CRISPR/Cas9 Delivery Mediated with Hydroxyl‐Rich Nanosystems for Gene Editing in Aorta
Abstract: A CRISPR/Cas9 system has emerged as a powerful tool for gene editing to treat genetic mutation related diseases. Due to the complete endothelial barrier, effective delivery of the CRISPR/Cas9 system to vasculatures remains a challenge for in vivo gene editing of genetic vascular diseases especially in aorta. Herein, it is reported that CHO‐PGEA (cholesterol (CHO)‐terminated ethanolamine‐aminated poly (glycidyl methacrylate)) with rich hydroxyl groups can deliver a plasmid based pCas9‐sgFbn1 system for the knockout of exon 10 in Fbn1 gene. This is the first report of a polycation‐mediated CRISPR/Cas9 system for gene editing in aorta of adult mice. CHO‐PGEA/pCas9‐sgFbn1 nanosystems can effectively contribute to the knockout of exon 10 in Fbn1 in vascular smooth muscle cells in vitro, which leads to the change of the phosphorylation of Smad2/3 and the increased expression of two downstream signals of Fbn1: Mmp‐2 and Ctgf. For in vivo application, the aortic enrichment of CHO‐PGEA/Cas9‐sgFbn1 is achieved by administering a pressor dose of angiotensin II (Ang II). The effects of the pCas9‐sgFbn1 system targeting Fbn1 demonstrate an increase in the expression of Mmp‐2 and Ctgf in aorta. Thus, the combination of CHO‐PGEA/pCas9‐sgFbn1 nanosystems with Ang II infusion can provide the possibility for in vivo gene editing in aorta.
- Standort
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Deutsche Nationalbibliothek Frankfurt am Main
- Umfang
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Online-Ressource
- Sprache
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Englisch
- Erschienen in
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CRISPR/Cas9 Delivery Mediated with Hydroxyl‐Rich Nanosystems for Gene Editing in Aorta ; volume:6 ; number:12 ; year:2019 ; extent:12
Advanced science ; 6, Heft 12 (2019) (gesamt 12)
- Urheber
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Zhang, Xiaoping
Xu, Chen
Gao, Shijuan
Li, Ping
Kong, Yu
Li, Tiantian
Li, Yulin
Xu, Fu‐Jian
Du, Jie
- DOI
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10.1002/advs.201900386
- URN
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urn:nbn:de:101:1-2022071309415636154810
- Rechteinformation
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Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Letzte Aktualisierung
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15.08.2025, 07:22 MESZ
Datenpartner
Deutsche Nationalbibliothek. Bei Fragen zum Objekt wenden Sie sich bitte an den Datenpartner.
Beteiligte
- Zhang, Xiaoping
- Xu, Chen
- Gao, Shijuan
- Li, Ping
- Kong, Yu
- Li, Tiantian
- Li, Yulin
- Xu, Fu‐Jian
- Du, Jie
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