Neural Regeneration Research ›› 2026, Vol. 21 ›› Issue (7): 2699-2715.doi: 10.4103/NRR.NRR-D-24-01546

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Brain organoids and genome editing: A new era in understanding human brain development and disorders

Min Zhou1, 2, 3, #, Yuanqing Cao4, 5, #, Ke Yue1, 3, #, Wenyu Wu1, 2, 3, Yutong Xie4, 5, Daiyu Hu4, 5, Jingjing Zhao1, 2, 3, Fang Xu5 , Jianrong Guo3 , Zhenzhou Li2, *, Huan Wang3, *, Zhengliang Gao3, 4, 6, *   

  1. 1 Clinical Research Center for Mental Health, Mental Health Center Affiliated to School of Medicine (Shanghai Hongkou Mental Health Center), Shanghai University, Shanghai, China;  2 Department of Anesthesiology and Perioperative Medicine, General Hospital of Ningxia Medical University, Yinchuan, Ningxia Hui Autonomous Region, China;  3 Department of Anesthesiology, Gongli Hospital of Shanghai Pudong New Area, Shanghai, China;  4 Fundamental Research Center, Shanghai Yangzhi Rehabilitation Hospital (Shanghai Sunshine Rehabilitation Center), School of Medicine, Tongji University, Shanghai, China;  5 School of Basic Medicine, Medical Genetics and Cell Biology, Ningxia Medical University, Yinchuan, Ningxia Hui Autonomous Region, China;  6 China-Japan Friendship Medical Research Institute, School of Medicine, Shanghai University, Shanghai, China
  • Online:2026-07-15 Published:2025-10-17
  • Contact: Zhengliang Gao, PhD, zhengliang_gao@shu.edu.cn; Huan Wang, PhD, Cheery_Wang@usst.edu.cn; Zhenzhou Li, PhD, lizhenzhou321@163.com.
  • Supported by:
    This work was supported by the Special Project for Clinical Research of Shanghai Municipal Health Commission, No. 202140403 (to HW); Key Disciplines Group Construction Project of Pudong Health Bureau of Shanghai, No. PWZxq 2022-05 (to JG); Natural Science Foundation of Ningxia Hui Autonomous Region, No. 2024AAC05084 (to ZL); Ningxia Hui Autonomous Region Key Research and Development Program, No. 2021BEG03084 (to FX); and National Natural Science Foundation of China, Nos. 32370895 and 32070862 (both to ZG).

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Abstract: Brain organoids are artificial neural tissues derived in vitro, containing a variety of cell types, as well as structural and/or functional brain regions. They can partially mimic brain physiological activities and diseased processes. Owing to their operability and sample accessibility, brain organoids serve as a bridge between in vitro monolayer cell culture models and in vivo animal models. An increasing number of induction protocols for brain organoids have been developed over the preceding decade. A key future research direction will focus on ensuring the complexity and quality of brain organoids. The integration of powerful technologies, such as the CRISPR/Cas9 genome editing and lineage tracing systems, shall precipitate practical and broad applications of brain organoids. In this review, we discuss the generation and application of brain organoids, as well as their integration with genome editing technologies, in the study of neural development, disease modeling, and mechanistic investigations. The innovative combination of these two technologies may offer a fresh perspective for exploring the fundamental aspects of the human nervous system and related diseases.

Key words: brain disorder, brain organoid, CRISPR/Cas9, disease modeling, drug screening, genome editing, human brain development, lineage tracing, organoid modeling, stem cell differentiation