In vitro genome editing rescues parkinsonism phenotypes in induced pluripotent stem cells-derived dopaminergic neurons carrying LRRK2 p.G2019S mutation.

Kuo-Hsuan ChangCheng-Yen HuangChih-Hsin Ou-YangChang-Han HoHan-Yi LinChia-Lang HsuYou-Tzung ChenYu-Chi ChouYi-Jing ChenYing ChenJia-Li LinJi-Kuan WangPei-Wen LinYing-Ru LinMiao-Hsia LinChi-Kang TsengChin-Hsien Lin

Published in: Stem cell research & therapy (2021)

The results of this study envision that ABE could directly correct the pathogenic mutation in iPSCs for reversing disease-related phenotypes in neuropathology and exploring novel pathophysiological targets in PD.

Keyphrases

induced pluripotent stem cells
genome editing
crispr cas
spinal cord
mouse model
parkinson disease
drug induced