| 게재년월 |
2019/07 |
| 논문제목 |
Programmable Nuclease-Based Integration into Novel Extragenic Genomic Safe Harbor Identified from Korean Population-Based CNV Analysis.
|
| 참여교수 |
김형범
|
| 학술지명 |
Mol Ther Oncolytics. 2019 Jul 23;14:253-265 |
| 초록 |
Here, 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. |
| 게재정보 |
Mol Ther Oncolytics. 2019 Jul 23;14:253-265 |
| 총저자명 |
Lee ES, Moon S, Abu-Bonsrah KD, Kim YK, Hwang MY, Kim YJ, Kim S, Hwang NS, Kim HH, Kim BJ |
