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

Authors
HYE WON JANGDong Hyun JoChang Sik ChoJEONG HONG SHINJUNGHWA SEOGOO SANG YUGOPALAPPA RAMUDaesik KimSUNG RAE CHOJeong Hun KimHYONGBUM KIM
Issue Date
Feb-2022
Publisher
Springer Nature
Keywords
Prime editing; genetic disease; gene editing; hereditary tyrosinemia; Leber congenital amaurosis
Citation
Nature biomedical engineering, v.6, no.2, pp.181 - 194
Journal Title
Nature biomedical engineering
Volume
6
Number
2
Start Page
181
End Page
194
URI
https://yscholarhub.yonsei.ac.kr/handle/2021.sw.yonsei/6285
DOI
10.1038/s41551-021-00788-9
ISSN
2157-846X
Abstract
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.
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College of Medicine > 의과대학 재활의학교실 > 1. Journal Articles
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