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

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

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.

Affiliation

Wei R; The Cardiology Division, Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China.
Yang J; Hong Kong-Guangdong Stem Cell and Regenerative Medicine Research Centre, The University of Hong Kong and Guangzhou Institutes of Biomedicine and Health, Hong Kong, China.
Cheng CW; Center for Translational Stem Cell Biology, Hong Kong, China.
Ho WI; The Cardiology Division, Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China.
Li N; Cell Inspire Therapeutics Co., Ltd and Cell Inspire Biotechnology Co., Ltd, Shenzhen 518102, China.
Hu Y; The Cardiology Division, Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China.
Hong X; Hong Kong-Guangdong Stem Cell and Regenerative Medicine Research Centre, The University of Hong Kong and Guangzhou Institutes of Biomedicine and Health, Hong Kong, China.
Fu J; The Cardiology Division, Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China.
Yang B; Hong Kong-Guangdong Stem Cell and Regenerative Medicine Research Centre, The University of Hong Kong and Guangzhou Institutes of Biomedicine and Health, Hong Kong, China.
Liu Y; The Cardiology Division, Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China.
Jiang L; Hong Kong-Guangdong Stem Cell and Regenerative Medicine Research Centre, The University of Hong Kong and Guangzhou Institutes of Biomedicine and Health, Hong Kong, China.
Lai WH; The Cardiology Division, Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China.
Au KW; Hong Kong-Guangdong Stem Cell and Regenerative Medicine Research Centre, The University of Hong Kong and Guangzhou Institutes of Biomedicine and Health, Hong Kong, China.
Tsang WL; The Cardiology Division, Department of Medicine, Li Ka Shing Faculty of Medicine, The University of Hong Kong, Hong Kong, China.
Tse YL; Cell Inspire Therapeutics Co., Ltd and Cell Inspire Biotechnology Co., Ltd, Shenzhen 518102, China.
Ng KM; Cell Inspire Therapeutics Co., Ltd and Cell Inspire Biotechnology Co., Ltd, Shenzhen 518102, China.
Esteban MA; Cell Inspire Therapeutics Co., Ltd and Cell Inspire Biotechnology Co., Ltd, Shenzhen 518102, China.
Tse HF; Cell Inspire Therapeutics Co., Ltd and Cell Inspire Biotechnology Co., Ltd, Shenzhen 518102, China.

JHEP Rep ; 4(1): 100389, 2022 Jan.

Article in En | MEDLINE | ID: mdl-34877514

Key words

AFP, alpha-fetoprotein; ALB, albumin; ATP7B, ATPase copper transporting beta; ATPase copper transporting beta polypeptide (ATP7B); Clustered regularly interspaced palindromic repeats (CRISPR)/Cas9; EB, embryoid body; RFLP, restriction fragment length polymorphism; Single-stranded Oligodeoxynucleotide (ssODN); TGN, trans-Golgi network; WD, Wilson's disease; Wilson's disease; cell therapy; gene correction; iHep(s), iPSC-derived hepatocyte(s); iPSC, induced pluripotent stem cell; iPSC-derived hepatocytes (iHeps); induced pluripotent stem cell (iPSC); sgRNA, single guide RNA; ssODN, single-stranded oligodeoxynucleotide

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Full text: 1 Collection: 01-internacional Database: MEDLINE Type of study: Prognostic_studies Language: En Journal: JHEP Rep Year: 2022 Document type: Article Affiliation country: China

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