Gene expression as a target for new drug discovery

Gene Expr. 1995;4(6):337-44.

Abstract

Over the last 7 years we have carried out a major research effort focused on gene transcription as a novel approach to drug discovery. The goal is to identify small molecular weight compounds that modulate the expression of a target gene in a specific manner, thereby either increasing or decreasing the concentration of the corresponding protein product. Transcriptional modulation not only provides a potential means to replace recombinant proteins as drugs, but also provides a novel approach to manipulate key gene targets in many therapeutic areas. This article describes some of the features and advantages of transcription-based pharmaceuticals and illustrates how this approach can be applied to drug discovery with a program we are pursuing to identify new treatments for sickle cell disease and beta-thalassemia.

Publication types

  • Review

MeSH terms

  • Anemia, Sickle Cell / drug therapy
  • Drug Design*
  • Drug Evaluation, Preclinical
  • Gene Expression / drug effects*
  • Gene Expression Regulation
  • Globins / genetics
  • Humans

Substances

  • Globins