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A quaternary ammonium-based nanosystem enables delivery of CRISPR/Cas9 for cancer therapy

Biomater Sci. 2024-02; 
Mengzhu Zhang, Siyu Sun, Xiao Liang, Zengguang Liu, Jiaxin Yin, Quanshun Li, Shengcai Yang
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摘要

Genome editing mediated by CRISPR/Cas9 is an attractive weapon for cancer therapy. However, delivery of CRISPR/Cas9 components to achieve therapeutic efficiency is still challenging. Herein, a quaternary ammonium-functionalized poly(L-lysine) and a cholesterol-modified PEG (QNP) were self-assembled with a negatively charged CRISPR Cas9/sgRNA ribonucleoprotein (RNP) to form a ternary complex (QNP/RNP). Such a delivery system of QNP exhibited multiplex genome editing capabilities (, the gene and the gene). In addition, QNP/RNP containing PLK1 sgRNA led to 30.99% of genome editing efficiency in MCF-7 cells with negligible cytotoxicity of the carrier. QNP/RNP, which was capable of simultaneously inhibiting cell... More

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