期刊
ACTA PHARMACEUTICA SINICA B
卷 12, 期 6, 页码 2624-2639出版社
INST MATERIA MEDICA, CHINESE ACAD MEDICAL SCIENCES
DOI: 10.1016/j.apsb.2022.04.0132211-3835
关键词
USA; Lipid nanoparticle; Combinatorial library; Drug delivery; Cancer therapy; Protein delivery; Gene therapy
资金
- National Institutes of Health (NIH) [R01 EB027170-04, UG3 TR002636-01]
Lipid nanoparticle (LNP) -based drug delivery systems hold great potential in clinical applications and can encapsulate and transport various bioactive substances. To ensure the safe and efficient delivery of cargo molecules, careful design of each LNP carrier system is necessary. Our research group has developed novel LNP delivery systems through combinatorial library synthesis and in vitro and in vivo screening, and evaluated and optimized their drug delivery effects.
Lipid nanoparticle (LNP)-based drug delivery systems have become the most clinically advanced non-viral delivery technology. LNPs can encapsulate and deliver a wide variety of bioactive agents, including the small molecule drugs, proteins and peptides, and nucleic acids. However, as the physicochemical properties of small- and macromolecular cargos can vary drastically, every LNP carrier system needs to be carefully tailored in order to deliver the cargo molecules in a safe and efficient manner. Our group applied the combinatorial library synthesis approach and in vitro and in vivo screening strategy for the development of LNP delivery systems for drug delivery. In this Review, we highlight our recent progress in the design, synthesis, characterization, evaluation, and optimization of combinatorial LNPs with novel structures and properties for the delivery of small- and macromolecular therapeutics both in vitro and in vivo. These delivery systems have enormous potentials for cancer therapy, antimicrobial applications, gene silencing, genome editing, and more. We also discuss the key challenges to the mechanistic study and clinical translation of new LNP-enabled therapeutics.
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