Li Xiangyang, Wang Hui, You Xiaoyan, Zhao Guoping
Henan Engineering Research Center of Food Microbiology, College of Food and Bioengineering, Henan University of Science and Technology, Luoyang 471023, China.
Master Lab for Innovative Application of Nature Products, National Center of Technology Innovation for Synthetic Biology, Tianjin Institute of Industrial Biotechnology, Chinese Academy of Sciences (CAS), Tianjin 300308, China.
Int J Mol Sci. 2025 May 4;26(9):4367. doi: 10.3390/ijms26094367.
The scarcity of robust models and therapeutic options for rare diseases continues to hamper their preclinical investigation. Traditional animal models and two-dimensional cell cultures are limited in their ability to replicate human heredity-associated traits and complex pathological features. Organoids-on-a-chip approaches open up new frontiers in rare-disease research via the integration of organ chips and organoid technology. This integrative strategy offers immense opportunities for the mimicry of disease-related traits, the clarification of the mechanisms underlying disease, and the prediction of treatment responses in a highly human-related manner. This forward-looking perspective suggests organoids on chips are transformative tools for parsing rare-disease pathogenesis, accelerating therapeutic discovery, and bridging the gap between basic research and precision medicine.
罕见病稳健模型和治疗选择的匮乏持续阻碍着它们的临床前研究。传统动物模型和二维细胞培养在复制人类遗传相关特征和复杂病理特征方面能力有限。芯片类器官方法通过整合器官芯片和类器官技术，为罕见病研究开辟了新领域。这种整合策略为以高度模拟人类的方式模拟疾病相关特征、阐明疾病潜在机制以及预测治疗反应提供了巨大机会。这种前瞻性观点表明，芯片类器官是剖析罕见病发病机制、加速治疗发现以及弥合基础研究与精准医学之间差距的变革性工具。
