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Gene therapy of epidermolysis bullosa.

Authors :
Bauer JW
Laimer M
Source :
Expert opinion on biological therapy [Expert Opin Biol Ther] 2004 Sep; Vol. 4 (9), pp. 1435-43.
Publication Year :
2004

Abstract

Easy access to the organ and identification of underlying mutations in epidermolysis bullosa (EB) facilitated the first cutaneous gene therapy experiments in vitro in the mid-1990s. The leading technology was transduction of the respective cDNA carried by a retroviral vector. Using this approach, the genotypic and phenotypic hallmark features of the recessive forms of junctional EB, which are caused by loss of function of the structural proteins laminin-5 or bullous pemphigoid antigen 2/type XVII collagen of the dermo-epidermal basement membrane zone, have been corrected in vitro and in vivo using xenograft mouse models. Recently, this approach has also been shown to be feasible for the large COL7A1 gene (mutated in dystrophic EB), applying PhiC31 integrase or lentiviral vectors. Neither of these approaches has made it into a successful Phase I study on EB patients. Therefore, alternative approaches to gene correction, including modulation of splicing, are being investigated for gene therapy in EB.

Details

Language :
English
ISSN :
1744-7682
Volume :
4
Issue :
9
Database :
MEDLINE
Journal :
Expert opinion on biological therapy
Publication Type :
Academic Journal
Accession number :
15335311
Full Text :
https://doi.org/10.1517/14712598.4.9.1435