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Recent years have witnessed tremendous developments and breakthroughs in the field of RNA-based therapeutics and vaccines. The distinct mechanisms of exogenous RNAs and analogs, including messenger RNAs, small interfering RNAs, microRNAs, and antisense oligonucleotides, have brought them unprecedented potential to treat a variety of pathological conditions. However, the widespread application of RNA therapeutics and vaccines is hampered by their intrinsic features (e.g., instability, large size, and dense negative charge) and formidable host barriers. Development of safe and efficient vectors is key for successful delivery and translation of RNA therapeutics and vaccines. In this talk, I will discuss our efforts towards the development of lipid nanoparticle (LNP) platforms that enable the delivery of RNA therapeutics and vaccines to a range of target cells and tissues in the body. Furthermore, I will describe new therapeutic strategies utilizing these LNPs including (i) in vivo reprogramming of immune cells for cancer immunotherapy and vaccination, (ii) in utero gene editing for treating disease before birth, and (iii) mRNA prenatal therapeutics for treating pregnancy disorders such as pre-eclampsia.

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