Journal
CELL STEM CELL
Volume 13, Issue 6, Pages 659-662Publisher
CELL PRESS
DOI: 10.1016/j.stem.2013.10.016
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Funding
- Ministry of Science and Technology of China [2014CB964803]
- National Natural Science Foundation of China [31225017, 91319310]
- Chinese Academy of Sciences [XDA01010403]
- Shanghai Municipal Commission for Science and Technology [12JC1409600, 13XD1404000]
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The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant allele. Correction occurred via homology-directed repair (HDR) based on an exogenously supplied oligonucleotide or the endogenous WT allele, with only rare evidence of off-target modifications. The resulting mice were fertile and able to transmit the corrected allele to their progeny. Thus, our study provides proof of principle for use of the CRISPR-Cas9 system to correct genetic disease.
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