siRNA Vehicles for High Endosomal Escapability

Methods Mol Biol. 2021:2282:171-179. doi: 10.1007/978-1-0716-1298-9_11.

Abstract

Small interfering RNA (siRNA) is a novel therapeutic modality for the treatment of intractable diseases; however, the development of a useful siRNA delivery vector is imperative for clinical use. Since siRNA works in the cytoplasm, the ability of the carrier to escape destruction in the endosomes is a highly required characteristic for the induction of a high knockdown effect. Here, we describe the step-by-step procedure for the evaluation of high endosomal escapability. The vector that has pH-responsive characteristics at around pH = 6.2-6.5 is important for the high endosomal escape.

Keywords: Confocal image; Endosome; Lipid nanoparticle; Small interfering RNA; pH-responsive vehicle.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line, Tumor
  • Endosomes / metabolism*
  • Fibrosarcoma / genetics*
  • Fibrosarcoma / metabolism
  • Gene Expression Regulation, Neoplastic
  • Gene Transfer Techniques*
  • Humans
  • Hydrogen-Ion Concentration
  • Liposomes / chemistry*
  • RNA Interference*
  • RNA Stability*
  • RNA, Small Interfering / chemistry
  • RNA, Small Interfering / genetics*
  • RNA, Small Interfering / metabolism
  • Research Design
  • Workflow

Substances

  • Liposomes
  • RNA, Small Interfering