Department of Neurology, Johns Hopkins University, Baltimore, MD 21287, USA +1 410 614 6551 ; +1 410 505 6737 ;
Expert Opin Drug Discov. 2008 Jan;3(1):83-97. doi: 10.1517/17460441.3.1.83.
Dystonia is a neurological disorder characterized by involuntary twisting movements and unnatural postures. There are many different forms of dystonia, which affect over three million people worldwide. Effective treatments are available only for a minority of patients, so new treatments are sorely needed. Several animal species have been used to develop models for different forms of dystonia, each with differing strengths and weaknesses. This review outlines the strategies that have been used to exploit these models for drug discovery. Some have been used to dissect the pathogenesis of dystonia for the identification of molecular targets for intervention. Others have been used for the empirical identification of candidate drugs. Therefore, the animal models provide promising new tools for developing better treatments for dystonia.
肌张力障碍是一种神经系统疾病,其特征为不自主的扭曲运动和异常姿势。肌张力障碍有许多不同的形式,全球有超过 300 万人受其影响。只有少数患者能得到有效治疗,因此迫切需要新的治疗方法。几种动物物种已被用于开发不同形式的肌张力障碍模型,每种模型都有其优缺点。这篇综述概述了利用这些模型进行药物发现的策略。有些模型用于剖析肌张力障碍的发病机制,以确定干预的分子靶点。其他模型则用于经验性地确定候选药物。因此,这些动物模型为开发更好的肌张力障碍治疗方法提供了有前景的新工具。