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CRISPR-targeted genome editing of human induced pluripotent stem cell-derived hepatocytes for the treatment of Wilson’s disease

BACKGROUND & AIMS: Wilson’s disease (WD) is an autosomal recessive disorder of copper metabolism caused by loss-of-function mutations in ATP7B, which encodes a copper-transporting protein. It is characterized by excessive copper deposition in tissues, predominantly in the liver and brain. We sou...

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Detalles Bibliográficos
Autores principales:	Wei, Rui, Yang, Jiayin, Cheng, Chi-Wa, Ho, Wai-In, Li, Na, Hu, Yang, Hong, Xueyu, Fu, Jian, Yang, Bo, Liu, Yuqing, Jiang, Lixiang, Lai, Wing-Hon, Au, Ka-Wing, Tsang, Wai-Ling, Tse, Yiu-Lam, Ng, Kwong-Man, Esteban, Miguel A., Tse, Hung-Fat
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2021
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8633686/ https://www.ncbi.nlm.nih.gov/pubmed/34877514 http://dx.doi.org/10.1016/j.jhepr.2021.100389

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