The CRISPR (clustered regularly interspaced short palindromic repeats)-associated (Cas) system, which consists of the Cas9 nuclease and a single guide RNA sequence against a desired target, is emerging as a powerful genome editing tool. This paper describes the first use of the CRISPR–Cas9 system to mediate genome editing in adult mammals. Injection of the system in a mouse model corrected a splicing mutation in the fumarylacetoacetate hydrolase (Fah) gene, which causes hereditary tyrosinaemia type I. Wild-type FAH protein was detected in ~1 out of 250 liver cells, and the expansion of FAH-positive hepatocytes rescued the weight loss phenotype of mice.
References
Yin, H. et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nature Biotech. http://dx.doi.org/10.1038/nbt.2884 (2014)
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Harrison, C. CRISPR corrects a disease phenotype. Nat Rev Drug Discov 13, 418 (2014). https://doi.org/10.1038/nrd4347
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DOI: https://doi.org/10.1038/nrd4347
