Poster Presentation Hunter Cell Biology Meeting 2022

Abnormal Behavior and Cortical Connectivity Deficits in Mice Lacking Usp9x (#71)

Michael Piper 1
  1. The University of QLD, Brisbane, QUEENSLAND, Australia

Genetic association studies have identified many factors associated with neurodevelopmental disorders such as autism

spectrum disorder (ASD). However, the way these genes shape neuroanatomical structure and connectivity is poorly

understood. Recent research has focused on proteins that act as points of convergence for multiple factors, as these may

provide greater insight into understanding the biology of neurodevelopmental disorders. USP9X, a deubiquitylating enzyme

that regulates the stability of many ASD-related proteins, is one such point of convergence. Loss of function variants in

human USP9X lead to brain malformations, which manifest as a neurodevelopmental syndrome that frequently includes

ASD, but the underlying structural and connectomic abnormalities giving rise to patient symptoms is unknown. Here, we

analyzed forebrain-specific Usp9x knockout mice (Usp9x−/y) to address this knowledge gap. Usp9x−/y mice displayed

abnormal communication and social interaction behaviors. Moreover, the absence of Usp9x culminated in reductions to the

size of multiple brain regions. Diffusion tensor magnetic resonance imaging revealed deficits in all three major forebrain

commissures, as well as long-range hypoconnectivity between cortical and subcortical regions. These data identify USP9X

as a key regulator of brain formation and function, and provide insights into the neurodevelopmental syndrome arising as a

consequence of USP9X mutations in patients.