MicroRNA research support

Timeframe: 2011 Goal: Profile microRNA in metastatic Fibrolamellar Carcinoma Principal Investigator: Yuzhou Wang, PhD, University of British Columbia MicroRNAs are small RNA species that regulate the expression of other genes and are important in many biological processes, including tumor development, growth, and metastasis. MicroRNAs have become increasingly useful as diagnostic markers, prognostic indicators, and therapeutic …

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Iodine transporter research

Timeframe: 2014 – 2015 Goal: Investigate the potential of radioactive iodine as a therapeutic in FLC Principle Investigator: Nancy Carrasco, MD, Professor, Cellular and Molecular Physiology, Yale University (Currently at Vanderbilt University) FLC cells express high levels of the iodine transporter NIS, raising the possibility that radioactive iodine may be a potential therapeutic for FLC. …

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Discovery of the chimera genetic mutation

Timeframe: 2014 – 2015 Goal: Identify genetic mutations in FLC Principal Investigator: Sandy Simon, Ph.D., Professor, Rockefeller University The goal of this project was to identify genetic mutations present in FLC. The team used whole genome sequencing together with RNA sequencing to identify mutations in FLC tumors compared to their normal liver counterparts. Identification of …

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Developing therapeutics for fibrolamellar hepatocellular carcinoma

Timeframe: 2016 – 2017 Goal: Develop new therapeutics for FLC Principal Investigator: Sandy Simon, Ph.D., ProfessorInvestigator Collaboration: Barbara A. Lyons, Ph.D., Professor, New Mexico State University The goal of this work was to develop therapeutics for FLC. The team identified three independent strategies based on their laboratory’s work showing that a single common DNA alteration …

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Flipping the switch on PKA: synthetic lethal approaches to block PKA-driven tumor growth in fibrolamellar liver cancer

Timeframe: 2016 – 2019 Goal: Understand growth mechanisms and identify potential therapeutic targets Principal Investigators: John Gordan. M.D., Ph.D, Clinical Instructor, University of California San Francisco; Nabeel Bardeesy, Ph.D, Associate Professor, Harvard University The discovery of a genetic change in the protein kinase A (PKA) gene in nearly all cases of fibrolamellar liver cancer (FLC) …

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Characterizing enzyme inhibition of the DnaJPKAc chimeric protein derived from fibrolamellar hepatocellular carcinomas

Timeframe: 2017 – 2019 Goal: Characterize inhibition of the DnaJ-PKAc chimeric protein in fibrolamellar carcinoma Principal Investigator: Hibba tul Rehman, M.D., University of Vermont The DNAJB1-PRKACA fusion gene produces the DnaJ-PKAc fusion kinase protein, which is present in nearly all FLC tumors and promotes liver tumor formation in mice. Kinases, including fusion kinases, have been …

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Kinase fusion function investigation

Timeframe: 2016 – 2018 Goal: Investigate DNAJB1-PRKACA fusion kinase function in new fibrolamellar carcinoma models Principal Investigator: Dr. Yi Guo, Ph.D, Associate Consultant – Asst Professor, Dept of Biochemistry and Molecular Biology The DNAJB1-PRKACA fusion kinase is found in nearly 100% of FLC cases. While this novel fusion has been shown to promote tumors in …

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Hedgehog and YAP signaling in fibrolamellar carcinoma: tumor-stroma crosstalk and the cancer stem cell niche

Timeframe: 2017 – 2019 Goal: Evaluate the role of Hedgehog and YAP signaling in fibrolamellar carcinoma Principal Investigators: Cynthia Guy, MD, Associate Professor of Pathology; Anna Mae Diehl, MD, Florence McAlister Professor of Medicine, Duke University School of Medicine Fibrolamellar carcinoma has a unique histological appearance consisting of large tumor cells surrounded by thick fibrous …

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Micro RNAs and long non-coding RNAs role in fibrolamellar and evaluation of RNA-based therapeutics

Timeframe: 2017 – 2020 Goal: Investigate the role of microRNAs and long non-coding RNAs in fibrolamellar carcinoma and evaluate RNA-based therapeutics Principal Investigator: Praveen Sethupathy Associate Professor Department of Biomedical Sciences, Cornell University, College of Veterinary Medicine This study aimed to leverage genome-scale approaches to discover the molecular factors most critical to FLC tumor formation, …

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