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Review
. 2025 May;26(5):389-412.
doi: 10.1038/s41580-024-00804-1. Epub 2024 Dec 12.

Modelling human brain development and disease with organoids

Marcella Birtele  1   2 , Madeline Lancaster  3   4 , Giorgia Quadrato  5   6
Affiliations
Review

Modelling human brain development and disease with organoids

Marcella Birtele et al. Nat Rev Mol Cell Biol. 2025 May.

Erratum in

Abstract

Organoids are systems derived from pluripotent stem cells at the interface between traditional monolayer cultures and in vivo animal models. The structural and functional characteristics of organoids enable the modelling of early stages of brain development in a physiologically relevant 3D environment. Moreover, organoids constitute a tool with which to analyse how individual genetic variation contributes to the susceptibility and progression of neurodevelopmental disorders. This Roadmap article describes the features of brain organoids, focusing on the neocortex, and their advantages and limitations - in comparison with other model systems - for the study of brain development, evolution and disease. We highlight avenues for enhancing the physiological relevance of brain organoids by integrating bioengineering techniques and unbiased high-throughput analyses, and discuss future applications. As organoids advance in mimicking human brain functions, we address the ethical and societal implications of this technology.

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Conflict of interest statement

Competing interests: The authors declare no competing interests.

References

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