骨形态发生蛋白（BMP）信号传导的干扰导致了多种罕见遗传疾病的发生和发展，包括纤维性骨化进行性畸形（FOP）、肺动脉高压（PAH）和遗传性出血性毛细血管扩张症（HHT）。经过几十年的动物研究，建立了对潜在分子机制的理解的坚实基础，基于诱导多能干细胞（iPSC）的患者衍生模型的逐步推行将通过在复杂人体化环境中评估药物的疗效、特异性和毒性，提高药物开发的效果。我们将综述这一领域iPSC衍生模型系统的现状文献，并特别强调对患者来源材料的获取和相关的困难。鉴于BMP在胚胎发育和干细胞分化过程中的关键作用，BMP信号传导通路中的增益或缺失功能突变可能会影响iPSC的生成、维持和分化过程。本综述强调了对使用的方案进行仔细优化的必要性。最后，我们将讨论近期对复杂体外培养模型的进展，旨在模拟组织微环境的多方面细胞因素输入，如细胞力学和细胞外基质（ECM），结合了器官样体、器官-芯片和微流体技术。

Disturbances in bone morphogenetic protein (BMP) signalling contribute to onset and development of a number of rare genetic diseases, including Fibrodysplasia ossificans progressiva (FOP), Pulmonary arterial hypertension (PAH), and Hereditary haemorrhagic telangiectasia (HHT). After decades of animal research to build a solid foundation in understanding the underlying molecular mechanisms, the progressive implementation of iPSC-based patient-derived models will improve drug development by addressing drug efficacy, specificity, and toxicity in a complex humanized environment. We will review the current state of literature on iPSC-derived model systems in this field, with special emphasis on the access to patient source material and the complications that may come with it. Given the essential role of BMPs during embryonic development and stem cell differentiation, gain- or loss-of-function mutations in the BMP signalling pathway may compromise iPSC generation, maintenance, and differentiation procedures. This review highlights the need for careful optimization of the protocols used. Finally, we will discuss recent developments towards complex in vitro culture models aiming to resemble specific tissue microenvironments with multi-faceted cellular inputs, such as cell mechanics and ECM together with organoids, organ-on-chip, and microfluidic technologies.