用于在肝脏外输送核酸的脂质纳米颗粒。

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Human gene therapy Pub Date : 2024-09-01 Epub Date: 2024-08-28 DOI:10.1089/hum.2024.106
Nadine Saber, Mariona Estapé Senti, Raymond M Schiffelers
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引用次数: 0

摘要

脂质纳米颗粒是临床上最先进的核酸治疗药物递送系统,COVID-19 mRNA 疫苗的成功就是例证。然而,由于脂质纳米颗粒在静脉注射时容易在肝脏中蓄积,因此其临床应用目前仅限于肝脏疾病和疫苗。要充分发挥它们的潜力,就必须了解并解决它们的肝脏滋养性问题,同时还要制定策略,加强向肝脏以外组织的输送。确保这些疗法到达靶细胞,同时避免脱靶细胞,对其疗效和安全性至关重要。目前有三种潜在的靶向策略--被动靶向、主动靶向和内源性靶向--可单独或组合使用,以靶向非肝脏组织。在这篇综述中,我们将深入探讨脂质纳米粒子工程学在向肝外输送核酸方面的最新进展。
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Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver.

Lipid nanoparticles (LNPs) are the most clinically advanced drug delivery system for nucleic acid therapeutics, exemplified by the success of the COVID-19 mRNA vaccines. However, their clinical use is currently limited to hepatic diseases and vaccines due to their tendency to accumulate in the liver upon intravenous administration. To fully leverage their potential, it is essential to understand and address their liver tropism, while also developing strategies to enhance delivery to tissues beyond the liver. Ensuring that these therapeutics reach their target cells while avoiding off-target cells is essential for both their efficacy and safety. There are three potential targeting strategies-passive, active, and endogenous-which can be used individually or in combination to target nonhepatic tissues. In this review, we delve into the recent advancements in LNP engineering for delivering nucleic acid beyond the liver.

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来源期刊
Human gene therapy
Human gene therapy 医学-生物工程与应用微生物
CiteScore
6.50
自引率
4.80%
发文量
131
审稿时长
4-8 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
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