RNAi-mediated CCR5 silencing by LFA-1-targeted nanoparticles prevents HIV infection in BLT mice

Mol Ther. 2010 Feb;18(2):370-6. doi: 10.1038/mt.2009.271. Epub 2009 Dec 8.

Abstract

RNA interference (RNAi)-mediated knockdown of gene expression offers a novel treatment strategy for human immunodeficiency virus (HIV) infection. However, the major hurdle for clinical use is a practical strategy for small interfering RNA (siRNA) delivery to the multiple immune cell types important in viral pathogenesis. We have developed a novel immunoliposome method targeting the lymphocyte function-associated antigen-1 (LFA-1) integrin expressed on all leukocytes and evaluated it for systemic delivery of siRNA in a humanized mouse model. We show that in vivo administration of the LFA-1 integrin-targeted and stabilized nanoparticles (LFA-1 I-tsNPs) results in selective uptake of siRNA by T cells and macrophages, the prime targets of HIV. Further, in vivo administration of anti-CCR5 siRNA/LFA-1 I-tsNPs resulted in leukocyte-specific gene silencing that was sustained for 10 days. Finally, humanized mice challenged with HIV after anti-CCR5 siRNA treatment showed enhanced resistance to infection as assessed by the reduction in plasma viral load and disease-associated CD4 T-cell loss. This study demonstrates the potential in vivo applicability of LFA-1-directed siRNA delivery as anti-HIV prophylaxis.

Publication types

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

MeSH terms

  • Animals
  • Gene Silencing / physiology*
  • HIV Infections / genetics
  • HIV Infections / immunology
  • HIV Infections / prevention & control*
  • Leukocytes / metabolism
  • Liposomes / therapeutic use
  • Lymphocyte Function-Associated Antigen-1 / genetics
  • Lymphocyte Function-Associated Antigen-1 / physiology*
  • Mice
  • Nanoparticles / therapeutic use*
  • RNA Interference
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / physiology*
  • Receptors, CCR5 / genetics*
  • Reverse Transcriptase Polymerase Chain Reaction

Substances

  • Liposomes
  • Lymphocyte Function-Associated Antigen-1
  • RNA, Small Interfering
  • Receptors, CCR5