Epidermolysis Bullosa (EB) is a group of genetic disorders characterized by extremely fragile skin that blisters and tears easily from minor friction or trauma. These debilitating conditions arise from mutations in genes responsible for producing proteins crucial for skin integrity and structure. Gene therapy offers a potential avenue for treating EB by aiming to correct these underlying genetic defects within the affected skin tissues.
Restoring the function of these crucial proteins within the skin’s structural layers holds the promise of significantly improving skin resilience and reducing blister formation in individuals with EB. This therapeutic approach offers a potential shift from managing symptoms to addressing the root cause of the disease. While still under development, gene therapy represents a significant advancement in the search for effective EB treatments, moving beyond palliative care towards a potential cure. Research continues to explore the most effective vectors for gene delivery and the optimal methods for achieving sustained therapeutic benefit.
