Gene Therapy in Patients with Epidermolysis Bullosa

• Authors: Bartłomiej Noszczyk , Józef Jethon
• Languages of publication: EN

Abstracts

EN

• Publisher: Index Copernicus International
• Journal: Polish Journal of Surgery
• Year: 2008
• Volume: 80
• Issue: 4
• Pages: 222-226

Dates

published

1 - 4 - 2008

online

10 - 5 - 2008

Contributors

author

Bartłomiej Noszczyk

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

author

Józef Jethon

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

References

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• 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]
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• 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.
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• 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]

Identifiers

DOI

10.2478/v10035-008-0027-0