STXBP1 Foundation Awards Innovation Grant to Helen Willsey for Gene Therapy Development

The Willsey Lab at UCSF focuses on uncovering the molecular mechanisms that drive autism spectrum disorder and its frequently co-occurring conditions.

The STXBP1 Foundation announces it has awarded an Innovation Grant worth up to $100,000 to Helen Willsey, PhD of University of California, San Francisco. Dr. Willsey is Assistant Professor, Psychiatry and Behavioral Sciences, and an Investigator with the Chan Zuckerberg Biohub. Her research uses frog models to study neurological disorders.

“I am deeply honored to receive this Innovation Award from the STXBP1 Foundation. While STXBP1 is best known for its role in neurons, we are excited to explore its functions beyond the brain, particularly in ciliated cells. By investigating how STXBP1 contributes to respiratory health, we hope to uncover new insights into why breathing complications remain a major cause of early mortality in individuals with STXBP1-related disorders. With this support, we will also leverage patient-derived nasal brush cultures to better understand ciliary function and to lay the groundwork for biomarker development. This funding enables us to pursue a new dimension of STXBP1 biology with the potential to directly inform clinical care.” says Dr Willsey.

The title of Dr. Willsey’s grant is “STXBP1 in respiratory cilia form and function”. As part of the grant work, Dr. Willsey’s lab will study the potential role of STXBP1 in cilia. Her lab has already performed foundational work comparing cilia function in STXBP1 patients and family member controls by measuring nitric oxide in the nose.

James R Goss, PhD, Scientific Director, STXBP1 Foundation adds, “Dr. Willsey’s research on STXBP1’s role in respiratory cilia and related respiratory issues highlights the Foundation’s commitment to funding projects that advance our understanding of STXBP1, including its function in non-neuronal cells”.

Learn more about Dr Willsey’s work here