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Programmable Nuclease-Based Integration into Novel Extragenic Genomic Safe Harbor Identified from Korean Population-Based CNV Analysis

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dc.contributor.authorEun-Seo Lee-
dc.contributor.authorSanghoon Moon-
dc.contributor.authorKwaku Dad Abu-Bonsrah-
dc.contributor.authorYun Kyoung Kim-
dc.contributor.authorMi Yeong Hwang-
dc.contributor.authorYoung Jin Kim-
dc.contributor.authorSeokjoong Kim-
dc.contributor.authorNathaniel S. Hwang-
dc.contributor.authorHyongbum Henry Kim-
dc.contributor.authorBong-Jo Kim-
dc.date.available2019-11-28T06:13:39Z-
dc.date.created2019-09-24-
dc.date.issued2019-09-
dc.identifier.issn2372-7705-
dc.identifier.urihttps://pr.ibs.re.kr/handle/8788114/6580-
dc.description.abstract© 2019 The AuthorsHere, we found two genomic safe harbor (GSH) candidates from chromosomes 3 and 8, based on large-scale population-based cohort data from 4,694 Koreans by CNV analysis. Furthermore, estimated genotype of these CNVRs was validated by quantitative real-time PCR, and epidemiological data examined no significant genetic association between diseases or traits and two CNVRs. After screening the GSH candidates by in silico approaches, we designed TALEN pairs to integrate EGFP expression cassette into human cell lines in order to confirm the functionality of GSH candidates in an in vitro setting. As a result, transgene insertion into one of the two loci using TALEN showed robust transgene expression comparable to that with an AAVS1 site without significantly perturbing neighboring genes. Changing the promoter or cell type did not noticeably disturb this trend. Thus, we could validate two CNVRs as a site for effective and safe transgene insertion in human cells-
dc.language영어-
dc.publisherCELL PRESS-
dc.subjectcopy number variation region-
dc.subjectgenome editing-
dc.subjectsafe harbor-
dc.subjectTALEN-
dc.titleProgrammable Nuclease-Based Integration into Novel Extragenic Genomic Safe Harbor Identified from Korean Population-Based CNV Analysis-
dc.typeArticle-
dc.type.rimsART-
dc.identifier.wosid000488089600024-
dc.identifier.scopusid2-s2.0-85070711832-
dc.identifier.rimsid69581-
dc.contributor.affiliatedAuthorHyongbum Henry Kim-
dc.identifier.doi10.1016/j.omto.2019.07.001-
dc.identifier.bibliographicCitationMOLECULAR THERAPY-ONCOLYTICS, v.14, pp.253 - 265-
dc.relation.isPartOfMOLECULAR THERAPY-ONCOLYTICS-
dc.citation.titleMOLECULAR THERAPY-ONCOLYTICS-
dc.citation.volume14-
dc.citation.startPage253-
dc.citation.endPage265-
dc.description.journalClass1-
dc.description.journalClass1-
dc.description.journalRegisteredClassscie-
dc.description.journalRegisteredClassscopus-
dc.relation.journalWebOfScienceCategoryOncology-
dc.relation.journalWebOfScienceCategoryMedicine, Research & Experimental-
dc.subject.keywordPlusCOPY NUMBER VARIATION-
dc.subject.keywordPlusGENE-THERAPY-
dc.subject.keywordPlusSTEM-CELLS-
dc.subject.keywordPlusEXPRESSION-
dc.subject.keywordAuthorcopy number variation region-
dc.subject.keywordAuthorgenome editing-
dc.subject.keywordAuthorsafe harbor-
dc.subject.keywordAuthorTALEN-
Appears in Collections:
Center for Nanomedicine (나노의학 연구단) > 1. Journal Papers (저널논문)
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