Production and validation of human induced pluripotent stem cell line from sporadic amyotrophic lateral sclerosis (SALS)
Production and validation of human induced pluripotent stem cell line from sporadic amyotrophic lateral sclerosis (SALS)
复制标题
散发性肌萎缩侧索硬化症 (SALS) 人诱导多能干细胞系的产生和验证。
DOI：
10.1016/j.scr.2020.101760
复制
发表时间：
2020-04-01
影响因子：
1.2
通讯作者：
Cui, Huixian
中科院分区：
文献类型：
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作者：
Ma, Jun;Feng, Baofeng;Cui, Huixian
肌萎缩侧索硬化症（amyotrophiclateralsclerosis，ALS）是一种以大脑皮层上运动神经元和脑干及脊髓下运动神经元退行性缺失为主要表现的神经退行性疾病。绝大多数ALS病例没有家族史，显然是散发性的（SALS），这使得SALS的建模对于ALS疗法的开发至关重要。因此，使用无病毒方案从64岁SALS患者的外周血单核细胞产生人诱导多能干细胞（iPSC），并使用标准验证方法表征。这种产生的iPSC细胞系可能有助于揭示SALS机制和筛选药物开发。
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with the loss of upper motor neurons in the cortex and lower motor neurons in the brain stem and spinal cord regressively. The vast majority of ALS cases have no familial history are apparently sporadic (SALS), making the modeling of SALS essential to the development of ALS therapeutics. Therefore, human induced pluripotent stem cell (iPSC) from peripheral blood mononuclear cells of a 64-year-old SALS patient were produced using a virus-free protocol and characterized using standard validate methods. This generated iPSC line could be useful to reveal SALS mechanisms and screen drug development.