Collaborative Laboratory to Foster Translation and Drug Discovery, Accelbio, 3030-197 Cantanhede, Portugal.
IBB-Institute for Bioengineering and Biosciences, Department of Bioengineering Instituto Superior Técnico, Universidade de Lisboa, 1049-001 Lisboa, Portugal.
Int J Mol Sci. 2023 Mar 2;24(5):4857. doi: 10.3390/ijms24054857.
Today, it is recognized that medicines will eventually be needed during pregnancy to help prevent to, ameliorate or treat an illness, either due to gestation-related medical conditions or pre-existing diseases. Adding to that, the rate of drug prescription to pregnant women has increased over the past few years, in accordance with the increasing trend to postpone childbirth to a later age. However, in spite of these trends, information regarding teratogenic risk in humans is often missing for most of the purchased drugs. So far, animal models have been the gold standard to obtain teratogenic data, but inter-species differences have limited the suitability of those models to predict human-specific outcomes, contributing to misidentified human teratogenicity. Therefore, the development of physiologically relevant in vitro humanized models can be the key to surpassing this limitation. In this context, this review describes the pathway towards the introduction of human pluripotent stem cell-derived models in developmental toxicity studies. Moreover, as an illustration of their relevance, a particular emphasis will be placed on those models that recapitulate two very important early developmental stages, namely gastrulation and cardiac specification.
如今,人们认识到,为了预防、改善或治疗妊娠相关疾病或已有疾病,孕妇最终可能需要用药。此外,近年来,随着生育年龄的推迟,孕妇的用药率有所上升。然而,尽管存在这些趋势,但对于大多数已购药物,其在人类致畸风险方面的信息往往缺失。到目前为止,动物模型一直是获得致畸数据的金标准,但种间差异限制了这些模型对人类特异性结果的适用性,导致对人类致畸性的错误识别。因此,开发与生理相关的体外人源化模型可能是克服这一限制的关键。在这种情况下,本文综述了将人类多能干细胞衍生模型引入发育毒性研究的途径。此外,作为相关性的例证,本文将特别强调那些能够重现两个非常重要的早期发育阶段(即原肠胚形成和心脏特化)的模型。