The National Center for Advancing Translational Sciences (NCATS), part of the National Institutes of Health, has awarded a $160,500 Project Grant to Childrens Hospital Medical Center in Cincinnati, Ohio. The grant, designated as CFDA 93.350, will fund the establishment of a novel zebrafish model to study the pathogenesis of Progressive Familial Intrahepatic Cholestasis Type I (PFIC1), a rare genetic liver disease caused by mutations in the ATP8B1 gene.
The key objectives are to: 1) Characterize the cholestatic phenotypes caused by ATP8B1 deficiency in zebrafish; 2) Determine the feasibility of using zebrafish to validate the biological function of ATP8B1 patient variants; and 3) Develop assays to evaluate the effects of pharmaceutical interventions on cholestasis in the zebrafish model. This research is expected to advance the understanding of ATP8B1 function in bile homeostasis and the mechanisms underlying PFIC1, providing critical preliminary data to support future NIH R01 grant applications for further disease modeling and drug discovery efforts.
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