hPSCs-derived brain organoids for disease modeling, toxicity testing and drug evaluation.

Due to the differences and variances in genetic background, in vitro and animal models cannot meet the modern medical exploration of real human brain structure and function. Recently, brain organoids generated by human pluripotent stem cells (hPSCs) can mimic the structure and physiological function of human brain, being widely used in medical research. Brain organoids generated from normal hPSCs or patient-derived induced pluripotent stem cells offer a more promising approach for the study of diverse human brain diseases. More importantly, the use of the established brain organoid model for drug evaluation is conducive to shorten the clinical transformation period. Herein, we summarize methods for the identification of brain organoids from cellular diversity, morphology and neuronal activity, brain disease modeling, toxicity testing, and drug evaluation. Based on this, it is hoped that this review will provide new insights into the pathogenesis of brain diseases and drug research and development, promoting the rapid development of brain science.