A partnership with the Interdisciplinary Neuroscience Program.
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"Development of a Humanized Mouse Model for Precision Therapeutic Testing in a Rare Neurological Disorder"Presented by: Date: March 2, 2026, 4:00-5:00 p.m. Location: Roy Blunt NextGen Precision Health Building To be added to the monthly calendar event, email Zezong Gu. |
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Description
Rare neurological disorders present significant challenges for therapeutic development due to limited patient populations and a lack of disease-relevant model systems. This seminar will highlight the development of a novel humanized mouse model of Baker-Gordon syndrome, a severe and understudied neurological disorder caused by pathogenic variants in SYT1. The model was engineered to carry a patient-specific mutation, enabling precise investigation of disease mechanisms in vivo and providing a platform for translational therapeutic testing.
The talk will focus on the design, generation, and validation of this humanized mouse model and discuss how it enables downstream testing of precision therapeutic strategies. Initial proof-of-concept data from human cell-based studies supporting allele-specific targeting of the mutant SYT1transcript using short interfering RNAs (siRNAs) will be briefly presented. Ongoing work will extend these studies into the mouse model to support future therapeutic evaluation. Together, this work demonstrates how patient-informed animal models can accelerate precision medicine efforts for rare neurological diseases.
Speaker
Dr. Daniel Davis is Director of the MU Animal Modeling Core and an Associate Research Professor in the School of Veterinary Medicine at the University of Missouri. His research focuses on rare neurological disorders and therapeutic development. Rare diseases are often understudied, leaving affected individuals and families with limited treatment options. Dr. Davis’s laboratory develops patient-informed, humanized animal models to better understand disease biology and to enable the development and testing of targeted genetic therapies. His work integrates close collaboration with clinicians and patient advocacy groups to ensure translational relevance. Dr. Davis received his bachelor’s degree in Cell and Molecular Biology from Missouri State University and his PhD in Pathobiology from the University of Missouri. He is a member of the Center for Translational Neurogenetics, where he leads efforts in humanized model development and genetic therapeutic design.
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