Article
In vitro genome editing rescues parkinsonism phenotypes in induced pluripotent stem cells-derived dopaminergic neurons carrying LRRK2 p.G2019S mutation.
Stem cell research & therapy - 22 Sept 2021
Chang Kuo-Hsuan, Huang Cheng-Yen, Ou-Yang Chih-Hsin, Ho Chang-Han, Lin Han-Yi, Hsu Chia-Lang, Chen You-Tzung, Chou Yu-Chi, Chen Yi-Jing, Chen Ying, Lin Jia-Li, Wang Ji-Kuan, Lin Pei-Wen, Lin Ying-Ru, Lin Miao-Hsia, Tseng Chi-Kang, Lin Chin-Hsien
Abstract excerpt
BACKGROUND: The c.G6055A (p.G2019S) mutation in leucine-rich repeat kinase 2 (LRRK2) is the most prevalent genetic cause of Parkinson's disease (PD). CRISPR/Cas9-mediated genome editing by homology-directed repair (HDR) has been applied to correct the mutation but may create small insertions and deletions (indels) due to double-strand DNA breaks. Adenine base editors (ABEs) could convert targeted A·T to G·C in...
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