Community Research and Development Information Service - CORDIS


Final Report - THERAPEUSKIN (Ex vivo gene therapy for recessive dystrophic epidermlysis bullosa: pre-clinical and clinical studies)

Project ID: 511974


This is the final report for the THERAPEUSKIN project. The report reveals how the partners have successfully developed a safe (Self inactivating - SIN) retroviral vector for genetic transfer of COL7A1 cDNA into keratinocytes and fibroblasts. The clinical protocol has been validated. The quality of the clinical-grade viral batch will be essential for the feasibility and the safety of this trial. This will allow completion and submission of the mandatory documents to the competent regulatory agency (AFSSAPS) in order to start the clinical trial.

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