siRNA therapy in dominant skin and eye disorders

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Incurable genetic diseases represent a major cost to the NHS, requiring specialist care costing billions per annum. RNA interference is a new Nobel prize winning technology that is suitable for treatment of a large number of genetic disorders but a major hurdle to applying this clinically is the the problem of delivery of these therapy molecules, that are slightly larger than conventional drugs, into the target tissues and organs. This programme grant focuses on a group of painful, debilitating genetic skin diseases and a related corneal eye disease, where the target tissues are very small and very accessible. The programme brings together leading researchers who uncovered the genes causing these disorders, with experts in developing new methods to deliver molecules into skin or cornea. State-of-the-art model systems will be developed to validate delivery of these new formulations into the skin and the cornea, setting the scene for taking this new therapy method into the clinic.