In Situ Gene Editing To Rescue Severe, Genetic Skin Diseases
Grantee: Sarah Hedtrich, Professor, University of British Columbia, Canada
Amount: DKK 4,191,726
Grant category: Research Grants
Year: 2025
Geography: Canada
Some rare skin conditions, like Epidermolytic Ichthyosis (EI) and Harlequin Ichthyosis (HI), are caused by changes in certain genes. These diseases can be severe and often have no effective treatments, making life very hard for patients. Gene editing—a powerful new technology—could fix these faulty genes and provide lasting cures, but delivering these tools into the skin and targeting the right cells is a major challenge. This project aims to develop new, skin-applied gene-editing treatments. The goals are to: (1) create ways to correct the gene changes that cause EI and HI, (2) design messenger RNA (mRNA) molecules that work well in skin cells, and (3) make tiny delivery packages, called lipid nanoparticles (LNPs), that carry the tools to skin stem cells. By improving mRNA design and targeting, the approach could lead to long-lasting, possibly permanent treatments for severe genetic skin diseases.