Heguy A, Stewart A A, Haley J D, Smith D E, Foulkes J G
Oncogene Science, Inc., Uniondale, NY 11553, USA.
Gene Expr. 1995;4(6):337-44.
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.
在过去7年里，我们开展了一项重大研究工作，重点是将基因转录作为药物发现的一种新方法。目标是识别能够以特定方式调节靶基因表达的小分子化合物，从而增加或降低相应蛋白质产物的浓度。转录调节不仅提供了一种替代重组蛋白作为药物的潜在手段，还为在许多治疗领域操纵关键基因靶点提供了一种新方法。本文描述了基于转录的药物的一些特点和优势，并通过我们正在进行的一项旨在识别镰状细胞病和β地中海贫血新疗法的项目，说明这种方法如何应用于药物发现。
