CRISPR-engineering of human liver organoids to study fibrolamellar carcinoma

Timeframe: 2023 – 2025 Goals: Model development and understanding the requirements for FLC tumor formation Principal Investigators: Benedetta Artegiani, PhD; and Delilah Hendriks, PhD Study overview: In comparison with more common cancers, research models of FLC have been extremely limited. Drs. Artegiani and Hendriks have addressed this problem by creating “synthetic” models of FLC, generating …

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Liquid Biopsy for Active Surveillance of Fibrolamellar Hepatocellular Carcinoma

Timeframe: 2021 – 2023 Goal: Develop a blood-based screening method to detect relapse in FLC patients Principal Investigator: Gary S. Stein, PhD Study overview: This study is focused on developing a blood-based screening method to detect relapse in FLC patients that have been diagnosed and treated by surgery. This screening protocol is intended to be …

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Creating a fibrolamellar cancer dependency map

Timeframe: 2020 – 2023 Goal: Create a comprehensive list of potential drug targets for FLC Principal Investigator: Jesse Boehm, PhD Study overview: This project is part of the Broad Institute’s Rare Cancer Dependency Map Initiative. The project has three main goals to identify potential FLC therapeutics: If fully successful, this effort will nominate high priority …

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Development of a human-derived liver progenitor cell line with DNAJB1-PRKACA fusion gene and characterization of mitochondrial dynamics in fibrolamellar carcinoma

Timeframe: 2022 – 2023 Goal: Development of a novel human-derived liver progenitor cell line model of fibrolamellar carcinoma Principal Investigator: Khashayar Vakili, MD Study overview: Currently, very few cell-line models of FLC exist, so the development of additional cellular models is critical for the identification and testing of new therapies. Therefore, one of the aims …

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Creation of Fibrolamellar Tissue Repository at The Rockefeller University

Timeframe: 2013 – 2014 Goal: Support costs related to establishment of new FLC biospecimen storage facility Principal Investigator: Sandy Simon, PhD Study background: Access to tumor tissue is critical to help cancer researchers understand what drives a disease and how the cancer responds to treatments. Because fibrolamellar is so rare, there has long been a …

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Fibrolamellar carcinoma model development and analysis

Timeframe: 2020 – 2022 Goal: Model development Principal Investigator: Nabeel Bardeesy, PhD Study overview: While multiple FLC models have recently been developed, additional model development remains a priority for the FLC community. This effort aimed to develop multiple different FLC models as resources for the FLC research fibrolamellar cancer research community, collaborating with the Fibrolamellar …

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CRISPR-engineering of human liver organoids to study fibrolamellar carcinoma

Timeframe: 2021 – 2022 Goal: Model development Principal Investigator: Benedetta Artegiani, PhD Study Overview: Research into FLC is complicated because of the limited experimental tools that can be used to study this rare cancer. While tests in laboratory animals such as mice are useful tools, they do not reflect the human disease due to inherent …

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