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Number of results
2008 | 80 | 4 | 222-226

Article title

Gene Therapy in Patients with Epidermolysis Bullosa


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1 - 4 - 2008
10 - 5 - 2008


  • Department of Plastic Surgery, Medical Center of Postgraduate Education, W. Orłowski Memorial Hospital, Warsaw
  • Department of Plastic Surgery, Medical Center of Postgraduate Education, W. Orłowski Memorial Hospital, Warsaw


  • Fivenson DP, Scherschun L, Cohen LV: Apligraf in the treatment of severe mitten deformity associated with recessive dystrophic epidermolysis bullosa. Plast Reconstr Surg 2003; 112(2): 584-88.[PubMed][Crossref]
  • Chen M, O'Toole EA, Muellenhoff M et al.: Development and characterization of a recombinant truncated type VII collagen "minigene". Implication for gene therapy of dystrophic epidermolysis bullosa. J Biol Chem 2000; 275(32): 24429-35.
  • Chen M, Kasahara N, Keene DR et al.: Restoration of type VII collagen expression and function in dystrophic epidermolysis bullosa. Nat Genet 2002; 32(4): 670-75.[PubMed][Crossref]
  • Chen M, Woodley DT: Fibroblasts as target cells for DEB gene therapy. J Invest Dermatol 2006; 126(4): 708-10.[PubMed][Crossref]
  • Chen M, Li W, Fan J, Kasahara N et al.: An efficient gene transduction system for studying gene function in primary human dermal fibroblasts and epidermal keratinocytes. Clin Exp Dermatol 2003; 28(2): 193-99.[PubMed][Crossref]
  • Woodley DT, Krueger GG, Jorgensen CM et al.: Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone. J Invest Dermatol 2003; 121(5): 1021-28.[Crossref][PubMed]
  • Woodley DT, Remington J, Huang Y et al.: Intravenously Injected Human Fibroblasts Home to Skin Wounds, Deliver Type VII Collagen, and Promote Wound Healing. Mol Ther 2007; 15(3): 628-35.[Crossref][PubMed][WoS]
  • Woodley DT, Keene DR, Atha T et al.: Injection of recombinant human type VII collagen restores collagen function in dystrophic epidermolysis bullosa. Nat Med 2004; 10(7): 693-95.[PubMed][Crossref]
  • Kotch FW, Raines RT: Self-assembly of synthetic collagen triple helices. Proc Natl Acad Sci USA 2006; 103(9): 3028-33.[Crossref]
  • Goto M, Sawamura D, Ito K et al.: Fibroblasts show more potential as target cells than keratinocytes in COL7A1 gene therapy of dystrophic epidermolysis bullosa. J Invest Dermatol 2006; 126(4): 766-72.[Crossref]
  • Gagnoux-Palacios L, Hervouet C, Spirito F et al.: Assessment of optimal transduction of primary human skin keratinocytes by viral vectors. J Gene Med 2005; 7(9): 1178-86.[Crossref]
  • Szala S: Terapia genowa. Wydawnictwo Naukowe PWN, Warszawa 2003.
  • Recchia A, Perani L, Sartori D et al.: Site-specific integration of functional transgenes into the human genome by adeno/AAV hybrid vectors. Mol Ther 2004; 10(4): 660-70.[Crossref][PubMed]
  • Ferrari S, Pellegrini G, Mavilio F et al.: Gene therapy approaches for epidermolysis bullosa. Clin Dermatol 2005; 23(4): 430-36.[Crossref][PubMed]
  • Mavilio F, Pellegrini G, Ferrari S et al.: Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat Med 2006; 12(12): 1397-402.[PubMed]
  • Ortiz-Urda S, Thyagarajan B, Keene DR et al.: Stable nonviral genetic correction of inherited human skin disease. Nat Med 2002; 8(10): 1166-70.[Crossref]
  • Groth AC, Olivares EC, Thyagarajan B et al.: A phage integrase directs efficient site-specific integration in human cells. Proc Natl Acad Sci USA 2000; 97(11): 5995-6000.[Crossref]
  • Ortiz-Urda S, Lin Q, Green CL et al.: Injection of genetically engineered fibroblasts corrects regenerated human epidermolysis bullosa skin tissue. J Clin Invest 2003; 111(2): 251-55.
  • 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(13): 1099-1104.[Crossref]
  • Ehrhardt A, Xu H, Huang Z et al.: A direct comparison of two nonviral gene therapy vectors for somatic integration: in vivo evaluation of the bacteriophage integrase phiC31 and the Sleeping Beauty transposase. Mol Ther 2005; 11(5): 695-706.[Crossref]
  • Ortiz-Urda S, Thyagarajan B, Keene DR et al.: PhiC31 integrase-mediated nonviral genetic correction of junctional epidermolysis bullosa. Hum Gene Ther 2003; 14(9): 923-28.
  • Yang Y, Walsh CE: Spliceosome-mediated RNA trans-splicing. Mol Ther 2005; 12(6): 1006-12.[Crossref][PubMed]
  • Dallinger G, Puttaraju M, Mitchell LG et al.: Development of spliceosome-mediated RNA transsplicing (SMaRT) for the correction of inherited skin diseases. Exp Dermatol 2003; 12(1): 37-46.[Crossref]

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