![]() Despite of various challenges, significant progress has been made in the development of delivery systems for therapeutic genetic materials. However, the limiting step for clinical development and application of gene therapy has been the safe and efficient delivery of genetic materials into target cells. Effective gene therapy requires correction of the mutated genes, replacement of the malfunctional genes with normal genes, or regulation of abnormal gene expression. Gene therapy corrects malfunctional genes and has brought hope to treat the diseases that are untreatable with conventional approaches. The flexibility and platform nature of LNP enable efficient intracellular delivery of a variety of therapeutic nucleic acids and provide many novel treatment options for the diseases that are previously untreatable. The evolution of structure and function of lipids as well as their LNP formulation from the early-stage simple formulations to multi-components LNP and multifunctional ionizable lipids have been discussed. ![]() Here, we highlight the key considerations for developing an LNP system. Due to the success of LNP systems, understanding the structure, formulation, and function relationship of the lipid components in LNP systems is crucial for design more effective LNP. ![]() Lipid nanoparticle (LNP) delivery technology has been a revolutionary development, which has been granted for clinical applications, including mRNA vaccines against SARS-CoV-2 infections. Gene therapies using DNA plasmid, RNAi, miRNA, mRNA, and gene editing hold great promise. Hereditary genetic diseases, cancer, and infectious diseases are affecting global health and become major health issues, but the treatment development remains challenging.
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