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使用脂质纳米颗粒和细胞衍生纳米囊泡进行靶向基因递送的策略。

Strategies for targeted gene delivery using lipid nanoparticles and cell-derived nanovesicles.

作者信息

Lee Dong-Yup, Amirthalingam Sivashanmugam, Lee Changyub, Rajendran Arun Kumar, Ahn Young-Hyun, Hwang Nathaniel S

机构信息

School of Chemical and Biological Engineering, Institute of Chemical Processes, Seoul National University Seoul 08826 Republic of Korea

Institute of Engineering Research, Seoul National University Seoul 08826 Republic of Korea.

出版信息

Nanoscale Adv. 2023 Jul 7;5(15):3834-3856. doi: 10.1039/d3na00198a. eCollection 2023 Jul 25.

DOI:10.1039/d3na00198a
PMID:37496613
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10368001/
Abstract

Gene therapy is a promising approach for the treatment of many diseases. However, the effective delivery of the cargo without degradation is one of the major hurdles. With the advent of lipid nanoparticles (LNPs) and cell-derived nanovesicles (CDNs), gene delivery holds a very promising future. The targeting of these nanosystems is a prerequisite for effective transfection with minimal side-effects. In this review, we highlight the emerging strategies utilized for the effective targeting of LNPs and CDNs, and we summarize the preparation methodologies for LNPs and CDNs. We have also highlighted the non-ligand targeting of LNPs toward certain organs based on their composition. It is highly expected that continuing the developments in the targeting approaches of LNPs and CDNs for the delivery system will further promote them in clinical translation.

摘要

基因治疗是治疗多种疾病的一种很有前景的方法。然而，在不发生降解的情况下有效递送载荷是主要障碍之一。随着脂质纳米颗粒（LNPs）和细胞衍生纳米囊泡（CDNs）的出现，基因递送有着非常光明的前景。这些纳米系统的靶向性是实现有效转染且副作用最小的先决条件。在这篇综述中，我们重点介绍了用于有效靶向LNPs和CDNs的新兴策略，并总结了LNPs和CDNs的制备方法。我们还强调了基于LNPs的组成对某些器官的非配体靶向作用。人们高度期望，继续推进LNPs和CDNs递送系统靶向方法的发展将进一步推动它们在临床转化中的应用。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/c6c5a93714e8/d3na00198a-p3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/f50c374c342d/d3na00198a-f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/4848a24ca541/d3na00198a-f2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/bc3ace750b69/d3na00198a-f3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/70edeac6425c/d3na00198a-f4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/88f5ff4f2db8/d3na00198a-p1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/0f7a87fb5bcc/d3na00198a-p2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/c6c5a93714e8/d3na00198a-p3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/f50c374c342d/d3na00198a-f1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/4848a24ca541/d3na00198a-f2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/bc3ace750b69/d3na00198a-f3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/70edeac6425c/d3na00198a-f4.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/88f5ff4f2db8/d3na00198a-p1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/0f7a87fb5bcc/d3na00198a-p2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/a4b8/10368001/c6c5a93714e8/d3na00198a-p3.jpg

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[6]
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[7]
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[8]
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[9]
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[10]
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本文引用的文献

[1]
Analysis of aptamer-target binding and molecular mechanisms by thermofluorimetric analysis and molecular dynamics simulation.

Front Chem. 2023-5-9

[2]
Recent Advances in Biomolecular Detection Based on Aptamers and Nanoparticles.

Biosensors (Basel). 2023-4-13

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Molecules. 2023-4-26

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Cell-derived nanovesicles prepared by membrane extrusion are good substitutes for natural extracellular vesicles.

Extracell Vesicle. 2022-12

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Expert Opin Drug Discov. 2023-2

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Clin Transl Oncol. 2023-5

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Exosomes─Nature's Lipid Nanoparticles, a Rising Star in Drug Delivery and Diagnostics.

ACS Nano. 2022-11-22

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Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs) using multiple technical methods for tissue-specific mRNA delivery.

Nat Protoc. 2023-1

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Pharmaceutics. 2022-9-22

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Mol Ther. 2023-5-3

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