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用于RNA递送的脂质纳米颗粒制剂与制备的综合分析。

Comprehensive analysis of lipid nanoparticle formulation and preparation for RNA delivery.

作者信息

Haque Md Anamul, Shrestha Archana, Mikelis Constantinos M, Mattheolabakis George

机构信息

School of Basic Pharmaceutical and Toxicological Sciences, College of Pharmacy, University of Louisiana at Monroe, Monroe, LA 71201, USA.

Laboratory of Molecular Pharmacology, Department of Pharmacy, University of Patras, Patras 26504, Greece.

出版信息

Int J Pharm X. 2024 Sep 10;8:100283. doi: 10.1016/j.ijpx.2024.100283. eCollection 2024 Dec.

DOI:10.1016/j.ijpx.2024.100283
PMID:39309631
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11415597/
Abstract

Nucleic acid-based therapeutics are a common approach that is increasingly popular for a wide spectrum of diseases. Lipid nanoparticles (LNPs) are promising delivery carriers that provide RNA stability, with strong transfection efficiency, favorable and tailorable pharmacokinetics, limited toxicity, and established translatability. In this review article, we describe the lipid-based delivery systems, focusing on lipid nanoparticles, the need of their use, provide a comprehensive analysis of each component, and highlight the advantages and disadvantages of the existing manufacturing processes. We further summarize the ongoing and completed clinical trials utilizing LNPs, indicating important aspects/questions worth of investigation, and analyze the future perspectives of this significant and promising therapeutic approach.

摘要

基于核酸的疗法是一种常见的方法,在广泛的疾病治疗中越来越受欢迎。脂质纳米颗粒(LNPs)是很有前景的递送载体,能提供RNA稳定性,具有强大的转染效率、良好且可定制的药代动力学、有限的毒性以及已确立的可转化性。在这篇综述文章中,我们描述了基于脂质的递送系统,重点是脂质纳米颗粒,阐述了使用它们的必要性,对每个组分进行了全面分析,并突出了现有制造工艺的优缺点。我们进一步总结了正在进行和已经完成的使用LNPs的临床试验,指出了值得研究的重要方面/问题,并分析了这种重要且有前景的治疗方法的未来前景。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/480c2d90fdb0/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/3f3eff34f40e/ga1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/5878c393cde3/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/f9f1688bfa3c/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/480c2d90fdb0/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/3f3eff34f40e/ga1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/5878c393cde3/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/f9f1688bfa3c/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9c0a/11415597/480c2d90fdb0/gr3.jpg

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