CORDIS Project
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This project focuses on developing advanced gene targeting and correction technologies for treating epidermolysis bullosa, a severe skin condition. By creating novel viral and non-viral vectors, the aim is to safely integrate therapeutic genes into specific locations in the genome, enhancing gene therapy applications f…
Epidermolysis bullosa (EB) is a family of severe skin adhesion defects characterized by disfiguring blistering, infections, visual impairment, and a high risk of cancer.
We showed through pre-clinical and clinical investigation that transplantation of genetically corrected epithelia leads to long-term functional correction of EB.
The current vectors, however, integrate in an uncontrolled fashion in the human genome, a cause of genotoxicity and potentially severe adverse events.
The objective of…
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