Stabilization and Movable Ligand-Modification by Folate-Appended Polyrotaxanes for Systemic Delivery of siRNA Polyplex

ACS Macro Lett. 2022 Nov 15;11(11):1225-1229. doi: 10.1021/acsmacrolett.2c00462. Epub 2022 Oct 10.

Abstract

To achieve a systemic targeted delivery of siRNA using polymeric carriers, there is a dilemma between ligand modification and stabilization of the polyplex. Namely, ligand modification often leads to destabilization of the polyplex in the blood circulation. In fact, we previously developed cyclodextrin (CD)/polyamidoamine dendrimer conjugates (CDE) as siRNA carriers, and the interaction of CDE/siRNA was decreased by the conjugation with folate-polyethylene glycol, leading to the destabilization. To overcome this dilemma, in this study, folate-appended polyrotaxanes (Fol-PRX) were developed. Fol-PRX stabilized CDE/siRNA polyplex by intermolecularly connecting CDE molecules through a host-guest interaction between adamantane at the terminals of Fol-PRX and β-CD in the polyplex. Moreover, the intermolecular connection of the polyplex with Fol-PRX provided movable folate moieties on the surface. As a result, Fol-PRXs enhanced the in vivo antitumor activity of the polyplex after intravenous administration, suggesting their utility as the dual-functional materials for systemic delivery of siRNA polyplexes.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Folic Acid
  • Ligands
  • Polyethylene Glycols
  • RNA, Small Interfering
  • Rotaxanes*

Substances

  • RNA, Small Interfering
  • Rotaxanes
  • Folic Acid
  • Ligands
  • Polyethylene Glycols