The present application provides a novel drug screening platform designed to identify drugs that favorably modulate cellular bioenergetics in the human brain of a zebrafish which has been modified by altering a teleost gene that corresponds to a human gene associated with a brain dysfunction disorder (such as epilepsy). The drug screening platform are also useful for determining a mutation in a human gene associated with a brain dysfunction disorder that is associated with a human individual responsive to the treatment of a compound. Also provided are modified teleosts useful for the methods described herein.
本申请提供了一种新颖的药物筛选平台,该平台的设计目的是鉴别出有利于调节斑马鱼人脑中细胞
生物能的药物,斑马鱼是通过改变与脑功能障碍疾病(如癫痫)相关人类
基因相对应的驯养鱼类
基因而被改造的。该药物筛选平台还可用于确定与脑功能障碍疾病相关的人类
基因中的突变,该突变与对化合物治疗有反应的人类个体相关。还提供了对本文所述方法有用的改良远缘动物。