Design advances and uses of mRNA treatments for cancer, heart disease, and rare genetic disorders beyond infections
Updated
Abstract
Messenger RNA (mRNA) therapeutics have evolved rapidly, expanding into oncology, cardiovascular diseases, and rare disorders.
- mRNA serves as a template for protein production, offering advantages over traditional protein and DNA drugs.
- The stability of mRNA and its sensitivity to immune detection limit its ability to enter cells without a delivery system.
- Lipid nanoparticles (LNPs) are commonly used for mRNA delivery, but they have limitations such as reactogenicity and liver targeting.
- Next-generation designs like SORT lipids and stimuli-responsive hybrids may enhance targeting beyond the liver.
- In oncology, LNP-mRNA is utilized for neoantigen vaccines, while rare diseases benefit from transient enzyme replacement.
- Safety profiles indicate that modifications can tune immune responses, but risks remain with chronic administration.
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Funding
Competing interests
The authors declare no conflicts of interest.
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