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| Type |
Poster Presentation |
| Area |
Medicinal Chemistry |
| Room No. |
Grand Ballroom |
| Time |
10월 18일 (목요일) 11:00~12:30 |
| Code |
MEDI.P-304 |
| Subject |
CRISPRgo: Ressurection of Non-sense mutated gene expression by ABE. |
| Authors |
Choongil Lee, Sangsu Bae1,*
department of chemistry, Seoul National University, Korea
1Department of Chemistry, Hanyang University, Korea
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| Abstract |
CRISPR-Cas9 systems are broadly applicable in genome engineering fields. In early stage of CRISPR-Cas9 system, the aim of using CRISPR-Cas9 focued on DNA cleavage and gene Knock-out. However, now we are facing new application of using CRISPR-Cas9 system, such as Cytidine Base-eitor(BE) and Adenine Base-editor(ABE). BE and ABE induces much less insertion or deletion than does Cas9. That mean we can achive intending genetic modification without the genomic damage that may inducing cell death or unexpected genetic mutations.
Moreover, ABE is a adenine base editro which is substitues A to G. Using the ABE, we can change the STOP codon (TAA, TAG, TGA) into the other amino acid (Trp, Gln and Arg). Therefore, ABE is able to restore the gene expressions which are stoped by non-sense mutation.
In this study, We demonstrated the capblity of ABE, restoring non-sense mutation, in EGFP expression prevented by whole stop codons. We compared three kinds of ABE (Cas9 ABE 7.10, xCas9 3.7-ABE3 7.10 and ABE-max) efficiency in EGFP-stop cell line by FACS and NGS results.
Futhermore non-sense mutation is critical in genetic diseas, therefore we did restore the gene expression in patient's fibroblast, such as XPC, GAA, and ENPP1genetic diseas.
Finally, ABE is an effiective system for restoring stoped gene expression without inducing the genetic damage by double strand DNA cleavages. |
| E-mail |
cilee777@snu.ac.kr |
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122nd General Meeting of the KCS