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Usher syndrome is the most common cause of deaf-blindness and affects an estimated 400,000 people worldwide. It is usually diagnosed in childhood, often in very young children. This project is a new and pioneering approach to the challenge of finding effective treatments.
Categories
Beneficiaries
Situation
Children born with Usher Syndrome face significant challenges as both their vision and hearing are impaired. The shock of diagnosis, followed by progressive sight loss, can be very difficult for the individual affected and their family. Finding a way to prevent loss of vision is vital, and we believe that work towards the development of viable treatments should be a priority. Sadly the level of funding available in the UK for research does not reflect this urgency.
Solution
This project, led by talented researcher Dr Mariya Moosajee at the UCL Institute of Ophthalmology and Moorfields Eye Hospital, aims to try a new approach to the exploration of possible treatments. This innovative investigation will test whether a non-viral alternative to traditional gene therapy is viable, not just for the treatment of Usher Syndrome, but for a wide range of inherited sight loss conditions. The aim is to create progress towards effective treatments for those affected.