title: Modeling of Autism Using Organoid Technology.
abstract: Autism is a neurodevelopmental disease caused by multiple mutations during development. However, a suitable disease model to study the molecular pathway of disease onset and progression is not available. Although many studies have used human stem cells such as induced pluripotent stem cells and embryonic stem cells to investigate the disease pathogenesis, these stem cell techniques are limited in their abilities to study the pathology and mechanism of pathogenesis of neurodevelopmental diseases such as autism. Therefore, researchers are focusing on the strengths of three-dimensional (3D) structures mimicking organs, organoids, for modeling autism. In this review, we highlight the advantages of 3D organoid systems to investigate the mechanisms of the pathogenesis of autism. Further, because the onset of autism is determined by genetic background, we suggest the application of the clustered regularly interspersed short palindromic repeat-associated protein 9 (CRISPR/Cas9) technique for genome editing in 3D organoid systems to study mutations that cause autism. We propose that 3D organoid systems combined with the CRISPR/Cas9 technique may advance autism research.
authors.affiliation: Department of Biomedical Engineering (BK21Plus), Lab of Stem Cells and Reprogramming, Dongguk University, 3-ga, Pil-dong, Chung-gu, Seoul, 100-715, South Korea.
authors.forename: Hwan
authors.initials: H
authors.lastname: Choi
authors.affiliation: Department of Neurology, Chonnam University Medical School, Gwangju, South Korea.
authors.forename: Juhyun
authors.initials: J
authors.lastname: Song
authors.affiliation: Department of Biomedical Engineering (BK21Plus), Lab of Stem Cells and Reprogramming, Dongguk University, 3-ga, Pil-dong, Chung-gu, Seoul, 100-715, South Korea.
authors.forename: Guiyeon
authors.initials: G
authors.lastname: Park
authors.affiliation: Department of Biomedical Engineering (BK21Plus), Lab of Stem Cells and Reprogramming, Dongguk University, 3-ga, Pil-dong, Chung-gu, Seoul, 100-715, South Korea. jk2316@gmail.com.
authors.forename: Jongpil
authors.initials: J
authors.lastname: Kim
mesh_terms: D000818:Animals; D001321:Autistic Disorder / Q000235:genetics / Q000473:pathology* / Q000628:therapy*; D064113:CRISPR-Cas Systems / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods / Q000639:trends; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000637:transplantation*; D009924:Organ Culture Techniques; D009940:Organoids / Q000166:cytology / Q000378:metabolism / Q000473:pathology*
keywords: Autism; Clustered regularly interspersed short palindromic repeat-associated protein 9 (CRISPR/Cas9); Induced pluripotent stem cells (iPSCs); Organoids