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Modeling of Autism Using Organoid Technology

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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.

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Acknowledgements

This work was supported by the Korea Health Technology R&D Project, Ministry of Health and Welfare (HI16C1176).

Author Contributions

Choi Hwan and Juhyun Song wrote the preliminary draft and revised details of the manuscript. Guiyeon Park drew the figure. Jongpil Kim revised all manuscript in detail.

Author information

Authors and Affiliations

  1. Department of Biomedical Engineering (BK21Plus), Lab of Stem Cells and Reprogramming, Dongguk University, 3-ga, Pil-dong, Chung-gu, Seoul, 100-715, South Korea

    Hwan Choi, Guiyeon Park & Jongpil Kim

  2. Department of Neurology, Chonnam University Medical School, Gwangju, South Korea

    Juhyun Song

Authors
  1. Hwan Choi
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  2. Juhyun Song
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  3. Guiyeon Park
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  4. Jongpil Kim
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Corresponding author

Correspondence to Jongpil Kim.

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The authors declare that they have no conflicts of interest.

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Hwan Choi and Juhyun Song are equally contributed

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Choi, H., Song, J., Park, G. et al. Modeling of Autism Using Organoid Technology. Mol Neurobiol 54, 7789–7795 (2017). https://doi.org/10.1007/s12035-016-0274-8

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  • DOI: https://doi.org/10.1007/s12035-016-0274-8

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