Webinars: Targeting BAP1 Mutations & Development of a Mouse Model
Presenter: Milind Javle, MD
Title of Presentation: Targeting BAP1 Mutations in Intrahepatic Cholangiocarcinoma
Summary of Presentation:
The purpose of this study is to identify molecular subgroups of cholangiocarcinoma by estimating the frequency of BAP1 mutations in patients with intrahepatic cholangiocarcinoma, examining BAP1 and other genes in cholangiocarcinoma to link with diagnosis and patient outcomes, and developing a mouse model of BAP1 that can be used for clinical studies of new drugs.
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Presenter: Rebecca Marcus, MD
Title of Presentation: Of mice and men – Development of a Mouse Model for intrahepatic Cholangiocarcinoma
Summary of Presentation:
Cholangiocarcinoma is a lethal bile duct malignancy, and the second most common primary hepatic malignancy. A subtype of this disease,intrahepatic cholangiocarcinoma (ICC), is increasing worldwide, and characterized by a particularly aggressive disease course including early metastasis. Currently, many aspects of this disease are incompletely defined and therapeutic options are few.
Genetically engineered mouse models (GEMMs) are a well-established model for studying the multi-step progression of many types of human cancer. Furthermore, the mouse currently represents the only organism in which induced cholangiocarcinoma faithfully recapitulates critical aspects of the disease. Given this, attempts are underway to generate GEMMs for ICC that carry many of the same DNA abnormalities that occur in human disease. These models will provide new insight into how ICC originates, grows, and spreads from the intrahepatic bile ducts. They will also help in the development of early detection methods for and targeted therapies against ICC.