骨靶向给药的进展:使用腺相关病毒的新策略。

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Human gene therapy Pub Date : 2024-04-25 DOI:10.1089/hum.2024.034
Tadatoshi Sato, Sachin Chaugule, Matthew Greenblatt, Guangping Gao, Jae-Hyuck Shim
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引用次数: 0

摘要

骨靶向给药系统的开发为改善骨骼疾病的治疗带来了巨大希望。通过将治疗药物精确地输送到骨骼的受影响区域,这些策略可以提高药物疗效,最大限度地减少脱靶效应,并促进患者的依从性,最终改善治疗效果,提高患者的生活质量。本综述旨在概述基于亲和力的骨靶向药物的现状,以及治疗小鼠骨骼疾病的创新性骨靶向腺相关病毒(AAV)策略的最新突破。特别是,本综述将深入探讨先进的 AAV 工程,包括骨靶向 AAV 血清型的选择和骨特异性滋养的囊膜修饰。此外,我们还将重点介绍 AAV 介导的骨骼疾病基因疗法的最新进展,并讨论这一前景广阔的治疗方法所面临的挑战和未来发展方向。
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Advances in bone-targeting drug delivery: Emerging strategies using adeno-associated virus.
The development of bone-targeting drug delivery systems holds immense promise for improving the treatment of skeletal diseases. By precisely delivering therapeutic agents to the affected areas of bone, these strategies can enhance drug efficacy, minimize off-target effects, and promote patient adherence, ultimately leading to improved treatment outcomes and an enhanced quality of life for patients. This review aims to provide an overview of the current state of affinity-based bone-targeting agents and recent breakthroughs in innovative bone-targeting adeno-associated virus (AAV) strategies to treat skeletal diseases in mice. In particular, this review will delve into advanced AAV engineering, including AAV serotype selection for bone targeting and capsid modifications for bone-specific tropism. Additionally, we will highlight recent advancements in AAV-mediated gene therapy for skeletal diseases and discuss challenges and future directions of this promising therapeutic approach.
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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.
期刊最新文献
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