Xiao Zhao, MD, MS

Biliary Atresia Fund for the Cure Pilot Research Award
$50,000 over one year

Columbia University

CRISPR/Cas9-based Chemogenomic Profiling to Map Gene-Environment Interactions underlying Biliary Atresia

Biliary atresia (BA) remains an enigmatic neonatal cholangiopathy that is the leading indication for liver transplantation in the pediatric population. We have isolated a novel plant toxin that is responsible for outbreaks of BA in newborn Australian livestock, and identified important signaling pathways induced by this toxin that are relevant to human BA. Our proposed experiments will use comprehensive CRISPR-based chemogenomic profiling followed by subsequent validation in order to identify genetic determinants driving cellular response to this toxin, with the goal of systematically deciphering host factors responsible for the BA phenotype and identifying novel therapeutic opportunities.