The Journal of pharmacology and experimental therapeutics

Using mRNA and gene therapy as new treatments for diabetes

Updated

Abstract

mRNA therapies have demonstrated potential in restoring insulin production and enhancing angiogenesis in diabetes.

  • Emerging mRNA and gene therapies focus on targeting the underlying mechanisms of diabetes rather than just managing symptoms.
  • mRNA therapies delivered via lipid nanoparticles have shown effectiveness in preclinical models for both type 1 and type 2 diabetes.
  • Vascular endothelial growth factor-A, fibroblast growth factor 21, and glucagon-like peptide-1 linked to the IgG4 Fc region are key mRNAs explored for therapeutic use.
  • Gene therapy aims for long-term correction of insulin signaling and pancreatic cell reprogramming, utilizing both viral and nonviral delivery methods.
  • CRISPR/Cas9 genome-editing tools are being investigated for their ability to correct metabolic defects and protect insulin-producing β cells.
  • Challenges such as delivery efficiency, immunogenicity, and off-target effects remain critical hurdles for the successful application of these therapies.

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Full Text

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