Human DDX3 protein is a valuable target to develop broad spectrum antiviral agents

Proc Natl Acad Sci U S A. 2016 May 10;113(19):5388-93. doi: 10.1073/pnas.1522987113. Epub 2016 Apr 26.

Abstract

Targeting a host factor essential for the replication of different viruses but not for the cells offers a higher genetic barrier to the development of resistance, may simplify therapy regimens for coinfections, and facilitates management of emerging viral diseases. DEAD-box polypeptide 3 (DDX3) is a human host factor required for the replication of several DNA and RNA viruses, including some of the most challenging human pathogens currently circulating, such as HIV-1, Hepatitis C virus, Dengue virus, and West Nile virus. Herein, we showed for the first time, to our knowledge, that the inhibition of DDX3 by a small molecule could be successfully exploited for the development of a broad spectrum antiviral agent. In addition to the multiple antiviral activities, hit compound 16d retained full activity against drug-resistant HIV-1 strains in the absence of cellular toxicity. Pharmacokinetics and toxicity studies in rats confirmed a good safety profile and bioavailability of 16d. Thus, DDX3 is here validated as a valuable therapeutic target.

Keywords: DDX3; broad spectrum antivirals; coinfections; host factors; resistance.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antiviral Agents / administration & dosage*
  • DEAD-box RNA Helicases / antagonists & inhibitors*
  • DEAD-box RNA Helicases / metabolism*
  • Drug Design
  • Enzyme Inhibitors
  • Molecular Targeted Therapy / methods*
  • Virus Replication / drug effects*
  • Virus Replication / physiology*

Substances

  • Antiviral Agents
  • Enzyme Inhibitors
  • DDX3X protein, human
  • DEAD-box RNA Helicases