Federal Project Grant Award Summary

The National Center for Advancing Translational Sciences (NCATS) awarded Princeton University $164,000 through CFDA 93.350 (National Center for Advancing Translational Sciences) beginning July 1, 2026, with completion targeted for June 30, 2027. This project grant funds research into understudied rare disease genes associated with heterotaxy syndrome, a congenital disorder characterized by mispositioned internal organs and cardiac malformations. The primary deliverables include functional characterization of candidate proteins involved in left-right patterning during embryonic development using antisense and CRISPR-based genetic approaches, generation of targeted mutations in genes affecting left-right development, and creation of transgenic zebrafish lines and antibodies to facilitate future functional studies and drug screening efforts.

The research leverages zebrafish as an in vivo model system to advance understanding of how understudied proteins contribute to rare disease phenotypes and to identify genes warranting evaluation as causative factors in human heterotaxy. The project outputs—including transgenic zebrafish models, antibody reagents, and functional data on candidate rare disease genes—will establish essential research tools and foundational knowledge to support therapeutic discovery efforts in subsequent research initiatives. This work addresses the significant health and economic burden posed by rare diseases affecting approximately 30 million people in the United States.