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基于蛋白质的纳米颗粒用于治疗性核酸递送。

Protein-based nanoparticles for therapeutic nucleic acid delivery.

机构信息

Department of Surgery, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, 02215, USA; Harvard and MIT Division of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA; Harvard/MIT MD-PhD Program, Boston, MA, USA, 02115; Wyss Institute of Biologically Inspired Engineering, Harvard University, Boston, MA, 02115, USA.

Department of Surgery, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, 02215, USA; Harvard and MIT Division of Health Sciences and Technology, Massachusetts Institute of Technology, Cambridge, MA, 02139, USA; Harvard/MIT MD-PhD Program, Boston, MA, USA, 02115.

出版信息

Biomaterials. 2024 Mar;305:122464. doi: 10.1016/j.biomaterials.2023.122464. Epub 2024 Jan 2.

Abstract

To realize the full potential of emerging nucleic acid therapies, there is a need for effective delivery agents to transport cargo to cells of interest. Protein materials exhibit several unique properties, including biodegradability, biocompatibility, ease of functionalization via recombinant and chemical modifications, among other features, which establish a promising basis for therapeutic nucleic acid delivery systems. In this review, we highlight progress made in the use of non-viral protein-based nanoparticles for nucleic acid delivery in vitro and in vivo, while elaborating on key physicochemical properties that have enabled the use of these materials for nanoparticle formulation and drug delivery. To conclude, we comment on the prospects and unresolved challenges associated with the translation of protein-based nucleic acid delivery systems for therapeutic applications.

摘要

为了充分发挥新兴核酸疗法的潜力,需要有效的输送剂将货物输送到感兴趣的细胞中。蛋白质材料具有多种独特的特性,包括可生物降解性、生物相容性、易于通过重组和化学修饰进行功能化等,这为治疗性核酸输送系统奠定了有前途的基础。在这篇综述中,我们强调了在体外和体内使用基于非病毒蛋白质的纳米粒子进行核酸输送方面所取得的进展,同时详细阐述了使这些材料能够用于纳米粒子配方和药物输送的关键物理化学性质。最后,我们对与治疗应用中基于蛋白质的核酸输送系统的转化相关的前景和未解决的挑战进行了评论。

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