Community Research and Development Information Service - CORDIS


Final Report - SKINTHERAPY (Gene therapy for Epidermolysis Bullosa: a model system for treatment of inherited skin diseases)

Project ID: 512073


This is the final report for the SKINTHERAPY project whose goal was to develop a gene therapy approach to Dystrophic epidermolysis bullosa (DEB), which is caused by inherited genetic defects of collagen type VII (encoded by the COL7A1 gene), and design appropriate phase I/II clinical trials to exploit the knowledge generated by the preclinical studies proposed by the SKINTHERAPY consortium. The report concludes by mentioning that the overall expected result was a model system for DEB treatment using ex vivo gene therapy. This model can be extended to other genetic skin diseases. The model system addressed the current weaknesses in related research and aimed particularly at the development of significantly improved, efficient and safe delivery systems, which are urgently needed.

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