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In vivo selective gene knockout through in situ delivery of RNA-guided Cas9 nuclease using lipidoid

Title
In vivo selective gene knockout through in situ delivery of RNA-guided Cas9 nuclease using lipidoid
Author
송명재
Advisor(s)
김형범
Issue Date
2014-02
Publisher
한양대학교
Degree
Master
Abstract
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.
URI
https://repository.hanyang.ac.kr/handle/20.500.11754/131031http://hanyang.dcollection.net/common/orgView/200000423331
Appears in Collections:
GRADUATE SCHOOL OF BIOMEDICAL SCIENCE AND ENGINEERING[S](의생명공학전문대학원) > BIOMEDICAL SCIENCE(의생명과학과) > Theses (Master)
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