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Updated: Jan 14, 2026

An Oncogenic Hepatocyte-Induced Orthotopic Mouse Model of Hepatocellular Cancer Arising in the Setting of Hepatic Inflammation and Fibrosis
Published on: September 12, 2019
Liver-Directed Molecular Therapies: Current and Upcoming Strategies
Patrik Asp1, Beata Malachowska2, Chandan Guha3
1Department of Surgery, Albert Einstein College of Medicine, Ullman Building 305, 1300 Morris Park Avenue, Bronx, NY 10461, USA.
Abstract:
Hundreds of rare monogenic metabolic liver diseases together affect 1 in 800 live births. Liver transplantation remains the only cure for many of these disorders. Liver-targeted DNA and RNA-based molecular therapies are potential alternatives, for which critical advances at preclinical level have led to clinical trials.This review will provide a concise profile of viral and nonviral vectors for liver-targeted delivery of therapeutic DNA and RNA payloads, as well as novel approaches to gene editing for the treatment of monogenic liver diseases.
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