Towards personalized CRISPR/Cas gene editing for dominant genetic disorders of the ocular and skin surface

This seminar will highlight our advancements in therapeutic strategies for Epidermolysis Bullosa (EB) and EEC syndrome, focusing on an established platform that incorporates: (i) ex vivo combined cell and gene therapy for recessive EB (Dystrophic and Junctional EB) and (ii) in vivo gene editing for dominant Simplex EB and EEC syndrome.

Despite the challenges associated with the latter approach, both strategies represent significant progress in addressing these debilitating conditions. The goal of these therapeutic approaches is to achieve sustained epithelial regeneration through precise targeting and correction of epidermal stem cells. These findings represent a major step forward in the development of curative treatments for dominant epithelial genetic disorders, paving the way for future clinical applications.