Lipid-encapsulated siRNA for hepatocyte-directed treatment of advanced liver disease

Cell Death Dis. 2020 May 11;11(5):343. doi: 10.1038/s41419-020-2571-4.

Abstract

Lipid-based RNA nanocarriers have been recently accepted as a novel therapeutic option in humans, thus increasing the therapeutic options for patients. Tailored nanomedicines will enable to treat chronic liver disease (CLD) and end-stage liver cancer, disorders with high mortality and few treatment options. Here, we investigated the curative potential of gene therapy of a key molecule in CLD, the c-Jun N-terminal kinase-2 (Jnk2). Delivery to hepatocytes was achieved using a lipid-based clinically employable siRNA formulation that includes a cationic aminolipid to knockdown Jnk2 (named siJnk2). After assessing the therapeutic potential of siJnk2 treatment, non-invasive imaging demonstrated reduced apoptotic cell death and improved hepatocarcinogenesis was evidenced by improved liver parenchyma as well as ameliorated markers of hepatic damage, reduced fibrogenesis in 1-year-old mice. Strikingly, chronic siJnk2 treatment reduced premalignant nodules, indicative of tumor initiation. Furthermore, siJnk2 treatment led to a significant activation of the immune cell compartment. In conclusion, Jnk2 knockdown in hepatocytes ameliorated hepatitis, fibrogenesis, and initiation of hepatocellular carcinoma (HCC), and hence might be a suitable therapeutic option, to define novel molecular targets for precision medicine in CLD.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis
  • Cell Line
  • Cell Transformation, Neoplastic / genetics
  • Cell Transformation, Neoplastic / metabolism
  • Cell Transformation, Neoplastic / pathology
  • Disease Models, Animal
  • Gene Transfer Techniques*
  • Hepatocytes / enzymology*
  • Hepatocytes / pathology
  • Intracellular Signaling Peptides and Proteins / genetics
  • Lipids / chemistry*
  • Liver / enzymology*
  • Liver / pathology
  • Liver Cirrhosis / enzymology
  • Liver Cirrhosis / genetics
  • Liver Cirrhosis / pathology
  • Liver Cirrhosis / therapy*
  • Liver Neoplasms / enzymology
  • Liver Neoplasms / genetics
  • Liver Neoplasms / pathology
  • Liver Neoplasms / prevention & control*
  • Male
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Mitogen-Activated Protein Kinase 9 / deficiency
  • Mitogen-Activated Protein Kinase 9 / genetics
  • Mitogen-Activated Protein Kinase 9 / metabolism*
  • Nanoparticles
  • RNA, Small Interfering / chemistry
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism*
  • RNAi Therapeutics*

Substances

  • Intracellular Signaling Peptides and Proteins
  • Lipids
  • NEMO protein, mouse
  • RNA, Small Interfering
  • Mitogen-Activated Protein Kinase 9