Therapeutic messenger RNA (mRNA) (mRNA)

Therapeutic messenger RNA (mRNA) is a class of nucleic acid-based drugs designed to deliver genetic instructions to cells, enabling them to produce specific proteins for therapeutic or prophylactic purposes (Sahin et al., 2014). Unlike traditional drugs that target proteins, mRNA acts as a template for protein synthesis, effectively turning the patient's own cells into bioreactors (Pardi et al., 2018). This modality is highly versatile, used in vaccines to encode viral antigens, in oncology to stimulate immune responses against tumor-specific antigens, and in rare diseases to replace deficient enzymes (Damase et al., 2021). The success of mRNA technology is heavily dependent on delivery systems, typically lipid nanoparticles (LNPs), which protect the mRNA from degradation and facilitate cellular uptake (Hou et al., 2021). While highly effective, challenges include managing the body's innate immune response to exogenous RNA and ensuring stable, tissue-specific delivery (Kowalski et al., 2019). Consequently, Therapeutic mRNA payload refers to the active genetic component of an RNA-based medicine rather than a biological target like a receptor or enzyme.

Other names

Messenger RNASynthetic mRNAModified mRNAmodRNAmRNA payload

Translation of the mRNA sequence into a functional protein or antigen by the host cell's ribosomes, followed by post-translational modification and localization to the appropriate cellular compartment (Sahin et al., 2014).