通过CRISPR/Cas9基因组编辑生成ST3GAL3基因敲除的诱导多能干细胞（iPSC）系（UKWMPi002-A-3）。
Generation of a ST3GAL3 null mutant induced pluripotent stem cell (iPSC) line (UKWMPi002-A-3) by CRISPR/Cas9 genome editing.
作者信息
Diouf David, Vitale Maria Rosaria, Zöller Johanna Eva Maria, Pineau Ana-Magdalena, Klopocki Eva, Hamann Catharina, Ziegler Georg Christoph, Vanmierlo Tim, Van den Hove Daniel, Lesch Klaus-Peter
机构信息
Division of Molecular Psychiatry, Center of Mental Health, University Hospital Würzburg, Würzburg, Germany.
Division of Molecular Psychiatry, Center of Mental Health, University Hospital Würzburg, Würzburg, Germany.
出版信息
Stem Cell Res. 2023 Mar;67:103038. doi: 10.1016/j.scr.2023.103038. Epub 2023 Jan 31.
Fibroblasts isolated from a skin biopsy of a healthy individual were infected with Sendai virus containing the Yamanaka factors to produce transgene-free human induced pluripotent stem cells (iPSCs). CRISPR/Cas9 was used to generate an isogenic cell line carrying an inactivation of ST3GAL3, a risk gene associated with neurodevelopmental and psychiatric disorders. This ST3GAL3 null mutant (ST3GAL3-/-) iPSC line, which displays the expression of pluripotency-associated markers, the ability to differentiate into cells of the three germ layers in vitro, and a normal karyotype, is a powerful tool to investigate the impact of deficient sialylation of glycoproteins in neural development and plasticity.
从健康个体的皮肤活检中分离出的成纤维细胞，用含有山中因子的仙台病毒感染，以产生无转基因的人诱导多能干细胞（iPSC）。利用CRISPR/Cas9技术构建了一个携带ST3GAL3基因失活的同基因细胞系，ST3GAL3是一种与神经发育和精神疾病相关的风险基因。该ST3GAL3基因敲除突变体（ST3GAL3-/-）iPSC系，表现出多能性相关标志物的表达、体外分化为三个胚层细胞的能力以及正常的核型，是研究神经发育和可塑性中糖蛋白唾液酸化缺陷影响的有力工具。
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