Respiratory diseases are among the leading causes of morbidity and mortality worldwide, representing a major unmet medical need. New chemical entities rarely make it into the clinic to treat respiratory diseases, which is partially due to a lack of adequate predictive disease models and the limited availability of human lung tissues to model respiratory disease. Human pluripotent stem cells (hPSCs) may help fill this gap by serving as a scalable human in vitro model. In addition, human in vitro models of rare genetic mutations can be generated using hPSCs. hPSC-derived epithelial cells and organoids have already shown great potential for the understanding of disease mechanisms, for finding new potential targets by using high-throughput screening platforms, and for personalised treatments. These potentials can also be applied to other hPSC-derived lung cell types in the future. In this review, we will discuss how hPSCs have brought, and may continue to bring, major changes to the field of respiratory diseases by understanding the molecular mechanisms of the pathology and by finding efficient therapeutics.

呼吸系统疾病是全球发病率和死亡率的主要原因之一，代表着未满足的主要医疗需求。新的化学实体很少进入临床治疗呼吸系统疾病，部分原因是缺乏足够的预测疾病模型以及用于模拟呼吸系统疾病的人体肺组织的可用性有限。人类多能干细胞 (hPSC) 可以作为可扩展的人类体外模型来帮助填补这一空白。此外，人体体外可以使用 hPSC 生成罕见基因突变模型。hPSC 衍生的上皮细胞和类器官已经在理解疾病机制、通过使用高通量筛选平台寻找新的潜在靶点以及进行个性化治疗方面显示出巨大潜力。这些潜力在未来也可以应用于其他 hPSC 衍生的肺细胞类型。在这篇综述中，我们将讨论 hPSC 如何通过了解病理学的分子机制和寻找有效的治疗方法，为呼吸系统疾病领域带来并将继续带来重大变化。