Back to Search Start Over

Short cell-penetration peptide conjugated bioreducible polymer enhances gene editing of CRISPR system.

Authors :
Wang, Xiaobo
Cai, Chengyuan
Lv, Weiqi
Chen, Kechen
Li, Jiaxin
Liao, Kaitong
Zhang, Yanqun
Huang, Hongxin
Lin, Ying
Rong, Zhili
Duan, Xiaopin
Source :
Journal of Nanobiotechnology. 5/24/2024, Vol. 22 Issue 1, p1-15. 15p.
Publication Year :
2024

Abstract

CRISPR-based gene therapy offers precise targeting and specific editing of disease-related gene sequences, potentially yielding long-lasting treatment effects. However, efficient delivery remains a significant challenge for its widespread application. In this study, we design a novel short peptide-conjugated bioreducible polymer named TSPscp as a safe and effective delivery vector for the CRISPR system. Our results show that TSPscp markedly boosts transcriptional activation and genome editing activities of multiple CRISPR systems as confirmed by decomposition-seq and Deep-seq, which is resulted from its capability in facilitating delivery of plasmid DNA by promoting cellular uptake and lysosomal escape. Additionally, TSPscp further enhances genome editing of CRISPR by delivery of minicircle DNA, a condensed form of regular plasmid DNA. More importantly, TSPscp significantly improves delivery and genome editing of CRISPR system in vivo. In summary, our study highlights TSPscp as a promising delivery tool for CRISPR applications in vivo. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
14773155
Volume :
22
Issue :
1
Database :
Academic Search Index
Journal :
Journal of Nanobiotechnology
Publication Type :
Academic Journal
Accession number :
177462099
Full Text :
https://doi.org/10.1186/s12951-024-02554-w