Gedion Mengistu Dejen
mRNA therapeutics are transitioning from a vaccine-centered technology to a versatile platform for precision medicine. Advances in delivery systems, scalable manufacturing, and artificial intelligence-assisted sequence design are expected to accelerate their integration into the treatment of chronic, genetic, and regenerative diseases.
BACKGROUND: Messenger RNA (mRNA) therapeutics have rapidly evolved from experimental nucleic acid constructs into a clinically validated therapeutic platform. Their successful application during the COVID-19 pandemic accelerated interest in broader therapeutic uses beyond infectious diseases.
AIMS: This review summarizes the expanding therapeutic applications of mRNA technologies, recent technological advances, translational progress, current challenges, and future prospects across diverse medical fields.
MATERIALS AND METHODS: A comprehensive review of the published literature was conducted, synthesizing evidence from preclinical investigations, clinical studies, and recent advances in mRNA engineering, delivery systems, and translational research.
RESULTS: Advances in mRNA design, nucleoside modification, codon optimization, and delivery platforms, particularly lipid nanoparticles and emerging organ-targeted carriers, have expanded applications to cancer immunotherapy, personalized vaccines, autoimmune and inflammatory diseases, protein replacement therapy, cardiovascular regeneration, neurological disorders, and rare genetic diseases. Emerging technologies, including self-amplifying and circular RNA systems, further enhance therapeutic potential.
DISCUSSION: Despite significant progress, challenges remain, including optimization of targeted delivery, long-term safety, repeat-dose tolerability, manufacturing complexity, cold-chain requirements, production costs, and equitable global access. Continued technological innovation and regulatory harmonization are critical for successful clinical translation.
CONCLUSION: mRNA therapeutics are transitioning from a vaccine-centered technology to a versatile platform for precision medicine. Advances in delivery systems, scalable manufacturing, and artificial intelligence-assisted sequence design are expected to accelerate their integration into the treatment of chronic, genetic, and regenerative diseases.