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...
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Autores principales: | Rui Wei, Jiayin Yang, Chi-Wa Cheng, Wai-In Ho, Na Li, Yang Hu, Xueyu Hong, Jian Fu, Bo Yang, Yuqing Liu, Lixiang Jiang, Wing-Hon Lai, Ka-Wing Au, Wai-Ling Tsang, Yiu-Lam Tse, Kwong-Man Ng, Miguel A. Esteban, Hung-Fat Tse |
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Formato: | article |
Lenguaje: | EN |
Publicado: |
Elsevier
2022
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Materias: | |
Acceso en línea: | https://doaj.org/article/7251fd1424cb470896b40e7153068479 |
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