Targeted Delivery of mRNA with Polymer-Lipid Nanoparticles for In Vivo Base Editing.

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Tác giả: Yan Chang, Jia Chen, Jiabei Chen, Qimingxing Chen, Yan Ding, Xiaoyan He, Jianfeng Li, Ran Luo, Runyuan Wang, Huiying Yang, Jialu Yuan, Guisheng Zhong

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: United States : ACS nano , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 184970

Messenger RNA (mRNA) encoding base editors, along with single guide RNAs (sgRNAs), have emerged as a promising therapeutic approach for various disorders. However, there is still insufficient exploration in achieving targeted and efficient delivery of mRNA and sgRNA to multiple organs while ensuring high biocompatibility and stability in vivo. To address this challenge, we synthesized a library of 108 poly(β-amino) esters (PBAEs) by incorporating 100% hydrophobic side chains and end-caps with varying amines. These PBAEs were further formulated with other excipients, including helper lipids, cholesterol, and PEGylated lipids, to form polymer-lipid nanoparticles (PLNPs). Structure-function analysis revealed that eLog
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