Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

详细信息    查看全文

• 作者：Yuxuan Wu ; Dan Liang ; Yinghua Wang ; Meizhu Bai ; Wei Tang ; Shiming Bao ; Zhiqiang Yan ; Dangsheng Li ; Jinsong Li
• 刊名：Cell Stem Cell
• 出版年：5 December, 2013
• 年：2013
• 卷：13
• 期：6
• 页码：659-662
• 全文大小：885 K

文摘

| Figures/TablesFigures/Tables | ReferencesReferences

Summary

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.