Description:

Benjamin Prosser Ph.D.,
Associate Professor of Physiology, University of Pennsylvania Perelman School of Medicine
Associate Director, Pennsylvania Muscle Institute (PMI), Perelman School of Medicine
Director, Center for Epilepsy and NeuroDevelopmental Disorders (ENDD)

The overarching mission of the Prosser Lab is to develop first-in-class therapeutics for rare, genetic neurodevelopmental disorders (NDDs). A central focus of the lab is precision strategies to correct gene haploinsufficiency through targeted upregulation of disease-causing genes. To this end, we have pioneered antisense oligonucleotide (ASO)–based approaches that modulate RNA processing through multiple mechanisms to increase functional gene expression. In this lecture, Dr. Prosser will highlight the conceptual framework and experimental validation of ASO-mediated gene upregulation, with a particular focus on synaptic disorders caused by STXBP1 and SYNGAP1 haploinsufficiency. He will also discuss complementary efforts within the Center for Epilepsy and Neurodevelopmental Disorders (ENDD) at Penn and CHOP to accelerate therapeutic translation for these conditions, including disease model development and coordinated initiatives to enable clinical trial readiness.