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Article

Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells.

Nature communications - 26 Nov 2014

Yang Luhan, Grishin Dennis, Wang Gang, Aach John, Zhang Cheng-Zhong, Chari Raj, Homsy Jason, Cai Xuyu, Zhao Yue, Fan Jian-Bing, Seidman Christine, Seidman Jonathan, Pu William, Church George

Abstract excerpt

CRISPR/Cas9 has demonstrated a high-efficiency in site-specific gene targeting. However, potential off-target effects of the Cas9 nuclease represent a major safety concern for any therapeutic application. Here, we knock out the Tafazzin gene by CRISPR/Cas9 in human-induced pluripotent stem cells with 54% efficiency. We combine whole-genome sequencing and deep-targeted sequencing to characterise the off-target...

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Identifiers

PMID: 25425480
DOI: 10.1038/ncomms6507
PMCID: PMC4352754

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Source receipt: pubmed, retrieved 7 Sept 2026.

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