| First Author | Wu Y | Year | 2013 |
| Journal | Cell Stem Cell | Volume | 13 |
| Issue | 6 | Pages | 659-62 |
| PubMed ID | 24315440 | Mgi Jnum | J:204144 |
| Mgi Id | MGI:5529718 | Doi | 10.1016/j.stem.2013.10.016 |
| Citation | Wu Y, et al. (2013) Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9. Cell Stem Cell 13(6):659-62 |
| abstractText | 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. |
