Work

mRNA Therapeutics: Bridging Pharmacological Mechanisms with Lipid Nanoparticle Drug Delivery Systems

Year: 2026
Type: article
Abstract: Messenger RNA (mRNA)-based therapeutics have emerged as a revolutionary platform in modern medicine due to their rapid development, high therapeutic adaptability, and ability to induce transient intracellular protein expression without genomic integration. The remarkable success of mRNA vaccines during the COVID-19 pandemic accelerated interest in RNA therapeutics for applications in infectious diseases, cancer immunotherapy, rare genetic disorders, regenerative medicine, and precision therapeutics. However, naked mRNA is highly unstable, susceptible to enzymatic degradation, and exhibits poor cellular uptake, creating major challenges for clinical translation. Lipid nanoparticles (LNPs) have become the leading non-viral delivery systems for mRNA therapeutics because they protect mRNA from degradation, improve intracellular transport, and facilitate cytoplasmic release. The pharmacological performance of mRNA-LNP formulations is significantly influenced by factors such as lipid composition, particle size, surface charge, encapsulation efficiency, and endosomal escape capability. Recent advances in ionizable and biodegradable lipids, targeted nanocarriers, and formulation engineering have improved the stability, safety, and tissue specificity of mRNA delivery systems. This review discusses the molecular basis of mRNA therapeutics and highlights the critical role of LNPs in overcoming biological and translational barriers associated with RNA delivery. It further examines intracellular trafficking mechanisms, immune interactions, formulation strategies, and clinical applications, while addressing manufacturing and regulatory challenges. Emerging approaches including organ-selective delivery, artificial intelligence-assisted formulation design, and personalized mRNA therapeutics are also explored as future directions in advanced pharmaceutical development.
Language: English
Open Access status: diamond
OpenAlex ID: W7217835196
FWCI:
Cites: 78
Cited by:
Related to: 10