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Diseases 2015, 3(4), 325-340;

New Tools for Molecular Therapy of Hepatocellular Carcinoma

Department of Cellular Biotechnologies and Hematology, Pasteur Institute-Cenci Bolognetti Foundation, Sapienza University of Rome, Viale Regina Elena, 324, 00161 Rome, Italy
National Institute for Infectious Diseases Lazzaro Spallanzani, IRCCS, Via Portuense, 292, 00149 Rome, Italy
Authors to whom correspondence should be addressed.
Academic Editor: Stephen L. Chan
Received: 15 September 2015 / Revised: 15 October 2015 / Accepted: 22 October 2015 / Published: 30 October 2015
(This article belongs to the Special Issue Targeted Therapy of Hepatocellular Carcinoma: Present and Future)
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Hepatocellular carcinoma (HCC) is the most common type of liver cancer, arising from neoplastic transformation of hepatocytes or liver precursor/stem cells. HCC is often associated with pre-existing chronic liver pathologies of different origin (mainly subsequent to HBV and HCV infections), such as fibrosis or cirrhosis. Current therapies are essentially still ineffective, due both to the tumor heterogeneity and the frequent late diagnosis, making necessary the creation of new therapeutic strategies to inhibit tumor onset and progression and improve the survival of patients. A promising strategy for treatment of HCC is the targeted molecular therapy based on the restoration of tumor suppressor proteins lost during neoplastic transformation. In particular, the delivery of master genes of epithelial/hepatocyte differentiation, able to trigger an extensive reprogramming of gene expression, could allow the induction of an efficient antitumor response through the simultaneous adjustment of multiple genetic/epigenetic alterations contributing to tumor development. Here, we report recent literature data supporting the use of members of the liver enriched transcription factor (LETF) family, in particular HNF4α, as tools for gene therapy of HCC. View Full-Text
Keywords: HCC; HNF4α; LETFs; miRNAs; TGFβ; EMT; gene therapy HCC; HNF4α; LETFs; miRNAs; TGFβ; EMT; gene therapy

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Marchetti, A.; Bisceglia, F.; Cozzolino, A.M.; Tripodi, M. New Tools for Molecular Therapy of Hepatocellular Carcinoma. Diseases 2015, 3, 325-340.

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