CRISPR/Cas Derivatives as Novel Gene Modulating Tools: Possibilities and In Vivo Applications

2020-04-25 | journal article; research paper. A publication with affiliation to the University of Göttingen.

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​CRISPR/Cas Derivatives as Novel Gene Modulating Tools: Possibilities and In Vivo Applications​
Xu, X.; Hulshoff, M. S.; Tan, X.; Zeisberg, M.   & Zeisberg, E. M. ​ (2020) 
International Journal of Molecular Sciences21(9) pp. 3038​.​ DOI: https://doi.org/10.3390/ijms21093038 

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Authors
Xu, Xingbo; Hulshoff, Melanie S.; Tan, Xiaoying; Zeisberg, Michael ; Zeisberg, Elisabeth M. 
Abstract
The field of genome editing started with the discovery of meganucleases (e.g., the LAGLIDADG family of homing endonucleases) in yeast. After the discovery of transcription activator-like effector nucleases and zinc finger nucleases, the recently discovered clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR associated proteins (Cas) system has opened a new window of applications in the field of gene editing. Here, we review different Cas proteins and their corresponding features including advantages and disadvantages, and we provide an overview of the different endonuclease-deficient Cas protein (dCas) derivatives. These dCas derivatives consist of an endonuclease-deficient Cas9 which can be fused to different effector domains to perform distinct in vitro applications such as tracking, transcriptional activation and repression, as well as base editing. Finally, we review the in vivo applications of these dCas derivatives and discuss their potential to perform gene activation and repression in vivo, as well as their potential future use in human therapy.
Issue Date
25-April-2020
Journal
International Journal of Molecular Sciences 
ISSN
1422-0067
eISSN
1422-0067
Language
English

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