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从携带 SQSTM1/p62 基因突变的神经退行性疾病表型患者中生成和鉴定人诱导多能干细胞系(IGIBi010-A)。

Generation and characterization of human-derived induced pluripotent stem cell line (IGIBi010-A) from a patient with neurodegenerative disease phenotype carrying mutation in SQSTM1/p62 gene.

机构信息

Genomics and Molecular Medicine Division, CSIR - Institute of Genomics and Integrative Biology, New Delhi 110007, India; Department of Neurology, All India Institute of Medical Sciences, New Delhi 110029, India.

Genomics and Molecular Medicine Division, CSIR - Institute of Genomics and Integrative Biology, New Delhi 110007, India.

出版信息

Stem Cell Res. 2024 Oct;80:103520. doi: 10.1016/j.scr.2024.103520. Epub 2024 Aug 5.

Abstract

SQSTM1 (Sequestosome 1) also known as p62, plays several important physiological roles in the cell. It regulates autophagy and mitochondrial homeostasis and can further lead to metabolic reprogramming. Pathogenic variants in SQSTM1 gene are known to cause Neurodegeneration with ataxia, dystonia, and gaze palsy in autosomal recessive inheritance fashion. We report here, the generation of induced pluripotent stem cell (iPSC) line (IGIBi010-A) carrying a novel homozygous frameshift variant in SQSTM1 i.e. p.Leu251SerfsTer4. In future, this iPSC line will be used as a resource to elucidate the molecular pathway, targeting strategies for disease biology derived by variation in SQSTM1 gene.

摘要

SQSTM1(自噬相关蛋白 1)也被称为 p62,在细胞中发挥着多种重要的生理作用。它调节自噬和线粒体稳态,并能进一步导致代谢重编程。SQSTM1 基因的致病性变异已知会导致常染色体隐性遗传方式的共济失调、肌张力障碍和眼球运动不能。我们在此报告,携带 SQSTM1 基因中新型纯合移码变异的诱导多能干细胞(iPSC)系(IGIBi010-A)的产生,即 p.Leu251SerfsTer4。将来,该 iPSC 系将被用作资源,以阐明由 SQSTM1 基因变异引起的疾病生物学的分子途径和靶向策略。

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