Article
CRISPR-Cas9 Gene Editing Protects from the A53T-SNCA Overexpression-Induced Pathology of Parkinson's Disease In Vivo.
The CRISPR journal - 1 Feb 2022
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, Lee C Justin, Nam Min-Ho, Hur Junseok W, Jeon Sang Ryong
Abstract excerpt
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...
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