Hit Discovery and Lead Profiling, Pfizer, Groton, CT 06340, USA.
Rare Diseases Research Unit, Pfizer, Cambridge, MA 02139, USA.
Sci Transl Med. 2015 Jun 24;7(293):293ps15. doi: 10.1126/scitranslmed.aab1201.
Phenotypic drug discovery approaches can positively affect the translation of preclinical findings to patients. However, not all phenotypic assays are created equal. A critical question then follows: What are the characteristics of the optimal assays? We analyze this question and propose three specific criteria related to the disease relevance of the assay-system, stimulus, and end point-to help design the most predictive phenotypic assays.
表型药物发现方法可以积极影响临床前发现向患者的转化。然而,并非所有表型测定都是平等的。那么接下来就会出现一个关键问题:最佳测定的特征是什么?我们分析了这个问题,并提出了与测定系统、刺激物和终点与疾病相关性相关的三个具体标准,以帮助设计最具预测性的表型测定。