Evaluation of rASO in a rat model of AS.

Primary Investigator: K. Nash

The use of ASOs to interrupt the paternal UBE3a allele silencing through antisense inhibition is a potential therapeutic approach for treating AS in humans. Here 3 University teams (USF, UC Davis and BCM) collaboratively propose to determine optimal sequence and concentration range for rat ASOs for the rat homologue. This study will allow the determination of a dose of ASOs to be used in a rat model of AS for a dose response and correlate that with in vivo expression and ATS knockdown changing the rat phenotypical behaviors and documenting if AS symptomatic rescue is achieved in an animal model. Expression will be determined from both CNS tissue and CSF fluid evaluation.