Bioorthogonal Engineered Virus-Like Nanoparticles for Efficient Gene Therapy

IF 31.6 1区材料科学 Q1 MATERIALS SCIENCE, MULTIDISCIPLINARY

Nano-Micro Letters Pub Date : 2023-08-12 DOI:10.1007/s40820-023-01153-y

Chun-Jie Bao, Jia-Lun Duan, Ying Xie, Xin-Ping Feng, Wei Cui, Song-Yue Chen, Pei-Shan Li, Yi-Xuan Liu, Jin-Ling Wang, Gui-Ling Wang, Wan-Liang Lu

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Abstract

AbstractSection Highlights

A virus-like nanoparticle (reBiosome) was developed via site-specific codon mutation for displaying unnatural amino acid (Azi) on virus envelope protein at a rational site, followed by conjugating weak acid-responsive polyethylene glycol polymer on Azi via bioorthogonal chemistry.
The reBiosome exhibited reduced virus-like immunogenicity, prolonged blood circulation and enhanced delivery to weakly acidic disease foci.
The reBiosome enabled efficient delivery of gene editing and gene silencing system, demonstrating remarkable therapeutic efficacy in breast cancer and arthritis, respectively.

Abstract Image

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用于有效基因治疗的生物正交工程病毒样纳米颗粒。

基因疗法为人类主要疾病提供了潜在的变革性策略。然而，基因治疗的关键挑战之一是开发一种有效的策略，将基因传递到特定的组织中。在这里，我们报道了一种新的病毒样纳米颗粒，生物正交工程病毒样重组生物体(reBiosome)，用于癌症和炎症性疾病的有效基因治疗。突变型病毒样生物小体(mBiosome)首先通过位点特异性密码子突变，将4-叠氮多-l -苯丙氨酸显示在水泡性口炎病毒糖蛋白上，然后通过生物正交化学将弱酸反应亲水性聚合物点击到mBiosome上，制备出突变型病毒样生物小体。结果表明，reBiosome具有降低病毒样免疫原性、延长血液循环时间和提高基因传递到弱酸性病灶(如肿瘤和关节炎组织)的效率。此外，reBiosome分别通过提供基因编辑和沉默系统，在乳腺癌和关节炎中显示出强大的治疗效果。总之，本研究为癌症和炎症性疾病的基因治疗提供了一个通用、安全、有效的平台。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Nano-Micro Letters NANOSCIENCE & NANOTECHNOLOGY-MATERIALS SCIENCE, MULTIDISCIPLINARY

CiteScore

42.40

自引率

4.90%

发文量

715

审稿时长

13 weeks

期刊介绍： Nano-Micro Letters is a peer-reviewed, international, interdisciplinary and open-access journal that focus on science, experiments, engineering, technologies and applications of nano- or microscale structure and system in physics, chemistry, biology, material science, pharmacy and their expanding interfaces with at least one dimension ranging from a few sub-nanometers to a few hundreds of micrometers. Especially, emphasize the bottom-up approach in the length scale from nano to micro since the key for nanotechnology to reach industrial applications is to assemble, to modify, and to control nanostructure in micro scale. The aim is to provide a publishing platform crossing the boundaries, from nano to micro, and from science to technologies.