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mRNA-Based Approaches to Treating Liver Diseases

Maximiliano L Cacicedo1, María José Limeres1, Stephan Gehring1

  • 1Children's Hospital, University Medical Center Mainz of the Johannes-Gutenberg University, Langenbeckstr. 1, 55131 Mainz, Germany.

Cells
|October 27, 2022
PubMed

Insights

Liver disease causes millions of deaths annually. mRNA-based gene therapy offers a promising alternative to viral vectors for treating liver conditions like cancer and hepatitis.

Area of Science:

  • Hepatology
  • Gene Therapy
  • Molecular Medicine

Background:

  • Liver diseases cause 2 million deaths yearly, with limited treatment options.
  • Hepatocytes are key targets for liver-directed gene therapy.
  • Current treatments like liver transplantation are insufficient for many patients.

Purpose of the Study:

  • To review the potential of mRNA-based therapeutics for liver diseases.
  • To discuss the advantages of mRNA therapy over viral vectors.
  • To explore applications in inherited metabolic diseases, cirrhosis, cancer, and viral infections.

Main Methods:

  • Review of clinical and preclinical studies on mRNA therapeutics.
  • Focus on lipid nanoparticle-encapsulated mRNA encoding therapeutic proteins.
  • Evaluation of mRNA therapy for liver cancer, hepatitis B, and cytomegalovirus.

Main Results:

  • mRNA therapy shows promise for treating various liver conditions.
  • Lipid nanoparticle delivery of mRNA is an effective strategy.
  • Studies cover inherited metabolic disorders, cirrhosis, fibrosis, and viral hepatitis.

Conclusions:

  • mRNA-based gene therapy is a viable and advantageous approach for liver disease treatment.
  • Further research and clinical trials are warranted to optimize mRNA therapies.
  • This modality offers a new avenue for managing liver pathologies.
Keywords:
cirrhosishepatocellular carcinomainherited metabolic diseaseslipid nanoparticlestherapeutic mRNA constructs

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