1st Research Funding Award

“Targeting miRNAs as a novel therapy for STXBP1 epileptic encephalopathy”

The STXBP1 Foundation has just made our first-ever research funding award for $75,000 to the University of Pennsylvania, Perelman School of Medicine. Principal Investigator Ben Prosser will continue progress on a promising genetic therapy approach that targets microRNAs, or miRNA. miRNA-based therapies are quickly emerging as a new type of therapy which allows targeting of specific genes. Prosser’s lab is pursuing a miRNA-based strategy to increase STXBP1 protein in neurons. STXBP1 is critical for neurotransmitter release, and patients with STXBP1 disorders do not produce enough STXBP1. This new funding will enable Prosser’s lab to identify and validate potential therapy candidates. Find out more here in the poster from our summer conference.