Article
Toward epidermal stem cell-mediated ex vivo gene therapy of junctional epidermolysis bullosa.
Human gene therapy - 1 Nov 2000
Dellambra E, Pellegrini G, Guerra L, Ferrari G, Zambruno G, Mavilio F, De Luca M
Abstract excerpt
Junctional epidermolysis bullosa (JEB) is a group of severe, inherited skin diseases caused by mutations in the genes encoding laminin 5 or other components of the hemidesmosome. Since human epidermis is a self-renewing tissue, gene therapy of JEB requires the stable integration of the transgene into the genome of the epidermal stem cell. Human epidermal stem cells can indeed be cultivated and stably transduced...
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