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인지 및 사회성 연구단
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CRISPR-Cas9 Gene Editing Protects from the A53T-SNCA Overexpression-Induced Pathology of Parkinson's Disease in Vivo

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Title
CRISPR-Cas9 Gene Editing Protects from the A53T-SNCA Overexpression-Induced Pathology of Parkinson's Disease in Vivo
Author(s)
Yoon, Hyung Ho; Ye, Sunghyeok; Lim, Sunhwa; Jo, Ara; Lee, Hawon; Hong, Felix; Lee, Seung Eun; Oh, Soo-Jin; Kim, Na-Rae; Kim, Kyoungmi; Kim, Bum-Joon; Kim, Hyunjin; C. Justin Lee; Nam, Min-Ho; Hur, Junseok W.; Jeon, Sang Ryong
Publication Date
2022-02-01
Journal
CRISPR Journal, v.5, no.1, pp.95 - 108
Publisher
Mary Ann Liebert Inc.
Abstract
© Copyright 2022, Mary Ann Liebert, Inc., publishers 2022.Mutations in specific genes, including synuclein alpha (SNCA) that encodes the α-synuclein protein, are known to be risk factors for sporadic Parkinson's disease (PD), as well as critical factors for familial PD. In particular, A53T-mutated SNCA (A53T-SNCA) is a well-studied familial pathologic mutation in PD. However, techniques for deletion of the mutated SNCA gene in vivo have not been developed. Here, we used the CRISPR-Cas9 system to delete A53T-SNCA in vitro as well as in vivo. Adeno-associated virus carrying SaCas9-KKH with a single-guide RNA targeting A53T-SNCA significantly reduced A53T-SNCA expression levels in vitro. Furthermore, we tested its therapeutic potential in vivo in a viral A53T-SNCA-overexpressing rat model of PD. Gene deletion of A53T-SNCA significantly rescued the overexpression of α-synuclein, reactive microgliosis, dopaminergic neurodegeneration, and parkinsonian motor symptoms. Our findings propose CRISPR-Cas9 system as a potential prevention strategy for A53T-SNCA-specific PD.
URI
https://pr.ibs.re.kr/handle/8788114/11302
DOI
10.1089/crispr.2021.0025
ISSN
2573-1599
Appears in Collections:
Center for Cognition and Sociality(인지 및 사회성 연구단) > 1. Journal Papers (저널논문)
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