Development of BDNF Potentiating Therapeutics for Motor and Cognitive Dysfunction in AS

Brain-derived neurotrophic factor (BDNF) is a key protein essential for regulating the cellular processes that underlie learning and memory. Studies show that the deficits in learning observed in a mouse model of Angelman syndrome (AS) stem from reduced BDNF activity. This research project will test a novel class of therapeutics, referred to as Syn compounds, that facilitate learning and restore normal mobility in AS mice. In humans, these compounds are envisaged to facilitate learning, for example, a new visual symbol, and enable the child to retain this information over an extended time until it becomes permanent. Syn compounds will undergo further animal studies to determine safety and ability to treat symptoms (learning and walking) in the Angelman animal models. The results of this project with the AS model mouse will be used to inform treatment of Angelman syndrome in patients.