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In vivo selective gene knockout through in situ delivery of RNA-guided Cas9 nuclease using lipidoid / 송명재
15 유전학 조회 수 123 추천 수 0 2017.02.14 14:10:22| 발행년 : | 2014 |
|---|---|
| 구분 : | 학위논문 |
| 학술지명 : | |
| 관련링크 : | http://www.riss.kr/link?id=T13381638 |
In vivo selective gene knockout through in situ delivery of RNA-guided Cas9 nuclease using lipidoid
= Lipidoid를 이용한 RNA-유도형 유전자 가위의 생체 내 전달에 의한 선택적인 유전자 녹아웃
- 저자
송명재
- 형태사항
iv, 29 p. : 삽도 ; 26 cm.
- 일반주기
지도교수: 김형범
권두에 Abstract, 권말에 국문요지 수록
참고문헌: p. 22-25 - 학위논문사항
학위논문(석사)-- 한양대학교 의생명공학전문대학원 : 의생명과학과 2014. 2
- 발행국
서울
- 언어
영어
- 출판년
2014
- Efficient methods for creating targeted genetic modifications have long been sought for the investigation of gene function and the development of therapeutic modalities for various diseases, including genetic disorders. Although such modifications are possible using custom-designed artificial nucleases are currently in use ie. Zinc finger nucleases (ZFNs), TAL effector nucleases (TALEN) and CRISPR/Cas9-based RNA guided RNA endonucleases (RGEN). Among them, RGEN based genome editing was found to be more efficient and easy to clone. RNA-guided endonucleases (RGENs), which are based on the clustered, regularly interspaced, short palindromic repeat (CRISPR)-CRISPR-associated (Cas) system, have recently emerged as a simple and efficient tool for genome editing. However, in vitro genome editing using CRISPR system is successfully achieved in mouse, drosophila, c eligans and human cells, it’s in vivo usage in multi-cellular organism is not yet been
iv
tested. In this study, we delivered RGEN plasmid DNA that express eGFP targeting guided RNA to eGFP expressing mouse by intradermal injection using Lipidoid. Our results showed significant decrease in eGFP signals at the site of injection indicating efficient silencing of eGFP gene in vivo. Thus, the RGEN system can be used to knock out specific genes at specified site in vivo, providing a potential tool for in vivo gene therapy using CRISPR.
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