siRNA therapy in dominant skin and eye disorders

A single injection of short RNA molecules could stop the production of faulty proteins that cause painful, incurable genetic skin and eye diseases. These disorders—such as severe blistering skin conditions and related corneal eye disease—currently require lifelong specialist care, costing the NHS billions each year. The underlying genes are known, but the therapy that could silence them, called RNA interference, has been stalled by a delivery problem: the therapeutic molecules are too large to slip into cells the way conventional drugs do. This programme brings together the geneticists who identified the disease genes with drug-delivery experts who specialise in getting molecules into skin and cornea. Because these target tissues are small and accessible, they offer a practical route for testing new delivery formulations. If the team succeeds in developing reliable delivery methods, the same approach could be adapted for other genetic disorders where the affected tissue is reachable. The project will build state-of-the-art model systems to validate the formulations, setting the stage for clinical trials. This is applied fundamental science—it tackles a specific delivery bottleneck rather than exploring a broad biological question—but the payoff could be a new class of treatments for diseases that currently have none.