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In vivo correction of genetic disease in adult mice

Nature Reviews Genetics volume 15, page 291 (2014)Cite this article

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The CRISPR–Cas9 gene editing system can introduce or correct specific mutations in various species. Yin et al. used a mouse model of hereditary tyrosinemia type 1, which is a liver disease caused by a homozygous mutation in the fumarylacetoacetate hydrolase (Fah) gene. By hydrodynamically injecting Fah-targeted CRISPR–Cas9 reagents into diseased adult Fahmut/mut mice, they were able to correct the genetic defect and achieve wild-type FAH expression in ∼1 in 250 hepatocytes. The selective advantage of Fah-corrected hepatocytes led to their expansion in the liver during the following 30 days to constitute ∼33% of liver cells and alleviate pathological symptoms.

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References

  1. 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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  1. Darren J. Burgess
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Burgess, D. In vivo correction of genetic disease in adult mice. Nat Rev Genet 15, 291 (2014). https://doi.org/10.1038/nrg3731

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