Development and application of CRISPR/Cas9 technologies in genomic editing

Abstract Genomic editing to correct disease-causing mutations is a promising approach for the treatment of human diseases. As a simple and programmable nuclease-based genomic editing tool, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) syste...

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Veröffentlicht in:Human molecular genetics 2018-08, Vol.27 (R2), p.R79-R88
Hauptverfasser: Zhang, Cui, Quan, Renfu, Wang, Jinfu
Format: Artikel
Sprache:eng
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Zusammenfassung:Abstract Genomic editing to correct disease-causing mutations is a promising approach for the treatment of human diseases. As a simple and programmable nuclease-based genomic editing tool, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has substantially improved the ability to make precise changes in the human genome. Rapid development of CRISPR-based technologies in recent years has expanded its application scope and promoted CRISPR-based therapies in preclinical trails. Here, we review the application of the CRISPR system over the last 2 years; including its development and application in base editing, transcription modulation and epigenetic editing, genomic-scale screening, and cell and embryo therapy. Finally, the prospects and challenges related to application of CRISPR/Cas9 technologies are discussed.
ISSN:0964-6906
1460-2083
DOI:10.1093/hmg/ddy120