Journal
SCIENCE CHINA-LIFE SCIENCES
Volume 60, Issue 5, Pages 447-457Publisher
SCIENCE PRESS
DOI: 10.1007/s11427-017-9032-4
Keywords
CRISPR/Cas9; genome editing; genetic disease; gene therapy
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Funding
- National Natural Science Foundation [NSFC81502677, NSFC81602699, NSFC81123003]
- National Key Research and Development Program of China [2016YFA0201402]
- Key Technologies R & D program of Sichuan Province [2015FZ0040]
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The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) protein 9 system (CRISPR/Cas9) provides a powerful tool for targeted genetic editing. Directed by programmable sequence-specific RNAs, this system introduces cleavage and double-stranded breaks at target sites precisely. Compared to previously developed targeted nucleases, the CRISPR/Cas9 system demonstrates several promising advantages, including simplicity, high specificity, and efficiency. Several broad genome-editing studies with the CRISPR/Cas9 system in different species in vivo and ex vivo have indicated its strong potential, raising hopes for therapeutic genome editing in clinical settings. Taking advantage of non-homologous end-joining (NHEJ) and homology directed repair (HDR)-mediated DNA repair, several studies have recently reported the use of CRISPR/Cas9 to successfully correct disease-causing alleles ranging from single base mutations to large insertions. In this review, we summarize and discuss recent preclinical studies involving the CRISPR/Cas9-mediated correction of human genetic diseases.
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