Characterization of an HIV-targeted transcriptional gene-silencing RNA in primary cells

Hum Gene Ther. 2012 May;23(5):473-83. doi: 10.1089/hum.2011.165. Epub 2012 Jan 26.

Abstract

Small antisense RNAs targeted to the HIV-1 promoter have been shown to remodel the surrounding chromatin to a state unfavorable for transcriptional activation, yet transcriptional gene silencing (TGS) of HIV-1 has, to date, not been shown in primary human cells. We demonstrate here that TGS can reduce viral transcription in primary human CD4(+) T cells; however, increasing viral burden results in the loss of this antiviral effect. This observation suggests a critical level at which viral RNA can dilute out effective targeting by TGS-based RNAs. Furthermore, studies into off-target effects have identified a potential interaction between the small nucleolar RNA pathway and the TGS-based antisense RNA, resulting in activation of p53. Although not overtly toxic to primary cells, this represents a novel interaction between antisense RNAs and a cellular pathway that should be considered when pursuing small antisense RNA-based therapeutics.

Publication types

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

MeSH terms

  • CD4-Positive T-Lymphocytes / metabolism
  • CD4-Positive T-Lymphocytes / virology*
  • Chromatin / metabolism
  • Gene Silencing*
  • Genetic Vectors / metabolism
  • HEK293 Cells
  • HIV-1 / genetics*
  • HIV-1 / metabolism
  • Humans
  • Jurkat Cells
  • Promoter Regions, Genetic / drug effects*
  • RNA, Antisense / genetics
  • RNA, Antisense / metabolism
  • RNA, Antisense / pharmacology*
  • Transcription, Genetic / genetics
  • Transcriptional Activation
  • Tumor Suppressor Protein p53 / genetics
  • Tumor Suppressor Protein p53 / metabolism

Substances

  • Chromatin
  • RNA, Antisense
  • Tumor Suppressor Protein p53