Discovery of selective, small-molecule inhibitors of RNA complexes--I. The Tat protein/TAR RNA complexes required for HIV-1 transcription

Bioorg Med Chem. 1997 Jun;5(6):1173-84. doi: 10.1016/s0968-0896(97)00064-3.

Abstract

We have developed a therapeutic program focusing on the inhibition of a human immunodeficiency virus-1 specific protein-RNA interaction. This program begins with a search for small organic molecules that would interfere with the binding of Tat protein to TAR RNA. The methodologies chosen to study the HIV-1 Tat-TAR interaction and inhibition include gel mobility shift assays, scintillation proximity assays, filtration assays, and mass spectrometry. These methods helped establish in vitro high-throughput screening assays which rapidly identified Tat-TAR inhibitors from our corporate compound library. Tat-activated reporter gene assays were then used to investigate the cellular activities of the Tat-TAR inhibitors. The cellular activity, selectivity, and toxicity data for select Tat-TAR inhibitors were determined. Evaluation of both the cellular data and the Tat-TAR inhibition results led to further testing in anti-HIV-1 infection assays.

MeSH terms

  • Amino Acid Sequence
  • Aminoglycosides
  • Anti-Bacterial Agents / pharmacology
  • Anti-HIV Agents / pharmacology*
  • Antineoplastic Agents, Phytogenic / pharmacology
  • Base Sequence
  • Benzodiazepinones / pharmacology
  • Camptothecin / pharmacology
  • Gene Products, tat / drug effects*
  • Gene Products, tat / metabolism*
  • HIV-1 / drug effects*
  • HIV-1 / metabolism
  • HIV-1 / physiology*
  • HeLa Cells / drug effects
  • Humans
  • Molecular Sequence Data
  • Pyrroles / pharmacology
  • RNA, Viral / antagonists & inhibitors*
  • RNA, Viral / metabolism*
  • Transcription, Genetic / drug effects*
  • Transcriptional Activation / drug effects
  • Transcriptional Activation / physiology
  • Virus Replication / drug effects
  • Virus Replication / physiology
  • tat Gene Products, Human Immunodeficiency Virus

Substances

  • Aminoglycosides
  • Anti-Bacterial Agents
  • Anti-HIV Agents
  • Antineoplastic Agents, Phytogenic
  • Benzodiazepinones
  • Gene Products, tat
  • Pyrroles
  • RNA, Viral
  • tat Gene Products, Human Immunodeficiency Virus
  • Ro 5-3335
  • Camptothecin