My work focuses on the development of clinically relevant genome editing strategies aimed at restoring the function of genes implicated in rare and severe skin disorders, particularly epidermolysis bullosa. My scientific expertise involves next-generation genome editing technologies, including CRISPR/Cas9, Base Editors, and Prime Editors, to correct recurrent COL7A1 mutations. This research addresses both fundamental questions, such as editing efficiency, specificity, and cellular consequences; and translational objectives, including the generation of corrected primary keratinocytes, fibroblasts, 3D skin equivalents and human: murine skin xenografts.
During a six-month international mobility program in 2024–2025 at the University of Colorado, I also developed expertise in the generation and characterization of iPSC-derived skin organoids. These advanced and physiologically relevant models are now being implemented in our laboratory as a versatile and scalable alternative to xenograft models, enabling the evaluation of genome editing efficacy and safety in combination with innovative delivery systems such as lipid nanoparticles (LNPs) and virus-like particles (VLPs), for future in vivo applications.