Fig. 2

Therapeutic development strategies using hiPSC-based 3D brain organoid technology. Patient-derived iPSCs can be differentiated into brain organoids exhibiting disease-specific phenotypes (a,b). Three main approaches can be used to develop innovative therapeutic strategies (C-E). (c) For drug development strategies, HTS enables a large number of chemicals to be screened, but the prospective drug approach only allows a small number of defined drugs to be examined on brain organoids. (d) For transcriptome analysis, RNA sequencing (RNA-Seq) along with brain organoids provides an opportunity for scientists in studying the transcriptional profiling of the human complex neurological disorders. (e) For genome-editing approach, CRISPR-Cas9 can correct a genetic defect associated with disease phenotypes in brain organoids. Gene-corrected brain organoids could be used to replace impaired brain tissue via transplantation