene DR, Atha T, Huang Y, Ram R, Kasahara N, Chen M. 2004b. Intradermal injection of lentiviral vectors corrects regenerated human dystrophic epidermolysis bullosa skin tissue in vivo. Mol Ther 10: 318-326.
112. Yoon C. S, Jung HS, Kwon MJ, Lee SH, Kim CW, Kim MK, Lee M., Park JH. 2009. Sonoporation of the minicircle-VEGF165 for wound healing of diabetic mice. Pharm Res 26: 794-801.
113. ZhengD, GiljohannDA, Chen DL, MassichMD, Wang X-Q, Iordanov H., Mirkin CA, Paller AS. 2012. Topical delivery of siRNA-based spherical nucleic acid nanoparticle conjugates for gene regulation. Proc Natl Acad Sci 109: 11975-11980.