Various RNAs, such as siRNA, microRNA, sgRNA, and mRNA, play pivotal roles in biological functions and have gained increasing importance for therapeutic applications. However, the lack of safe and efficient delivery vehicles remains the major challenge to RNA-based therapeutics. Our lab has designed both small molecule and polymer-based molecular carriers for safe and efficient delivery of both short and long RNAs into cells. Using natural amino acids and peptides as building blocks, we have developed dendronized bolaamphiphiles and polypeptides that are biocompatible, biodegradable, and highly efficient for universal delivery of various short and long RNAs. Our systematic investigation into structure–property relationships revealed important correlations between molecular design, self-assembled nanostructure, and biological activity. Through collaborations with biomedical researchers, we further investigate RNA delivery for various therapeutic applications including diabetes, vaccination, immunotherapy, and CRISPR-Cas based gene editing. In this talk, I will discuss the design, synthesis, biological studies, and biomedical applications of our new molecular carriers.

Amino acid and peptide functionalized unique molecular carriers for universal delivery of various RNAs

2019 Fall National Meeting

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