Identification and characterization of receptor-specific peptides for siRNA delivery

ACS Nano. 2012 Oct 23;6(10):8620-31. doi: 10.1021/nn301975s. Epub 2012 Aug 27.

Abstract

Tumor-targeted delivery of siRNA remains a major barrier in fully realizing the therapeutic potential of RNA interference. While cell-penetrating peptides (CPP) are promising siRNA carrier candidates, they are universal internalizers that lack cell-type specificity. Herein, we design and screen a library of tandem tumor-targeting and cell-penetrating peptides that condense siRNA into stable nanocomplexes for cell type-specific siRNA delivery. Through physiochemical and biological characterization, we identify a subset of the nanocomplex library of that are taken up by cells via endocytosis, trigger endosomal escape and unpacking of the carrier, and ultimately deliver siRNA to the cytosol in a receptor-specific fashion. To better understand the structure-activity relationships that govern receptor-specific siRNA delivery, we employ computational regression analysis and identify a set of key convergent structural properties, namely the valence of the targeting ligand and the charge of the peptide, that help transform ubiquitously internalizing cell-penetrating peptides into cell type-specific siRNA delivery systems.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • HeLa Cells
  • Humans
  • Nanocapsules / chemistry
  • Nanocapsules / therapeutic use*
  • Neoplasms, Experimental / physiopathology*
  • Neoplasms, Experimental / therapy
  • Peptide Library
  • Peptides / pharmacokinetics*
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / genetics*
  • Receptors, Cell Surface / metabolism*
  • Transfection / methods*

Substances

  • Nanocapsules
  • Peptide Library
  • Peptides
  • RNA, Small Interfering
  • Receptors, Cell Surface