CORDIS Project
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This project aims to develop a gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), a severe genetic skin disease. It focuses on creating genetically corrected skin grafts using patient-derived cells to treat affected individuals, with plans for a clinical trial to assess safety and efficacy.
RDEB is one of the most severe rare genetic skin diseases of children and adults characterized by skin blistering resulting from lack of expression of type VII collagen.
There is no treatment for this life-threatening disease.
In March 2009, we obtained the orphan drug designation for the Medicinal product: “Skin equivalent graft genetically corrected with a COL7A1-encoding SIN retroviral vector”, which was developed during the THERAPEUSKIN project (FP6) to treat RDEB patients by ex vivo gene…
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France, Paris
Type: Research institute
Activity type: Research Organisations
SME: No
United Kingdom, London
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Germany, IDAR OBERSTEIN
Type: SME
Activity type: Private for-profit entities (excluding Higher or Secondary Education Establishments)
SME: Yes
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