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

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 del...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8456557/
https://www.ncbi.nlm.nih.gov/pubmed/34551822
http://dx.doi.org/10.1186/s13287-021-02585-2