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Targeting Estrogen Receptor Signaling for Oligodendroglial Dysfunction in Angelman Syndrome

$150,000 2024 FT2024-001

Targeting Estrogen Receptor Signaling for Oligodendroglial Dysfunction in Angelman Syndrome

Stem cell technology would prove invaluable in finally discovering effective treatments for developmental brain disorders, including Angelman syndrome (AS). In the brain, information of learning and memory is processed and stored in the form of electrical signals transmitted through millions of circuits made of neurons. The supporting glial cells, such as oligodendrocytes, are active partners in such information processing by providing structural support to neurons to speed up the flow of electric signals. In AS, this function of oligodendrocyte falls short in children and young adults, and may slow the brain development and increase seizures. To date, scientists still do not know the cause and hence are unable to come up with a treatment for this dysfunction. This project aims to understand and translate how the unique biology and genetics of AS make a difference in the oligodendrocyte function. Specifically, a few selected compounds identified from a stem cell-based drug screen will be tested for their efficacy by using a mouse model and the induced pluripotent stem cells (iPS cells) donated from AS patients and health individuals. In doing so, we believe this project will significantly contribute to the creation of urgently needed new treatments for AS, helping reduce the heavy disease burden.

Principle Investigator

Dr. Yu-Wen Alvin Huang

Dr. Y. Alvin Huang is the GLF Translational Assistant Professor of Molecular Biology, Cell Biology and Biochemistry, joining the faculty of Brown Biology and Medicine in July, 2019. He acquired his M.D. and Neurology residency from National Taiwan University, Taipei, Taiwan, sub-specializing in neurodegenerative disorders. His Ph.D. in Neuroscience was received from Johns Hopkins University School of Medicine, where he studied the gene regulation in the context of memory formation. For his postdoctoral fellowship at Stanford University School of Medicine (mentored by Dr. Thomas Südhof), he innovated stem cell technologies and uncovered cellular mechanisms important for brain functions in health and in disease. His current research focuses on molecular biology of Alzheimer's disease and aging, aided by new modeling tools developed in his laboratory. He is the recipient of several academic awards, including the NIH Pathway to Independence Award.

Dr. Yu-Wen Alvin Huang headshot