Mapping Molecular and Behavioral Impacts of SETBP1 Deficiency in Zebrafish

MDBRSETBP1
Written By Million Dollar Bike Ride

Awardee: Alexandra Little

Institution: McMaster University

Grant Amount: $68,544

Funding Period: February 1, 2025 - January 31, 2026

Summary:

This research aims to investigate how the loss of the SETBP1 gene affects zebrafish behavior and biology, with the goal of identifying potential treatments for SETBP1 haploinsufficiency disorder (SETBP1-HD), a genetic condition that causes developmental delays and intellectual disabilities in humans. Zebrafish are a valuable model for studying human diseases and screening potential therapies. We have created two zebrafish models that replicate aspects of SETBP1-HD by knocking out the SETBP1 gene, providing a unique opportunity to study compensatory mechanisms that may mitigate the effects of the disorder. Our study will explore the behavioral and physical changes in these zebrafish, examine how SETBP1 deficiency impacts the brain and muscle tissue, and use computational analysis to screen over 500 FDA-approved drugs to identify potential therapeutic candidates. Promising drugs will then be tested to evaluate their ability to reverse the effects of SETBP1 deficiency, potentially leading to viable treatments for SETBP1-HD.

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