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2015 ; 4
(11
): e264
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Off-target Effects in CRISPR/Cas9-mediated Genome Engineering
#MMPMID26575098
Zhang XH
; Tee LY
; Wang XG
; Huang QS
; Yang SH
Mol Ther Nucleic Acids
2015[Nov]; 4
(11
): e264
PMID26575098
show ga
CRISPR/Cas9 is a versatile genome-editing technology that is widely used for
studying the functionality of genetic elements, creating genetically modified
organisms as well as preclinical research of genetic disorders. However, the high
frequency of off-target activity (?50%)-RGEN (RNA-guided endonuclease)-induced
mutations at sites other than the intended on-target site-is one major concern,
especially for therapeutic and clinical applications. Here, we review the basic
mechanisms underlying off-target cutting in the CRISPR/Cas9 system, methods for
detecting off-target mutations, and strategies for minimizing off-target
cleavage. The improvement off-target specificity in the CRISPR/Cas9 system will
provide solid genotype-phenotype correlations, and thus enable faithful
interpretation of genome-editing data, which will certainly facilitate the basic
and clinical application of this technology.