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Messenger RNA-based therapeutics for the treatment of apoptosis-associated diseases

Akitsugu Matsui1, Satoshi Uchida1, Takehiko Ishii2

  • 1Laboratory of Clinical Biotechnology, Center for Disease Biology and Integrative Medicine, Graduate School of Medicine, The University of Tokyo, Tokyo, 113-0033, Japan.

Scientific Reports
|October 29, 2015
PubMed

Insights

Messenger RNA (mRNA) therapeutics offer a safer alternative to gene therapy for diseases linked to excessive apoptosis. This study shows mRNA effectively delivers anti-apoptotic factors to liver cells, outperforming plasmid DNA and reducing disease severity.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Hepatology

Background:

  • Gene therapy shows promise for diseases with excessive apoptosis by introducing anti-apoptotic factors.
  • Plasmid DNA (pDNA) delivery risks genomic integration, potentially causing cancer.
  • Messenger RNA (mRNA) offers a non-integrating alternative for therapeutic factor delivery.

Purpose of the Study:

  • To evaluate mRNA-based therapeutics for delivering anti-apoptotic factors.
  • To compare the efficacy of mRNA versus pDNA delivery in a mouse model of fulminant hepatitis.
  • To assess the potential of mRNA therapeutics combined with polyplex nanomicelles for treating apoptosis-related diseases.

Main Methods:

  • Utilized a mouse model of fulminant hepatitis.
  • Employed hydrodynamic intravenous injection with synthesized polymer-based polyplex nanomicelles for liver mRNA delivery.
  • Used Green Fluorescent Protein (GFP) as a reporter to quantify protein expression.
  • Tracked Cy5-labelled mRNA and pDNA to analyze intracellular mechanisms and localization.

Main Results:

  • mRNA delivery achieved nearly 100% liver cell protein expression, significantly higher than pDNA.
  • mRNA expression relied on a direct intracellular mechanism, bypassing nuclear entry.
  • Bcl-2 mRNA demonstrated greater effectiveness in reducing liver apoptosis compared to Bcl-2 pDNA in the disease model.

Conclusions:

  • mRNA-based therapeutics are a potent strategy for delivering anti-apoptotic factors.
  • Polyplex nanomicelles serve as an effective delivery system for mRNA to liver cells.
  • This mRNA-based approach holds significant promise for treating intractable diseases characterized by excessive apoptosis.

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