利用纳米材料输送核酸。

IF 6.3 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY Molecular biomedicine Pub Date : 2023-12-14 DOI:10.1186/s43556-023-00160-0
Yuyang Qin, Liyuan Ou, Lili Zha, Yue Zeng, Ling Li
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引用次数: 0

摘要

越来越多的核酸疗法获得批准,这表明核酸疗法具有预防和治疗多种疾病的潜力。这一趋势凸显了核酸疗法在医学领域的重大影响和前景。然而,由于核酸容易被核酸酶降解,而且其不利的理化特性阻碍了核酸向细胞内的传递,因此将核酸作为治疗药物具有挑战性。适当的载体在提高核酸稳定性和将核酸送入特定细胞方面发挥着关键作用。随着递送系统的成熟,以 DNA、siRNA 和 mRNA 等核酸为基础的治疗药物的开发取得了突破性进展。非病毒载体具有免疫原性低、易于制造、成本效益高且易于大规模生产等优点,在众多纳米材料中脱颖而出。在此,我们将概述用于核酸递送的纳米材料的最新进展。具体来说,我们详细介绍了聚合物、脂质和聚合物-脂质混合物的特性,并全面阐述了它们在核酸递送中的应用。此外,我们还讨论了核酸的生物屏障、给药途径和器官选择性递送策略。总之,这篇综述为合理设计下一代核酸递送载体提供了启示。
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Delivery of nucleic acids using nanomaterials.

The increasing number of approved nucleic acid therapeutics demonstrates the potential for the prevention and treatment of a broad spectrum of diseases. This trend underscores the significant impact and promise of nucleic acid-based treatments in the field of medicine. Nevertheless, employing nucleic acids as therapeutics is challenging due to their susceptibility to degradation by nucleases and their unfavorable physicochemical characteristics that hinder delivery into cells. Appropriate vectors play a pivotal role in improving nucleic acid stability and delivering nucleic acids into specific cells. The maturation of delivery systems has led to breakthroughs in the development of therapeutics based on nucleic acids such as DNA, siRNA, and mRNA. Non-viral vectors have gained prominence among the myriad of nanomaterials due to low immunogenicity, ease of manufacturing, and simplicity of cost-effective, large-scale production. Here, we provide an overview of the recent advancements in nanomaterials for nucleic acid delivery. Specifically, we give a detailed introduction to the characteristics of polymers, lipids, and polymer-lipid hybrids, and provide comprehensive descriptions of their applications in nucleic acid delivery. Also, biological barriers, administration routes, and strategies for organ-selective delivery of nucleic acids are discussed. In summary, this review offers insights into the rational design of next-generation delivery vectors for nucleic acid delivery.

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来源期刊
CiteScore
6.30
自引率
0.00%
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0
审稿时长
10 weeks
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