• recruiting, genotyping and characterising at molecular level cohorts of patients with RDEB and DDEB

• isolating epithelial stem cells from the skin of DEB patients and DEB dogs, a spontaneous large animal model of the disease

• developing viral vectors (oncoretroviral, lentiviral and hybrid adeno/AAV vectors) to integrate and stably express the collagen VII cDNA in DEB epidermal cells ex vivo

• transducing highly clonogenic DEB keratinocytes with the most appropriate vector(s)

• constructing skin equivalents made with genetically corrected DEB cells to assess the full morphological and functional reversion of the DEB phenotype

• constructing transplantable epithelia using genetically corrected DEB cells and preclinical assessment of their performance in vivo in appropriate immune competent animal models

• developing a technology for production and downstream processing of clinical-grade viral vectors prepared under Good Manufacturing and Laboratory Practices (GMP/GLP) standards

• establishing Standard Operating Procedures (SOP), protocols and guidelines to genetically modify epidermal stem cells used to produce skin implants for clinical use

• disseminating results.

Participating groups

INSERM –U634, Faculty of Medicine of Nice. (France) Project co-ordinator

Regional Epithelial Stem Cell Research Centre, Fondazione Banca Degli Occhi del Veneto (FBOV) (Italy)

Department of Biomedical sciences, University of Modena and Reggio Emilia (Italy)

Department of Dermatology, University of Freiburg (Germany)

Centro de Investigaciones Energeticas Medioambiantes y Technologicas (Spain)

MolMed S.p.a.(Italy)

DEBRA Europe (UK based)