Drug toxicity and efficacy are difficult to predict partly because they are both poorly defined, which I aim to remedy here from a transcriptomic perspective. There are two major categories of drugs: (1) restorative drugs aiming to restore an abnormal cell, tissue, or organ to normal function (e.g., restoring normal membrane function of epithelial cells in cystic fibrosis), and (2) disruptive drugs aiming to kill pathogens or malignant cells. These two types of drugs require different definition of efficacy and toxicity. I outlined rationales for defining transcriptomic efficacy and toxicity and illustrated numerically their application with two sets of transcriptomic data, one for restorative drugs (treating cystic fibrosis with lumacaftor/ivacaftor aiming to restore the cellular function of epithelial cells) and the other for disruptive drugs (treating acute myeloid leukemia with prexasertib). The conceptual framework presented will help and sensitize researchers to collect data required for determining drug toxicity.

药物毒性和功效很难预测，部分原因是它们的定义不明确，我打算从转录组学角度对此进行补救。药物分为两大类：（1）旨在使异常细胞，组织或器官恢复正常功能（例如，在囊性纤维化中恢复上皮细胞的正常膜功能）的修复药物，以及（2）旨在破坏细胞，组织或器官的功能。杀死病原体或恶性细胞。这两类药物要求对功效和毒性有不同的定义。我概述了定义转录组功效和毒性的基本原理，并通过两组转录组数据以数字方式说明了它们的应用，一种用于修复性药物（使用lumacaftor / ivacaftor治疗囊性纤维化，旨在恢复上皮细胞的细胞功能），另一种用于破坏性药物（使用prexasertib治疗急性髓性白血病）。提出的概念框架将有助于并使研究人员更加敏感地收集确定药物毒性所需的数据。