用于基因递送的刺激响应性非病毒纳米粒子。

Q1 Pharmacology, Toxicology and Pharmaceutics Handbook of experimental pharmacology Pub Date : 2024-01-01 DOI:10.1007/164_2023_694

Liên S Reichel, Anja Traeger

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引用次数: 0

摘要

核酸是生命的语言，基因组是细胞的说明书，因此对核酸进行有针对性的调控有望为治疗和治愈疾病带来巨大的机遇。除病毒基因转移外，非病毒 mRNA 疫苗的巨大威力也推动了新型基因转运体的发展。因此，DNA（pDNA）或 RNA（mRNA、siRNA、miRNA、gRNA 或 ASOs）等各种核酸需要进行转运，由于其摩尔质量高且带负电荷，与传统制剂相比需要转运体。本章介绍了使用核酸的具体生物学障碍，并说明了新材料如何克服这些障碍。

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Stimuli-Responsive Non-viral Nanoparticles for Gene Delivery.

Considering nucleic acids as the language of life and the genome as the instruction manual of cells, their targeted modulation promises great opportunities in treating and healing diseases. In addition to viral gene transfer, the overwhelming power of non-viral mRNA-based vaccines is driving the development of novel gene transporters. Thereby, various nucleic acids such as DNA (pDNA) or RNA (mRNA, siRNA, miRNA, gRNA, or ASOs) need to be delivered, requiring a transporter due to their high molar mass and negative charge in contrast to classical agents. This chapter presents the specific biological hurdles for using nucleic acids and shows how new materials can overcome these.

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来源期刊

Handbook of experimental pharmacology Pharmacology, Toxicology and Pharmaceutics-Pharmacology, Toxicology and Pharmaceutics (all)

CiteScore

5.20

自引率

0.00%

发文量

期刊介绍： The Handbook of Experimental Pharmacology is one of the most authoritative and influential book series in pharmacology. It provides critical and comprehensive discussions of the most significant areas of pharmacological research, written by leading international authorities. Each volume in the series represents the most informative and contemporary account of its subject available, making it an unrivalled reference source.