Generation of homozygous PRKN, PINK1 and double PINK1/PRKN knockout cell lines from healthy induced pluripotent stem cells using CRISPR/Cas9 editing

被引：11

作者：

Chen, Carol X. -Q. ^{[1
]}

You, Zhipeng ^{[1
]}

Abdian, Narges ^{[1
]}

Sirois, Julien ^{[1
]}

Shlaifer, Irina ^{[1
]}

Tabatabaei, Mahdieh ^{[2
]}

Boivin, Marie-Noelle ^{[2
]}

Gaborieau, Lydiane ^{[2
]}

Karamchandani, Jason ^{[2
]}

Beitel, Lenore K. ^{[1
]}

Fon, Edward A. ^{[3
,4
]}

Durcan, Thomas M. ^{[1
]}

机构：

[1] McGill Univ, Neuros Early Drug Discovery Unit EDDU, 3801 Univ St, Montreal, PQ H3A 2B4, Canada

[2] McGill Univ, C BIG Repository C BIG, Montreal Neurol Inst, Montreal, PQ H3A 2B4, Canada

[3] Montreal Neurol Inst, Dept Neurol & Neurosurg, McGill Parkinson Program, Montreal, PQ H3A 2B4, Canada

[4] Montreal Neurol Inst, Dept Neurol & Neurosurg, Neurodegenerat Dis Grp, Montreal, PQ H3A 2B4, Canada

来源：

STEM CELL RESEARCH | 2022年 / 62卷

关键词：

D O I：

10.1016/j.scr.2022.102806

中图分类号：

Q813 [细胞工程];

学科分类号：

摘要：

Autosomal recessive mutations in either PRKN or PINK1 are associated with early-onset Parkinson's disease. The corresponding proteins, PRKN, an E3 ubiquitin ligase, and the mitochondrial serine/threonine-protein kinase PINK1 play a role in mitochondrial quality control. Using CRISPR/CAS9 technology we generated three human iPSC lines from the well characterized AIW002-02 control line. These isogenic iPSCs contain homozygous knockouts of PRKN (PRKN-KO, CBIGi001-A-1), PINK1 (PINK1-KO, CBIGi001-A-2) or both PINK1 and PRKN (PINK1-KO/PRKN-KO, CBIGi001-A-3). The knockout lines display normal karyotypes, express pluripotency markers and upon differentiation into relevant brain cells or midbrain organoids may be valuable tools to model Parkinson's disease.

引用

页数：7

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