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Gene therapy of epidermolysis bullosa.
- 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.
- Subjects :
- Animals
Autoantigens genetics
Autoantigens physiology
Carrier Proteins
Cell Adhesion Molecules deficiency
Cell Adhesion Molecules genetics
Cell Adhesion Molecules physiology
Cytoskeletal Proteins
DNA, Complementary genetics
DNA-Binding Proteins
Disease Models, Animal
Dystonin
Epidermolysis Bullosa classification
Epidermolysis Bullosa genetics
Genetic Heterogeneity
Genetic Vectors administration & dosage
Genetic Vectors genetics
Genetic Vectors therapeutic use
Humans
Integrin beta4 genetics
Integrin beta4 physiology
Keratin-14
Keratinocytes metabolism
Keratinocytes transplantation
Keratins deficiency
Keratins genetics
Keratins physiology
Matrix Metalloproteinases physiology
Mice
Mice, Nude
Nerve Tissue Proteins
Non-Fibrillar Collagens deficiency
Non-Fibrillar Collagens genetics
Non-Fibrillar Collagens physiology
Protease Inhibitors therapeutic use
RNA Splicing
RNA, Catalytic therapeutic use
Telomerase genetics
Telomerase physiology
Kalinin
Collagen Type XVII
Epidermolysis Bullosa therapy
Genetic Therapy
Subjects
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