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

Human molecular genetics - 15 Aug 2011

Zhang Shiqiang, Chen Shen, Li Wen, Guo Xiangpeng, Zhao Ping, Xu Jianyong, Chen Yan, Pan Qiong, Liu Xiaorong, Zychlinski Daniela, Lu Hai, Tortorella Micky D, Schambach Axel, Wang Yan, Pei Duanqing, Esteban Miguel A

Abstract excerpt

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...

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PMID: 21593220
DOI: 10.1093/hmg/ddr223

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Related research

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Research trail→

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Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin.PMID 21593220Read ↗

Establishment of hepatic and neural differentiation platforms of Wilson's disease specific induced pluripotent stem cells.Semantic proximity Read ↗

CRISPR-targeted genome editing of human induced pluripotent stem cell-derived hepatocytes for the treatment of Wilson’s diseaseSemantic proximity Read ↗

ATP7B gene therapy of autologous reprogrammed hepatocytes alleviates copper accumulation in a mouse model of Wilson's disease.Semantic proximity Read ↗

Generation of ZJUi003-A, an induced pluripotent stem cell line from a Wilson's disease patient carrying a c.180_181del mutation in ATP7B gene.Semantic proximity Read ↗

Characterization of the most frequent ATP7B mutation causing Wilson disease in hepatocytes from patient induced pluripotent stem cells.Semantic proximity Read ↗

Retinoids rescue ceruloplasmin secretion and alleviate oxidative stress in Wilson's disease-specific hepatocytes.Semantic proximity Read ↗

Retinoids rescue ceruloplasmin secretion and alleviate oxidative stress in Wilson’s disease-specific hepatocytesSemantic proximity Read ↗

Generation of two induced pluripotent stem cell lines from a female adult homozygous for the Wilson disease associated ATP7B variant p.H1069Q (AKOSi008-A) and a healthy control (AKOSi009-A).Semantic proximity Read ↗

Generation of an induced pluripotent stem cell (iPSC) line (THSJTUi001-A) from a Wilson's disease patient harboring a homozygous Arg778Leu mutation in ATP7B gene.Semantic proximity Read ↗

Stem Cells Treatment for Wilson Disease.Semantic proximity Read ↗

[Research progress in gene therapy for Wilson's disease].Semantic proximity Read ↗

CRISPR/Cas9-mediated correction of mutated copper transporter ATP7B.Semantic proximity Read ↗

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