Mutations in any one of hundreds of genes cause neurodevelopmental disorders, like autism spectrum disorders and intellectual disability. How the diminished function of many of these genes drives abnormal brain development and function remains unknown. We seek to identify what processes are similar and different across disorders to understand the underlying mechanisms driving abnormal brain development and function and engineer therapies to reverse these abnormalities. Our lab is joint between the Departments of Cell Biology and Biomedical Engineering and we utilize a broad, interdisciplinary set of tools to tackle these challenging problems!
Research theme #1 - "Goldilocks genes" important for brain development.
For some unique genes, either an increase or decrease in their expression or function cause separate neurodevelopmental disorders. We are developing new human stem cell models to globally explore and understand why some genes are toxic if they are "too hot" or "too cold". In using these models, we hope to identify the biological processes that help these critical genes working "just right".
Research theme #2 - Growth factor signals required for normal brain function.
Growth factors are powerful proteins that control development and cell fate. We have identified a growth factor pathway, centered around the growth factor GDF11, that is critical for normal brain function. How and why exactly GDF11 is necessary for brain development and function remains unknown. Understanding how this growth factor guides brain development may allow us to develop therapies to promote healthy brain function.
Research theme #3 - Computational systems approaches to dissect the molecular pathogenesis of neurodevelopmental disorders.
The exact molecular consequences from a mutation in a neurodevelopmental disorder-causing gene tends to be subtle. We are leveraging high throughput molecular techniques to globally survey what processes go awry in disease and developing new computational tools, models, and frameworks to separate the important biological signals from the noise.
