Prasanna Venkatesh Ramachandran, MD, PHD

Biliary Atresia Fund for the Cure Postdoctoral Research Fellowship Award
$25,000 over one year

Boston Children's Hospital

The role of primary cilia in hepatobiliary development and disease

Mentor: Wolfram Goessling, MD, PhD

Biliary atresia (BA) is a serious liver disease that affects infants, leading to the obstruction of bile ducts and causing significant liver damage early in life. BA is the most common reason for liver transplants in children, yet we do not fully understand what causes this condition. Our research focuses on tiny cellular structures called primary cilia, which are present on the surface of many cells, including those that form the bile ducts in the liver. These cilia act like antennae, helping cells sense their environment and control important growth signals. Recent evidence suggests that defects in primary cilia may be a key factor in the development of BA.

We will use zebrafish, a small fish that shares many similarities in liver development with humans, to study how these cilia contribute to the formation of healthy bile ducts. We will also look at how disrupting the function of a specific ciliary gene, PKD1L1, affects liver development and may lead to BA-like symptoms. Our goal is to better understand how cilia work in liver development and how their dysfunction might lead to diseases like BA. By learning more about the role of these cilia, we hope to discover new ways to identify and treat BA early, potentially reducing the need for liver transplants in affected children.