Advancing brain organoids from disease phenotypes to mechanisms and translation across developmental, infectious, and neurodegenerative diseases.

Human brain organoids are advancing from modeling central nervous system disease phenotypes toward defining human-specific molecular and cellular mechanisms and trajectories.

• Why it matters: Central nervous system diseases remain an unmet medical challenge, and animal models can miss human-specific processes needed for mechanism discovery and therapy development.
• What they did: The field integrates human genetics with longitudinal, single-cell, multi-omic, and functional analyses, using well-characterized organoids as human new approach methodologies for preclinical therapeutic evaluation.
• The result: Reproducibility, benchmarking, and appropriate validation are critical to establishing predictive value, enabling mechanism-anchored therapeutic targets, and supporting drug development and regulatory use.

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