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Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR–Cas platform

Yoshimi, K., Kaneko, T., Voigt, B., Mashimo, T.·2014·Nature Communications
DOI 10.1038/ncomms5240PMC PMC4083438
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Abstract

The bacterial CRISPR/Cas system has proven to be an efficient gene-targeting tool in various organisms. Here we employ CRISPR/Cas for accurate and efficient genome editing in rats. The synthetic chimeric guide RNAs (gRNAs) discriminate a single-nucleotide polymorphism (SNP) difference in rat embryonic fibroblasts, allowing allele-specific genome editing of the dominant phenotype in (F344 × DA)F1 hybrid embryos. Interestingly, the targeted allele, initially assessed by the allele-specific gRNA, is repaired by an interallelic gene conversion between homologous chromosomes. Using single-stranded oligodeoxynucleotides, we recover three recessive phenotypes: the albino phenotype by SNP exchange; the non-agouti phenotype by integration of a 19-bp DNA fragment; and the hooded phenotype by eliminating a 7,098-bp insertional DNA fragment, evolutionary-derived from an endogenous retrovirus. Successful in vivo application of the CRISPR/Cas system confirms its importance as a genetic engineering tool for creating animal models of human diseases and its potential use in gene therapy.

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  • Studies using gene-editing in animals such as rats, cattle, sheep and pigs indicate that it is possible to delete or disable genes in an embryo-a simpler process than actually correcting DNA sequences-in only some of the cells.

    “Studies using gene-editing in animals such as rats 5 , cattle 6 , sheep 7 and pigs 8 , indicate that it is possible to delete or disable genes in an embryo - a simpler process than actually correcting DNA sequences - in only some of the cells.”

    From Don’t edit the human germ line (2015) · cites this paper as [5]

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Authors
Yoshimi, K., Kaneko, T., Voigt, B., & Mashimo, T.
Venue
Nature Communications
Publisher
Springer Science and Business Media LLC
Published
2014-06-26
Volume
5
Issue
1
ISSN
2041-1723
DOI
10.1038/ncomms5240
PMC
PMC4083438
Cited by
163
References
12
Language
en
Licence

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