Efficient delivery of sticky siRNA and potent gene silencing in a prostate cancer model using a generation 5 triethanolamine-core PAMAM dendrimer

Mol Pharm. 2012 Mar 5;9(3):470-81. doi: 10.1021/mp2006104. Epub 2012 Jan 20.

Abstract

Successful achievement of RNA interference in therapeutic applications requires safe and efficient vectors for siRNA delivery. In the present study, we demonstrate that a triethanolamine (TEA)-core PAMAM dendrimer of generation 5 (G(5)) is able to deliver sticky siRNAs bearing complementary A(n)/T(n) 3'-overhangs effectively to a prostate cancer model in vitro and in vivo and produce potent gene silencing of the heat shock protein 27, leading to a notable anticancer effect. The complementary A(n)/T(n) (n = 5 or 7) overhangs characteristic of these sticky siRNA molecules help the siRNA molecules self-assemble into "gene-like" longer double-stranded RNAs thus endowing a low generation dendrimer such as G(5) with greater delivery capacity. In addition, the A(n)/T(n) (n = 5 or 7) overhangs act as protruding molecular arms that allow the siRNA molecule to enwrap the dendrimer and promote a better interaction and stronger binding, ultimately contributing toward the improved delivery activity of G(5). Consequently, the low generation dendrimer G(5) in combination with sticky siRNA therapeutics may constitute a promising gene silencing-based approach for combating castration-resistant prostate tumors or other cancers and diseases, for which no effective treatment currently exists.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoptosis / genetics
  • Apoptosis / physiology
  • Blotting, Western
  • Cell Line, Tumor
  • Dendrimers / administration & dosage
  • Dendrimers / chemistry*
  • Ethanolamines / chemistry*
  • Flow Cytometry
  • Gene Silencing / physiology*
  • Humans
  • Immunohistochemistry
  • Male
  • Mice
  • Mice, Nude
  • Microscopy, Confocal
  • Prostatic Neoplasms / genetics*
  • Prostatic Neoplasms / therapy*
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / genetics
  • Real-Time Polymerase Chain Reaction
  • Xenograft Model Antitumor Assays

Substances

  • Dendrimers
  • Ethanolamines
  • PAMAM Starburst
  • RNA, Small Interfering
  • triethanolamine