Delivery of mRNA Therapeutics via Novel Ionizable Lipid Nanoparicles
발표자
()
초록
내용
Lipid nanoparticles (LNP) formulations based on ionizable lipids have shown great promise for the in vivo delivery of RNA therapeutics. Their delivery efficacy has been well verified, however, challenges associated with cellular and immune toxicity still remain. To overcome these limitations, we aim to develop new class of ionizable lipids with low cellular toxicity. By incorporating biodegradable bonds within the lipid structures, they can be easily removed from the cellular environment to maintain normal homeostasis. With newly synthesized ionizable lipids, we have performed comparative analysis of these materials in vitro and in vivo. These results demonstrate that the biodegradable LNPs have a comparative gene-silencing activity as compared to the non-degradable LNPs while lowering the toxicity issues.