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Updated: Mar 12, 2026
Generation of iPSC-derived Human Brain Organoids to Model Early Neurodevelopmental Disorders
Published on: April 14, 2017
Modeling of Autism Using Organoid Technology
Hwan Choi1, Juhyun Song2, Guiyeon Park1
1Department of Biomedical Engineering (BK21Plus), Lab of Stem Cells and Reprogramming, Dongguk University, 3-ga, Pil-dong, Chung-gu, Seoul, 100-715, South Korea.
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.
Insights
Three-dimensional organoids offer a promising model for studying autism, a neurodevelopmental disease. Combining organoids with CRISPR/Cas9 gene editing can advance autism research by modeling genetic mutations.
Area of Science:
- Neuroscience
- Developmental Biology
- Genetics
Background:
- Autism Spectrum Disorder (ASD) is a complex neurodevelopmental disease driven by multiple genetic mutations.
- Current stem cell models have limitations in fully recapitulating ASD pathogenesis.
- A need exists for advanced models to study ASD molecular pathways and mechanisms.
Purpose of the Study:
- To review the advantages of three-dimensional (3D) organoid systems for modeling autism.
- To explore the potential of combining 3D organoids with genome editing technologies for ASD research.
Main Methods:
- Review of existing literature on autism modeling using stem cells and organoids.
- Discussion of the application of Clustered Regularly Interspaced Short Palindromic Repeat-associated protein 9 (CRISPR/Cas9) gene editing in organoid systems.
Main Results:
- 3D organoid systems provide a more physiologically relevant platform for studying neurodevelopmental diseases like autism.
- Organoids can better mimic the complex cellular architecture and interactions relevant to ASD.
- CRISPR/Cas9 technology enables precise genetic manipulation within organoids to model specific autism-associated mutations.
Conclusions:
- 3D organoid systems represent a significant advancement in modeling autism pathogenesis.
- The integration of CRISPR/Cas9 with 3D organoids offers a powerful tool for dissecting the genetic underpinnings of autism.
- This combined approach holds great potential to accelerate the understanding and development of therapeutic strategies for autism.
