Identifying therapeutic vulnerabilities in fibrolamellar carcinoma

Timeframe: 2020 – 2023 Goal: Investigate the impact on FLC growth and survival of inhibiting two specific oncogenes identified in previous works Principal Investigator: Praveen Sethupathy, PhD Study overview: Based on previous epigenomic, metabolomic, and microRNA profiling, as well as initial drug studies, the study team has developed two new exciting hypotheses about therapeutic vulnerabilities …

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Retinoic acid-induced loss of DNAJB1-PRKACA fusion protein expression

Timeframe: 2019 – 2023 Goal: Investigate the potential of retinoic acid therapy Principal Investigators: Andrew Yen, PhD and Praveen Sethupathy, PhD Study overview: FLC is driven by the DNAJ-PKAc fusion protein. A potential therapeutic strategy would be to induce loss of this key driver protein. One approach to substantially alter gene expression in cancer cells …

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Multicenter consortium to define the single-cell activity landscape of fibrolamellar carcinoma.

Timeframe: 2022 – 2024 Goals: Advance immunotherapy for FLC by defining promising immunological targets that can be translated into effective cell-based immunotherapies Principal Investigators: Praveen Sethupathy, PhD (Cornell University); Mark Yarchoan, MD (Johns Hopkins University); Paul G. Thomas, PhD (St. Jude Children’s Research Hospital) Study overview: Like tumors of other cancer types, the microenvironment of …

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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, PhD Study overview: This study aimed to leverage genome-scale approaches to discover the molecular factors most critical to FLC tumor formation, metastasis, and drug resistance. MicroRNAs and long non-coding RNAs are …

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