Toward brain organoid-based precision medicine in neurodegenerative diseases

Abstract

In recent years, the concept of precision medicine—an approach to developing personalized drugs for disease prevention or treatment—has emerged as an up-and-coming field in medical research. However, there are numerous limitations to applying this approach to the treatment of neurodegenerative diseases, such as Alzheimer disease, because of the invasiveness of obtaining human brain samples. Meanwhile, the development of human brain organoids has become one of the most powerful in vitro research tools because these organoids can be established from induced pluripotent stem cells or embryonic stem cells. In this review, we discuss how we can effectively utilize brain organoids for precision medicine research in conjunction with a variety of high-end techniques such as clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (CRISPR-Cas9) genomic editing, integrative multi-omics analysis, 3D brain tissue clearing, and high-content screening confocal microscopy imaging systems. We herein provide new insights in order to materialize organoid-based precision medicine therapy and future directions.