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Correction of a genetic disease in mouse via use of CRISPR-Cas9.

Abstract：

: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.

journal_name

Cell Stem Cell

journal_title

Cell stem cell

authors

Wu Y,Liang D,Wang Y,Bai M,Tang W,Bao S,Yan Z,Li D,Li J

doi

10.1016/j.stem.2013.10.016

subject

Has Abstract

pub_date

2013-12-05 00:00:00

pages

659-62

issue

6

eissn

1934-5909

issn

1875-9777

pii

S1934-5909(13)00462-1

journal_volume

13

pub_type

杂志文章

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