Kieburtz Karl, Olanow C Warren
Neurology Department, University of Rochester Medical Center, Rochester, New York 14620, USA.
Mt Sinai J Med. 2007 Apr;74(1):7-14. doi: 10.1002/msj.20006.
In the past decade, there has been an increasing emphasis on laboratory-based translational research. This has led to significant scientific advances in our understanding of disease mechanisms and in the development of novel approaches to therapy such as gene therapy, RNA interference, and stem cells. However, the translation of these remarkable scientific achievements into new and effective disease-modifying therapies has lagged behind these scientific accomplishments. We use the term "translational experimental therapeutics" to describe the pathway between the discovery of a basic disease mechanism or novel therapeutic approach and its translation into an effective treatment for patients with a specific disease. In this article, we review the components of this pathway, and discuss issues that might impede this process. Only by optimizing this pathway can we realize the full therapeutic potential of current scientific discoveries and translate the astounding advances that have been accomplished in the laboratory into effective treatments for our patients.
在过去十年中，基于实验室的转化研究越来越受到重视。这在我们对疾病机制的理解以及新型治疗方法（如基因治疗、RNA干扰和干细胞）的开发方面带来了重大科学进展。然而，将这些卓越的科学成就转化为新的、有效的疾病修饰疗法却落后于这些科学成果。我们使用“转化实验治疗学”一词来描述从发现基本疾病机制或新型治疗方法到将其转化为针对特定疾病患者的有效治疗方法的过程。在本文中，我们回顾了这一过程的组成部分，并讨论了可能阻碍这一过程的问题。只有优化这一过程，我们才能实现当前科学发现的全部治疗潜力，并将实验室中取得的惊人进展转化为针对我们患者的有效治疗方法。
