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Therapeutic application of the CRISPR system: current issues and new prospects

Authors
 Minyoung Lee  ;  Hyongbum Kim 
Citation
 Human Genetics, Vol.138(6) : 563-590, 2019 
Journal Title
 Human Genetics 
ISSN
 0340-6717 
Issue Date
2019
MeSH
• CRISPR-Cas Systems* ; Epigenomics/methods ; Gene Editing/methods* ; Gene Rearrangement ; Gene Transfer Techniques* ; Genetic Therapy/methods ; Humans ; Mutagenesis* ; Reproducibility of Results
Abstract
Since its discovery, the Clustered Regularly Interspaced Short Palindromic Repeat (the CRISPR) system has been increasingly applied to therapeutic genome editing. Employment of several viral and non-viral vectors has enabled efficient delivery of the CRISPR system to target cells or tissues. In addition, the CRISPR system is able to modulate the target gene's expression in various ways, such as mutagenesis, gene integration, epigenome regulation, chromosomal rearrangement, base editing and mRNA editing. However, there are still limitations hindering an ideal application of the system: inefficient delivery, dysregulation of the delivered gene, the immune response against the CRISPR system, the off-target effects or the unintended on-target mutations. In addition, there are recent discoveries that have not been yet applied to CRISPR-mediated therapeutic genome editing. Here, we review the overall principles related to the therapeutic application of the CRISPR system, along with new strategies for the further application and prospects to overcome the limitations.
Full Text
https://link.springer.com/article/10.1007%2Fs00439-019-02028-2
DOI
10.1007/s00439-019-02028-2
Appears in Collections:
1. College of Medicine (의과대학) > Dept. of Pharmacology (약리학교실) > 1. Journal Papers
Yonsei Authors
Kim, Hyongbum(김형범) ORCID logo https://orcid.org/0000-0002-4693-738X
URI
https://ir.ymlib.yonsei.ac.kr/handle/22282913/171305
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