TY - JOUR
T1 - Modeling of Autism Using Organoid Technology
AU - Choi, Hwan
AU - Song, Juhyun
AU - Park, Guiyeon
AU - Kim, Jongpil
N1 - Publisher Copyright:
© 2016, Springer Science+Business Media New York.
PY - 2017/12/1
Y1 - 2017/12/1
N2 - 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.
AB - 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.
KW - Autism
KW - Clustered regularly interspersed short palindromic repeat-associated protein 9 (CRISPR/Cas9)
KW - Induced pluripotent stem cells (iPSCs)
KW - Organoids
UR - http://www.scopus.com/inward/record.url?scp=84995444927&partnerID=8YFLogxK
U2 - 10.1007/s12035-016-0274-8
DO - 10.1007/s12035-016-0274-8
M3 - Review article
C2 - 27844287
AN - SCOPUS:84995444927
SN - 0893-7648
VL - 54
SP - 7789
EP - 7795
JO - Molecular Neurobiology
JF - Molecular Neurobiology
IS - 10
ER -