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

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
Formato: article
Lenguaje:EN
Publicado: Elsevier 2022
Materias:
Wilson’s disease
induced pluripotent stem cell (iPSC)
iPSC-derived hepatocytes (iHeps)
ATPase copper transporting beta polypeptide (ATP7B)
Clustered regularly interspaced palindromic repeats (CRISPR)/Cas9
Single-stranded Oligodeoxynucleotide (ssODN)
Diseases of the digestive system. Gastroenterology
RC799-869
Acceso en línea:https://doaj.org/article/7251fd1424cb470896b40e7153068479
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https://doaj.org/article/7251fd1424cb470896b40e7153068479

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