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Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases

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Title
Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases
Author(s)
Hyewon Jang; Dong Hyun Jo; Chang Sik Cho; Jeong Hong Shin; Jung Hwa Seo; Goosang Yu; Ramu Gopalappa; Daesik Kim; Sung-Rae Cho; Jeong Hun Kim; Hyongbum Henry Kim
Publication Date
2022-02
Journal
Nature Biomedical Engineering, v.6, no.2, pp.181 - 194
Publisher
Nature Research
Abstract
© 2021, The Author(s), under exclusive licence to Springer Nature Limited.The use of prime editing—a gene-editing technique that induces small genetic changes without the need for donor DNA and without causing double strand breaks—to correct pathogenic mutations and phenotypes needs to be tested in animal models of human genetic diseases. Here we report the use of prime editors 2 and 3, delivered by hydrodynamic injection, in mice with the genetic liver disease hereditary tyrosinemia, and of prime editor 2, delivered by an adeno-associated virus vector, in mice with the genetic eye disease Leber congenital amaurosis. For each pathogenic mutation, we identified an optimal prime-editing guide RNA by using cells transduced with lentiviral libraries of guide-RNA-encoding sequences paired with the corresponding target sequences. The prime editors precisely corrected the disease-causing mutations and led to the amelioration of the disease phenotypes in the mice, without detectable off-target edits. Prime editing should be tested further in more animal models of genetic diseases.
URI
https://pr.ibs.re.kr/handle/8788114/11370
DOI
10.1038/s41551-021-00788-9
ISSN
2157-846X
Appears in Collections:
Center for Nanomedicine (나노의학 연구단) > 1. Journal Papers (저널논문)
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