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나노의학 연구단
나노의학 연구단
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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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Title
Programmable Nuclease-Based Integration into Novel Extragenic Genomic Safe Harbor Identified from Korean Population-Based CNV Analysis
Author(s)
Eun-Seo Lee; Sanghoon Moon; Kwaku Dad Abu-Bonsrah; Yun Kyoung Kim; Mi Yeong Hwang; Young Jin Kim; Seokjoong Kim; Nathaniel S. Hwang; Hyongbum Henry Kim; Bong-Jo Kim
Publication Date
2019-09
Journal
MOLECULAR THERAPY-ONCOLYTICS, v.14, no., pp.253 - 265
Publisher
CELL PRESS
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
URI
https://pr.ibs.re.kr/handle/8788114/6580
ISSN
2372-7705
Appears in Collections:
Center for Nanomedicine (나노의학 연구단) > Journal Papers
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