STXBP1 Foundation Awards Innovation Grant to Soo-Kyung Lee for Gene Therapy Development

Dr Soo Lee, a well-known neurodevelopmental biologist, holds two named professorships at UB (aka, SUNY-Buffalo). Her studies have been centered around how motor neurons develop.

The STXBP1 Foundation announces it has awarded a $100,000 Innovation Grant to Soo-Kyung Lee, PhD of University of Buffalo. Dr. Lee is Empire Innovation Professor and Om P. Bahl Endowed Professor of Biological Sciences, and the Director of the FOXG1 Research Center at University of Buffalo. Her research focuses on understanding the genetic and mechanistic basis for neurodevelopmental disorders. Her lab recently developed a gene therapy for FOXG1 syndrome.

“We are honored to receive an STXBP1 Innovation Grant, which will allow us to develop a gene therapy vector for STXBP1 disorders. This support provides a vital step toward translating our research into meaningful therapies for patients and families affected by this devastating condition” says Soo-Kyung Lee, PhD.

The title of Dr. Lee’s grant is “STXBP1 Gene Therapy”. As part of the grant work, Dr. Lee’s lab will aim to develop an AAV9-based gene therapy for STXBP1. STXBP1 expression level and distribution will be measured in mouse brain regions, and phenotypic rescue with the gene therapy will be evaluated in a STXBP1 mouse model. 

The funding for this project was provided by a generous donor who made the gift on behalf of STXBP1 patients Mackenzie and Megan Mitchell.

Learn more about Dr Lee’s work here.