Qin Yuyang, Ou Liyuan, Zha Lili, Zeng Yue, Li Ling
West China School of Public Health and West China Fourth Hospital, and State Key Laboratory of Biotherapy, Sichuan University, Chengdu, 610041, China.
Mol Biomed. 2023 Dec 14;4(1):48. doi: 10.1186/s43556-023-00160-0.
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.
获批的核酸疗法数量不断增加,这表明其在预防和治疗多种疾病方面具有潜力。这一趋势凸显了基于核酸的治疗方法在医学领域的重大影响和前景。然而,将核酸用作治疗剂具有挑战性,因为它们易被核酸酶降解,且其不利的物理化学特性阻碍了它们进入细胞。合适的载体在提高核酸稳定性以及将核酸递送至特定细胞方面起着关键作用。递送系统的成熟已在基于核酸(如DNA、小干扰RNA和信使核糖核酸)的治疗方法开发中取得突破。非病毒载体由于免疫原性低、易于制造且成本效益高、大规模生产简单,在众多纳米材料中备受瞩目。在此,我们概述了用于核酸递送的纳米材料的最新进展。具体而言,我们详细介绍了聚合物、脂质和聚合物 - 脂质杂化物的特性,并全面描述了它们在核酸递送中的应用。此外,还讨论了生物屏障、给药途径以及核酸器官选择性递送的策略。总之,本综述为核酸递送的下一代递送载体的合理设计提供了见解。