Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin.

@article{Zhang2011RescueOA,
  title={Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin.},
  author={Shiqiang Zhang and Shen Chen and Wen Li and Xiangpeng Guo and Ping Zhao and Jianyong Xu and Yan Chen and Qiong Pan and Xiaorong Liu and Daniela Zychlinski and Hongchao Lu and Micky D. Tortorella and Michellle Cross and Yan Wang and Duanqing Pei and Miguel Aguilar Esteban},
  journal={Human molecular genetics},
  year={2011},
  volume={20 16},
  pages={
          3176-87
        }
}
Directed hepatocyte differentiation from human induced pluripotent stem cells (iPSCs) potentially provides a unique platform for modeling liver genetic diseases and performing drug-toxicity screening in vitro. Wilson's disease is a genetic disease caused by mutations in the ATP7B gene, whose product is a liver transporter protein responsible for coordinated copper export into bile and blood. Interestingly, the spectrum of ATP7B mutations is vast and can influence clinical presentation (a… CONTINUE READING

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