Research Infrastructure
Research Infrastructure
The goal of this proposal is to create a stable infrastructure for the rapid testing of potential therapeutics in human stem cell-derived models of Angelman syndrome (AS) including two-dimensional neurons and three-dimensional cerebral organoids. The evaluation of the cellular entry, neuronal function, and molecular readouts to inform a potential therapeutic, in a high throughput and reliable manner, is highly desirable. This funding is meant to create a stable infrastructure to reliabily test drug candidates for academic researchers and industry. Overall, this can decrease the time and maximize the expertise for all working on AS. This effort also benefits the training and retention of skilled personnel dedicated to AS research, having an even longer-term impact on the Angelman field. Creating a highly synergistic system for FAST to have a rapid, reliable, and interactive resource for testing therapeutic candidates, developing shareable assays and tools, and creating a team of researchers focused on advacing AS drug development, will advance the field.
Principle Investigator
Albert Keung, PhD
Dr. Albert Keung is the Goodnight Distinguished Scholar, Associate Professor of Chemical and Biomolecular Engineering, and Director of The Biotechnology Program at North Carolina State University. His group applies synthetic biology approaches to advance knowledge and treatment of neuroepigenetic disorders. Their work includes engineering new models for Angelman syndrome research and therapeutic testing, and using human stem cell models to study neuroepigenetic mechanisms underlying neurodevelopmental disorders.
