Restoration of FVIII expression by targeted gene insertion in the FVIII locus in hemophilia A patient-derived iPSCs

Target-specific genome editing, using engineered nucleases zinc finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and type II clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9), is considered a promising approach to corr...

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Udgivet i:Exp Mol Med
Main Authors: Sung, Jin Jea, Park, Chul-Yong, Leem, Joong Woo, Cho, Myung Soo, Kim, Dong-Wook
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2019
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6470126/
https://ncbi.nlm.nih.gov/pubmed/30996250
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s12276-019-0243-1
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