Stem Cell Research ( IF 0.8 ) Pub Date : 2020-01-29 , DOI: 10.1016/j.scr.2020.101725 Hyeyeon Park 1 , Jinu Han 2 , Youngsun Lee 1 , Sungwook Kwak 1 , Soo Kyung Koo 1
Leber congenital amaurosis (LCA) is an inherited retinal dystrophy that is characterized by severe visual impairment in early infancy. We generated a human induced pluripotent stem cell (hiPSC) line, DKHi090-A, from peripheral blood mononuclear cells (PBMCs) of a patient with LCA, by using a Sendai virus-based gene delivery system. We confirmed that DKHi090-A has a nicotinamide mononucleotide adenyltransferase 1 (NMNAT1) mutation and normal karyotype. DKHi090-A line is pluripotent and is capable of multilineage differentiation. This cell line is registered and is available at the National Stem Cell Bank, Korea National Institute of Health.
中文翻译:
从Leber先天性黑蒙病患者中产生人诱导的多能干细胞系。
莱伯先天性黑蒙症(LCA)是一种遗传性视网膜营养不良,其特征是婴儿早期严重视力障碍。我们使用基于仙台病毒的基因传递系统,从LCA患者的外周血单核细胞(PBMC)生成了人诱导的多能干细胞(hiPSC)系DKHi090-A。我们证实DKHi090-A具有烟酰胺单核苷酸腺苷酸转移酶1(NMNAT1)突变和正常的核型。DKHi090-A品系多能,能够进行多系分化。该细胞系已注册,可在韩国国立卫生研究院国家干细胞库中获得。