Partnership Grant 2003 The Board of Directors of The National MPS Society approved a 2003 Partnership Grant with Julia's Hope Medical Research Foundation for Sanfilippo Syndrome. A total of 3 grant proposals were received, all of which were for MPS III research. The funding is for one year, with both groups granting $41,000. "Sleeping Beauty Transgene Vehicle ? Potential New Therapy for MPS III-A" Dr. John Hopwood, Lysosomal Diseases Research Unit, North Adelaide, Australia. This grant is a Partnership Grant with Julia's Hope Medical Research Foundation for Sanfilippo Syndrome. The funding is for one year with both groups granting $41,000. The aims of this research project are to utilize a unique and recently described non-viral transposon system (named Sleeping Beauty [SB]) to facilitate gene augmentation therapy in lysosomal storage diseases (LSD) that affect the brain, with the view to preventing or reversing clinical signs and symptoms. We will begin by testing our potential new therapy in the mouse model of MPS III-A. This research will determine the breadth of the effect of the treatment on the brain. As LSD affect widespread areas within the brain, our treatment needs to have access to most of the brain to be effective in reversing the clinical signs and symptoms. The research will also determine whether the clinical manifestations of these disorders can be prevented in newborn animals, delayed/reversed in young animals or reversed in older animals