Research Priority Area
Stem Cell and Organoid Medicine

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Pioneering the Future of Human Biology with Stem Cell and Organoid Science

Cincinnati Children’s is a global leader in stem cell and organoid research, advancing technologies that are redefining how we study human development, disease mechanisms, and develop new therapeutic strategies. Through the Center for Stem Cell & Organoid Medicine (CuSTOM), researchers across more than a dozen divisions are developing models that mimic human tissues with remarkable fidelity—offering new insights into biology and unlocking opportunities for regenerative medicine, drug discovery, and clinical translation.

Since generating the world’s first functional intestinal organoid from iPSCs in 2010, our teams have expanded the science to include brain, liver, lung, stomach, esophagus, pancreas, colon, kidney, and vascular systems. These platforms now serve as living models to study conditions ranging from neurodegenerative disease and cancer to diabetes, inflammatory bowel disease (IBD), and COVID-19. With integrated immune systems and advanced tissue engineering, our organoids increasingly reflect the complexity of human physiology.

Recent advances include blood-brain barrier assembloids for modeling neurological disease, liver organoids for drug screening and bilirubin clearance, and gut organoids with immune cells to study IBD. Our researchers are also pioneering applications in biocomputing, vascular regeneration, and organoid-based tissue therapeutics—supported by major international collaborations and investments in translational infrastructure.

By merging stem cell biology, clinical need, and engineering innovation, Cincinnati Children’s is helping set the future course of organoid science and regenerative medicine.

View looking into the CuSTOM Accelerator laboratory, pictured, Thursday, Feb. 6, 2025, at the Burnet Campus of Cincinnati Children’s.
Research Spotlight

Organoid Breakthrough: Human Cell Therapy Repairs Damaged Intestine in Preclinical Study

In a major milestone for regenerative medicine, Cincinnati Children’s researchers report the first successful use of human organoid tissue to repair intestinal damage in a rodent model. Published in Cell Stem Cell, the study sets the stage for upcoming first-in-human clinical trials targeting Crohn’s disease, ulcerative colitis, and more.

Research Centers Pushing the Boundaries of Stem Cell and Organoid Medicine

From basic biology to translational breakthroughs, our research centers are pioneering how stem cells and organoids can transform diagnosis, treatment, and healing.