Article
A Microinjected <i>COL7A1</i> -PAC Vector Restores Synthesis of Intact Procollagen VII in a Dystrophic Epidermolysis Bullosa Keratinocyte Cell Line
1 Sept 2002
Abstract excerpt
Dystrophic epidermolysis bullosa (DEB) comprises a family of inherited blistering skin disorders for which no corrective therapy currently exists. In the most severe form, the Hallopeau-Siemens subtype (RDEB-HS), the epidermal adhesion protein collagen VII is absent from the skin as a consequence of null mutations in the COL7A1 gene. In order to develop an ex vivo gene therapy approach for DEB, we aimed to...
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