Article
Gene-Corrected Fibroblast Therapy for Recessive Dystrophic Epidermolysis Bullosa using a Self-Inactivating COL7A1 Retroviral Vector.
The Journal of investigative dermatology - 1 Jul 2016
Jacków Joanna, Titeux Matthias, Portier Soizic, Charbonnier Soëli, Ganier Clarisse, Gaucher Sonia, Hovnanian Alain
Abstract excerpt
Patients with recessive dystrophic epidermolysis bullosa (RDEB) lack type VII collagen and therefore have severely impaired dermal-epidermal stability causing recurrent skin and mucosal blistering. There is currently no specific approved treatment for RDEB. We present preclinical data showing that intradermal injections of genetically corrected patient-derived RDEB fibroblasts using a Good Manufacturing Practices...
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