神经系统疾病基因治疗药物传递系统的应用与发展

IF 3.7 4区 医学 Q2 PHARMACOLOGY & PHARMACY Advanced Therapeutics Pub Date : 2024-11-02 DOI:10.1002/adtp.202400269
Ngoc Hong Nguyen, Phuong-Trang Nguyen-Thi, Thuy Trang Nguyen, Vu Khac Hoang Bui, Nhat Thang Thi Nguyen, Giau Van Vo
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引用次数: 0

摘要

神经系统疾病(NDs),如阿尔茨海默病、帕金森病、缺血性中风、脊髓损伤和其他类似疾病,继续在全球范围内造成重大的健康和经济负担。关键是要解决现有药物由于副作用及其免疫反应而带来的困难,以开发改进的神经退行性疾病治疗方法。基因治疗目前被广泛用于各种疾病的临床前和临床研究,因为它能够增强治疗的交付和有效性。各种基因传递技术,包括信使RNA、小干扰RNA、反义寡核苷酸、microRNA、CRISPR/Cas9系统和质粒DNA,已经被创造出来解决这些困难。本研究的目的是为NDs疾病的病理生理基础提供一个清晰的概述,同时也阐明了基因传递载体技术的最新发展。它介绍了这些载体的主要分类,它们各自的优点和缺点,以及它们在基因治疗中的具体应用。
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Applications and Developments of Gene Therapy Drug Delivery Systems for Neurological Disorders

Neurological diseases (NDs) such as Alzheimer's disease, Parkinson's disease, ischemic strokes, spinal cord injuries, and other similar conditions that continue to pose a substantial health and economic burden on a global scale. It is crucial to tackle the difficulties provided by current medications due to the adverse effects and its immunological reactions to develop improved treatments for neurodegenerative illnesses. Gene therapy is currently being extensively used in preclinical and clinical studies for various diseases because of its ability to enhance the delivery and effectiveness of treatments. Various gene delivery techniques, including messenger RNA, small interfering RNA, antisense oligonucleotides, microRNA, CRISPR/Cas9 system, and plasmid DNA, have been created to address these difficulties. The goal of this study is to provide a clear overview of the pathophysiological underpinnings of NDs illnesses while also illuminating recent developments in gene delivery vector technologies. It goes over the main classifications of these vectors, their individual benefits and drawbacks, and their specific applications in the delivery of gene therapy.

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来源期刊
Advanced Therapeutics
Advanced Therapeutics Pharmacology, Toxicology and Pharmaceutics-Pharmaceutical Science
CiteScore
7.10
自引率
2.20%
发文量
130
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