[Research progress on lipid nanoparticle messenger RNA delivery system].

Area of Science:

• Biotechnology
• Nanomedicine
• Drug Delivery

Background:

• Messenger RNA (mRNA) therapeutics offer a promising approach for disease treatment and prevention by enabling intracellular production of target proteins.
• The efficacy of mRNA therapeutics is heavily dependent on effective delivery systems, with lipid nanoparticles (LNPs) being the predominant choice in clinical settings.

Purpose of the Study:

• To review the fundamental roles of the four key lipid components within LNPs for efficient mRNA delivery.
• To explore advanced strategies for targeted mRNA delivery to specific organs and tissues, including the liver, lung, spleen, pancreas, bone marrow, and placenta.
• To discuss current applications and inherent challenges of LNP-based mRNA therapeutics to guide future clinical translation and innovation.

Main Methods:

• Review of existing literature on LNP composition and function in mRNA delivery.
• Analysis of strategies employed for targeted organ delivery, such as antibody modification, lipid structure optimization, and specialized administration routes.
• Discussion of LNP-based mRNA therapeutic applications and associated challenges in disease treatment.

Main Results:

• LNPs, comprising ionizable lipids, phospholipids, cholesterol, and PEG-lipids, effectively protect mRNA and facilitate cellular uptake.
• Targeted delivery to organs like the liver, lung, and spleen is achievable through modifications in LNP formulation and administration.
• Various strategies are being developed to enhance the specificity and efficiency of LNP-mediated mRNA delivery.

Conclusions:

• LNPs are essential for the clinical success of mRNA therapeutics, addressing critical delivery barriers.
• Targeted delivery approaches are expanding the therapeutic potential of mRNA beyond the liver.
• Continued innovation in LNP design and targeting is vital for advancing mRNA-based disease treatment.