Proven and novel strategies for efficient editing of the human genome

Current Opinion in Pharmacology
Claudio MussolinoToni Cathomen

Abstract

Targeted gene editing with designer nucleases has become increasingly popular. The most commonly used designer nuclease platforms are engineered meganucleases, zinc-finger nucleases, transcription activator-like effector nucleases and the clustered regularly interspaced short palindromic repeat/Cas9 system. These powerful tools have greatly facilitated the generation of plant and animal models for basic research, and harbor an enormous potential for applications in biotechnology and gene therapy. This review recapitulates proven concepts of targeted genome engineering in primary human cells and elaborates on novel concepts that became possible with the dawn of RNA-guided nucleases and RNA-guided transcription factors.

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Jan 7, 2016·Trends in Genetics : TIG·Goran Kungulovski, Albert Jeltsch
Dec 15, 2015·Molecular Therapy : the Journal of the American Society of Gene Therapy·Maximilian MüllerClaudio Mussolino
Dec 10, 2015·Nature Biotechnology·Giorgia Santilli, Adrian J Thrasher
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Aug 16, 2017·Human Gene Therapy·Claudio MussolinoToni Cathomen
Jan 30, 2020·International Journal of Molecular Sciences·Yongxing FangClaudio Mussolino
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Jun 26, 2020·Molecular Biotechnology·Zhixi LiuHongtao Xiao
Aug 12, 2017·Current Opinion in Biotechnology·Joyce Van Eck

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