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Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases
DC Field | Value | Language |
---|---|---|
dc.contributor.author | Jang, Hyewon | - |
dc.contributor.author | Jo, Dong Hyun | - |
dc.contributor.author | Cho, Chang Sik | - |
dc.contributor.author | Shin, Jeong Hong | - |
dc.contributor.author | Seo, Jung Hwa | - |
dc.contributor.author | Yu, Goosang | - |
dc.contributor.author | Gopalappa, Ramu | - |
dc.contributor.author | Kim, Daesik | - |
dc.contributor.author | Cho, Sung-Rae | - |
dc.contributor.author | Kim, Jeong Hun | - |
dc.contributor.author | Kim, Hyongbum Henry | - |
dc.date.accessioned | 2023-04-25T07:30:01Z | - |
dc.date.available | 2023-04-25T07:30:01Z | - |
dc.date.created | 2022-03-15 | - |
dc.date.created | 2022-03-15 | - |
dc.date.created | 2022-03-15 | - |
dc.date.issued | 2022-02 | - |
dc.identifier.citation | Nature Biomedical Engineering, Vol.6 No.2, pp.181-194 | - |
dc.identifier.issn | 2157-846X | - |
dc.identifier.uri | https://hdl.handle.net/10371/191466 | - |
dc.description.abstract | In mouse models of hereditary tyrosinaemia and of Leber congenital amaurosis, prime editing can precisely correct the disease-causing mutations, ameliorating the disease phenotypes. 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. | - |
dc.language | 영어 | - |
dc.publisher | NATURE PUBLISHING GROUP | - |
dc.title | Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases | - |
dc.type | Article | - |
dc.identifier.doi | 10.1038/s41551-021-00788-9 | - |
dc.citation.journaltitle | Nature Biomedical Engineering | - |
dc.identifier.wosid | 000689523800001 | - |
dc.identifier.scopusid | 2-s2.0-85113750952 | - |
dc.citation.endpage | 194 | - |
dc.citation.number | 2 | - |
dc.citation.startpage | 181 | - |
dc.citation.volume | 6 | - |
dc.description.isOpenAccess | N | - |
dc.contributor.affiliatedAuthor | Jo, Dong Hyun | - |
dc.contributor.affiliatedAuthor | Kim, Jeong Hun | - |
dc.type.docType | Article | - |
dc.description.journalClass | 1 | - |
dc.subject.keywordPlus | ADENOASSOCIATED VIRUS VECTOR | - |
dc.subject.keywordPlus | GENE-THERAPY | - |
dc.subject.keywordPlus | MOUSE MODEL | - |
dc.subject.keywordPlus | VISION | - |
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