Grant: Project Grant

Certain genetic mutations cause rare skin conditions like Epidermolytic Ichthyosis (EI) and Harlequin Ichthyosis (HI), as well as common diseases like atopic dermatitis (AD). These diseases often lack effective treatments, leaving patients with significant challenges. Gene editing, a groundbreaking technology, has the potential to correct these mutations and provide lasting cures. However, delivering gene-editing tools directly to the skin and targeting the right cells remains a significant challenge. This research aims to develop innovative, topical gene-editing therapies to address these unmet needs. The project focuses on three goals: (1) designing strategies to correct genetic mutations responsible for EI, HI, and AD, (2) optimizing messenger RNA (mRNA) molecules to work specifically and effectively in skin cells, and (3) creating lipid nanoparticles (LNPs) that can deliver gene-editing tools directly to skin stem cells. Using advanced technologies, the team will refine mRNA designs to improve how they interact with human skin and enhance the precision of gene editing. By targeting skin stem cells, the approach aims to offer long-lasting and potentially curative treatments. If successful, this work will pave the way for new therapies for severe genetic skin diseases and conditions like atopic dermatitis, transforming care for patients with few or no current options.