Identification of a potent ionizable lipid for efficient macrophage transfection and systemic anti-interleukin-1β siRNA delivery against acute liver failure

J Mater Chem B. 2021 Jul 7;9(25):5136-5149. doi: 10.1039/d1tb00736j. Epub 2021 Jun 16.

Abstract

RNA interference (RNAi) therapy has great potential for treating inflammatory diseases. However, the development of potent carrier materials for delivering siRNA to macrophages is challenging. Herein, we design a set of ionizable lipid nanoparticles (LNPs) to screen and identify a potent carrier of siRNA for silencing an essential pro-inflammatory cytokine, interleukin-1β (IL-1β) in macrophages. The top performance LNP (114-LNP), containing ionizable lipid with spermine as an amine-head group, facilitated efficient siRNA internalization via multiple endocytosis pathways and achieved effective endosome escape in macrophages. The optimized LNP/siIL-1β achieved strong silencing of IL-1β in both activated Raw 264.7 cells and primary macrophages. Furthermore, systematic administration of 114-LNP/siIL-1β complexes could effectively inhibit IL-1β expression in an acute liver failure model and significantly attenuated hepatic inflammation and liver damage. These results suggest that the optimized ionizable lipid nanoparticle represents a promising platform for anti-inflammation therapies.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cells, Cultured
  • Drug Delivery Systems
  • Interleukin-1beta / antagonists & inhibitors*
  • Interleukin-1beta / metabolism
  • Lipids / chemistry*
  • Liver Failure, Acute / drug therapy*
  • Liver Failure, Acute / metabolism
  • Macrophages / drug effects*
  • Macrophages / metabolism
  • Mice
  • Molecular Structure
  • Nanoparticles / chemistry*
  • RAW 264.7 Cells
  • RNA, Small Interfering / pharmacology*

Substances

  • IL1B protein, mouse
  • Interleukin-1beta
  • Lipids
  • RNA, Small Interfering