Evaluation of the rASO in the rad model of AS

Primary Investigator: J. Silverman

The objective of this grant is to evaluate the behavioral rescue in a rat model of AS after treatment with a rat specific ASO designed by Dr. Dindot’s lab. FAST’s unique rat model provides the opportunity to investigate more complex behaviors that have been difficult to capture in mice, which include developmental milestones, neonatal calling as communication, juvenile behaviors, acoustic communication and advanced learning and memory. Furthermore, motor dysfunction, communication, and cognitive dysfunction are translatable to humans with AS, and we can leverage this sophisticated model for human translational therapies. In addition, this study will identify if ASO treatment rescued the MRI changes seen in the AS rat like axonal loss and microcephaly.