RNA纳米医学:递送策略和应用。

IF 5 3区 医学 Q1 PHARMACOLOGY & PHARMACY AAPS Journal Pub Date : 2023-10-02 DOI:10.1208/s12248-023-00860-z
Junho Byun, Yina Wu, Jinwon Park, Jung Suk Kim, Qiaoyun Li, Jaehyun Choi, Namjo Shin, Meng Lan, Yu Cai, Jaiwoo Lee, Yu-Kyoung Oh
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引用次数: 0

摘要

使用纳米材料递送RNA已成为一种新的方式,以扩大生物医学研究中的治疗应用。然而,RNA的递送由于其易降解性和对高效细胞内递送的要求而带来了独特的挑战。纳米技术与RNA递送的结合解决了其中的许多挑战。在这篇综述中,我们讨论了用于RNA递送的纳米材料的设计和开发中使用的不同策略。我们还强调了通过纳米材料递送的RNA在药物应用方面的最新进展。各种纳米材料,如脂质、聚合物、肽、核酸和无机纳米材料,已被用于递送功能性RNA,包括信使核糖核酸(信使核糖核酸)、小干扰核糖核酸、单引导核糖核酸和微小核糖核酸。此外,纳米材料的利用扩大了功能性RNA作为活性药物成分的应用。例如,使用纳米材料递送编码抗原的信使核糖核酸能够使疫苗抗原瞬时表达,从而实现免疫原性和预防传染病。此外,纳米材料介导的RNA递送已被研究用于工程细胞表达外源功能蛋白。纳米材料也被用于共同递送单引导RNA和mRNA,以促进遗传疾病的基因编辑。除了在RNA医学方面取得的进展外,我们还讨论了该领域目前的挑战和未来的方向。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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RNA Nanomedicine: Delivery Strategies and Applications.

Delivery of RNA using nanomaterials has emerged as a new modality to expand therapeutic applications in biomedical research. However, the delivery of RNA presents unique challenges due to its susceptibility to degradation and the requirement for efficient intracellular delivery. The integration of nanotechnologies with RNA delivery has addressed many of these challenges. In this review, we discuss different strategies employed in the design and development of nanomaterials for RNA delivery. We also highlight recent advances in the pharmaceutical applications of RNA delivered via nanomaterials. Various nanomaterials, such as lipids, polymers, peptides, nucleic acids, and inorganic nanomaterials, have been utilized for delivering functional RNAs, including messenger RNA (mRNA), small interfering RNA, single guide RNA, and microRNA. Furthermore, the utilization of nanomaterials has expanded the applications of functional RNA as active pharmaceutical ingredients. For instance, the delivery of antigen-encoding mRNA using nanomaterials enables the transient expression of vaccine antigens, leading to immunogenicity and prevention against infectious diseases. Additionally, nanomaterial-mediated RNA delivery has been investigated for engineering cells to express exogenous functional proteins. Nanomaterials have also been employed for co-delivering single guide RNA and mRNA to facilitate gene editing of genetic diseases. Apart from the progress made in RNA medicine, we discuss the current challenges and future directions in this field.

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来源期刊
AAPS Journal
AAPS Journal 医学-药学
CiteScore
7.80
自引率
4.40%
发文量
109
审稿时长
1 months
期刊介绍: The AAPS Journal, an official journal of the American Association of Pharmaceutical Scientists (AAPS), publishes novel and significant findings in the various areas of pharmaceutical sciences impacting human and veterinary therapeutics, including: · Drug Design and Discovery · Pharmaceutical Biotechnology · Biopharmaceutics, Formulation, and Drug Delivery · Metabolism and Transport · Pharmacokinetics, Pharmacodynamics, and Pharmacometrics · Translational Research · Clinical Evaluations and Therapeutic Outcomes · Regulatory Science We invite submissions under the following article types: · Original Research Articles · Reviews and Mini-reviews · White Papers, Commentaries, and Editorials · Meeting Reports · Brief/Technical Reports and Rapid Communications · Regulatory Notes · Tutorials · Protocols in the Pharmaceutical Sciences In addition, The AAPS Journal publishes themes, organized by guest editors, which are focused on particular areas of current interest to our field.
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