利用 CRISPR/Cas9 技术生成 GPAM 基因敲除人胚胎干细胞系 SYSUe-008-A。
Generation of GPAM knockout human embryonic stem cell line SYSUe-008-A using CRISPR/Cas9.
机构信息
Center for Stem Cell Biology and Tissue Engineering, Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Department of Histology and Embryology, Zhongshan School of Medicine, Sun Yat-sen University, Guangzhou 510275, China; Laboratory of Medical Systems Biology, Guangzhou Institute of Pediatrics, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou 510623, China.
Center for Stem Cell Biology and Tissue Engineering, Key Laboratory for Stem Cells and Tissue Engineering, Ministry of Education, Department of Histology and Embryology, Zhongshan School of Medicine, Sun Yat-sen University, Guangzhou 510275, China.
出版信息
Stem Cell Res. 2021 May;53:102303. doi: 10.1016/j.scr.2021.102303. Epub 2021 Mar 19.
GPAM (glycerol-3-phosphateacyltransferase1) is a mitochondrial enzyme that catalyze an essential step in glycerolphospholipids and triacylglycerol biosynthesis process. Loss-of-function mutation of GPAM has been shown to lead to hypomyelination of corticospinal tract in cerebral palsy patient. To model this rare disease with human brain organoid, we generated a GPAM knockout human embryonic stem cell line SYSUe-008-A by CRISPR/cas9. The GPAM knockout cell line maintains a normal karyotype and shows comparable level of pluripotent stem cell marker expression and differentiation potential as wild-type human embryonic stem cells.
甘油-3-磷酸酰基转移酶 1（GPAM）是一种线粒体酶，可催化甘油磷脂和三酰基甘油生物合成过程中的一个必需步骤。已证实 GPAM 的功能丧失突变会导致脑瘫患者皮质脊髓束的髓鞘形成不良。为了用人脑类器官来模拟这种罕见疾病，我们通过 CRISPR/cas9 技术生成了 GPAM 敲除的人胚胎干细胞系 SYSUe-008-A。GPAM 敲除细胞系保持正常核型，并且具有与野生型人胚胎干细胞相当的多能干细胞标志物表达和分化潜能。
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