Organoids-on-Chips Technology: Unveiling New Perspectives in Rare-Disease Research

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Abstract
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.
Funding Information
  • Strategic Priority Research Program of the Chinese Academy of Sciences (XDC 0110300, E2M9560201, TSBICIP-CXRC-008, 32301210, 31200035, 2022M713330)
  • Major Project of the Haihe Laboratory of Synthetic Biology (XDC 0110300, E2M9560201, TSBICIP-CXRC-008, 32301210, 31200035, 2022M713330)
  • Tianjin Synthetic Biotechnology Innovation Capacity Improvement Projects (XDC 0110300, E2M9560201, TSBICIP-CXRC-008, 32301210, 31200035, 2022M713330)
  • National Natural Science Foundation of China (XDC 0110300, E2M9560201, TSBICIP-CXRC-008, 32301210, 31200035, 2022M713330)
  • China Postdoctoral Science Foundation (XDC 0110300, E2M9560201, TSBICIP-CXRC-008, 32301210, 31200035, 2022M713330)

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