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

Authors
 Hyewon Jang  ;  Dong Hyun Jo  ;  hang Sik Cho  ;  Jeong Hong Shin  ;  Jung Hwa Seo  ;  Goosang Yu  ;  Ramu Gopalappa  ;  Daesik Kim  ;  Sung-Rae Cho  ;  Jeong Hun Kim  ;  Hyongbum Henry Kim 
Citation
 NATURE BIOMEDICAL ENGINEERING, Vol.6(2) : 181-194, 2022-02 
Journal Title
NATURE BIOMEDICAL ENGINEERING
Issue Date
2022-02
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.
Full Text
https://www.nature.com/articles/s41551-021-00788-9
DOI
10.1038/s41551-021-00788-9
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pharmacology (약리학교실) > 1. Journal Papers
1. College of Medicine (의과대학) > Dept. of Rehabilitation Medicine (재활의학교실) > 1. Journal Papers
Yonsei Authors
Kim, Hyongbum(김형범) ORCID logo https://orcid.org/0000-0002-4693-738X
Gopalappa, Ramu(라무고파라파) ORCID logo https://orcid.org/0000-0001-5574-4371
Seo, Jung Hwa(서정화)
Cho, Sung-Rae(조성래) ORCID logo https://orcid.org/0000-0003-1429-2684
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/188078
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