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用于非病毒基因治疗的脂质包被磷酸钙纳米颗粒。

Lipid-coated calcium phosphate nanoparticles for nonviral gene therapy.

作者信息

Haynes Matthew T, Huang Leaf

机构信息

Division of Molecular Pharmaceutics and Center for Nanotechnology in Drug Delivery, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

出版信息

Adv Genet. 2014;88:205-29. doi: 10.1016/B978-0-12-800148-6.00007-9.

Abstract

Though calcium phosphate (CaP) nanoparticles have been utilized as transfection agents in laboratory settings for nearly half a century, their power as a platform for therapeutically focused approaches has only recently been realized. With regard to modern advances in lipid- and polymer-mediated transfection, we present liposome CaP, a novel combination of a stable yet acid-labile CaP core with a surface functional, asymmetric lipid bilayer. Herein, the structural and functional dynamics of such nanoparticles are described in detail, including alteration of hepatocellular tropism through PEGylation and a multifunctional approach to endosomal escape, along with primary therapeutic advances, which these nanoparticles contribute to the field overall. Through these and other multifunctional nanotechnologies for gene therapy, great promise is shown in their ability to tackle truly complex and mechanistically demanding diseases.

摘要

尽管磷酸钙(CaP)纳米颗粒在实验室环境中作为转染剂已被使用了近半个世纪,但它们作为治疗性聚焦方法平台的潜力直到最近才被认识到。关于脂质和聚合物介导转染的现代进展,我们展示了脂质体CaP,它是一种稳定但酸不稳定的CaP核心与表面功能性不对称脂质双层的新型组合。本文详细描述了此类纳米颗粒的结构和功能动力学,包括通过聚乙二醇化改变肝细胞嗜性以及内体逃逸的多功能方法,以及这些纳米颗粒为整个领域带来的主要治疗进展。通过这些以及其他用于基因治疗的多功能纳米技术,它们在应对真正复杂且机制要求高的疾病方面展现出了巨大的前景。

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