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

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.