Generation of integration-free induced pluripotent stem cell line (NJMUi001-A) from a phenylketonuria patient
2017
Tianhui Xu | Dong Liang | Jingjing Zhang | Xiuqing Ji | Huanran Hu | Yun Sun | Tao Jiang | Xia Wang | Ping Hu | Zhengfeng Xu
PKU is a prevalent type of inherited metabolic disease, caused by the defective phenylalanine metabolism. In most PKU cases, mutations in the PAH gene could be found. Dysfunction of this hepatic enzyme will lead to diverse clinical symptoms due to a failure in converting phenylalanine into tyrosine. Here, we report an integration-free human induced pluripotent stem cell line (NJMUi001-A) generated from peripheral blood mononuclear cells of a PKU patient by using Sendai virus. This iPS cell line has characteristics of pluripotent stem cells and can be used as a useful tool for the investigation of this inherited metabolic disease.
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