Cited 6 times in
In vivo gene correction with targeted sequence substitution through microhomology-mediated end joining
DC Field | Value | Language |
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dc.contributor.author | 김형범 | - |
dc.contributor.author | 이준원 | - |
dc.date.accessioned | 2018-11-16T16:57:24Z | - |
dc.date.available | 2018-11-16T16:57:24Z | - |
dc.date.issued | 2018 | - |
dc.identifier.issn | 0006-291X | - |
dc.identifier.uri | https://ir.ymlib.yonsei.ac.kr/handle/22282913/165509 | - |
dc.description.abstract | Genome editing technology using programmable nucleases has rapidly evolved in recent years. The primary mechanism to achieve precise integration of a transgene is mainly based on homology-directed repair (HDR). However, an HDR-based genome-editing approach is less efficient than non-homologous end-joining (NHEJ). Recently, a microhomology-mediated end-joining (MMEJ)-based transgene integration approach was developed, showing feasibility both in vitro and in vivo. We expanded this method to achieve targeted sequence substitution (TSS) of mutated sequences with normal sequences using double-guide RNAs (gRNAs), and a donor template flanking the microhomologies and target sequence of the gRNAs in vitro and in vivo. Our method could realize more efficient sequence substitution than the HDR-based method in vitro using a reporter cell line, and led to the survival of a hereditary tyrosinemia mouse model in vivo. The proposed MMEJ-based TSS approach could provide a novel therapeutic strategy, in addition to HDR, to achieve gene correction from a mutated sequence to a normal sequence. | - |
dc.description.statementOfResponsibility | restriction | - |
dc.language | English | - |
dc.publisher | Elsevier | - |
dc.relation.isPartOf | BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS | - |
dc.rights | CC BY-NC-ND 2.0 KR | - |
dc.rights | https://creativecommons.org/licenses/by-nc-nd/2.0/kr/ | - |
dc.subject.MESH | Animals | - |
dc.subject.MESH | DNA End-Joining Repair* | - |
dc.subject.MESH | Disease Models, Animal | - |
dc.subject.MESH | Genetic Therapy/methods* | - |
dc.subject.MESH | HEK293 Cells | - |
dc.subject.MESH | Humans | - |
dc.subject.MESH | Hydrolases/genetics* | - |
dc.subject.MESH | Mice | - |
dc.subject.MESH | Mutation | - |
dc.subject.MESH | RNA, Guide/genetics | - |
dc.subject.MESH | RNA, Messenger/genetics | - |
dc.subject.MESH | Tyrosinemias/genetics* | - |
dc.subject.MESH | Tyrosinemias/pathology | - |
dc.subject.MESH | Tyrosinemias/therapy* | - |
dc.title | In vivo gene correction with targeted sequence substitution through microhomology-mediated end joining | - |
dc.type | Article | - |
dc.contributor.college | College of Medicine (의과대학) | - |
dc.contributor.department | Dept. of Pharmacology (약리학교실) | - |
dc.contributor.googleauthor | Jeong Hong Shin | - |
dc.contributor.googleauthor | Soobin Jung | - |
dc.contributor.googleauthor | Suresh Ramakrishna | - |
dc.contributor.googleauthor | Hyongbum Henry Kim | - |
dc.contributor.googleauthor | Junwon Lee | - |
dc.identifier.doi | 10.1016/j.bbrc.2018.05.130 | - |
dc.contributor.localId | A01148 | - |
dc.contributor.localId | A03179 | - |
dc.relation.journalcode | J00281 | - |
dc.identifier.eissn | 1090-2104 | - |
dc.identifier.pmid | 29787760 | - |
dc.identifier.url | https://www.sciencedirect.com/science/article/pii/S0006291X18311999 | - |
dc.subject.keyword | Hereditary tyrosinemia | - |
dc.subject.keyword | Homology-directed repair (HDR) | - |
dc.subject.keyword | In vivo gene correction | - |
dc.subject.keyword | Microhomology-mediated end-joining (MMEJ) | - |
dc.subject.keyword | Targeted sequence substitution (TSS) | - |
dc.contributor.alternativeName | Kim, Hyongbum | - |
dc.contributor.alternativeName | Lee, Jun Won | - |
dc.contributor.affiliatedAuthor | 김형범 | - |
dc.contributor.affiliatedAuthor | 이준원 | - |
dc.citation.volume | 502 | - |
dc.citation.number | 1 | - |
dc.citation.startPage | 116 | - |
dc.citation.endPage | 122 | - |
dc.identifier.bibliographicCitation | BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, Vol.502(1) : 116-122, 2018 | - |
dc.identifier.rimsid | 59120 | - |
dc.type.rims | ART | - |
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