Principal Investigator: Alyssa Kriegermeier, MD

Children with liver disease frequently suffer from jaundice caused by the poor flow of bile from the liver to the intestines—a disease state called cholestasis. Currently no treatments are available that effectively prevent liver failure due to pediatric cholestatic liver diseases. Lifesaving liver transplantation is often the only answer. Cells within the liver deal with the stress from cholestasis via stress-induced pathways known as the unfolded protein response (UPR). While the UPR has been studied in adult liver diseases, little is known about its activation in children. Dr. Kriegermeier previously demonstrated, in an animal model of primary sclerosing cholangitis (PSC), one form of cholestatic liver disease affecting children and adults (about 80 percent of whom also have inflammatory bowel disease (IBD- Crohn’s disease, ulcerative colitis, etc.)), that removing parts of the UPR affects disease progression. This study will seek to better understand the differences in adults and children within this cell stress response during times of cholestasis. Identifying new therapeutic targets will provide a springboard for developing treatments for these children that will hopefully prevent them from needing liver transplantation.