利用腺相关病毒作为治疗遗传疾病或人类癌症的载体。

IF 3.7 3区 生物学 Q2 BIOCHEMISTRY & MOLECULAR BIOLOGY
IUBMB Life Pub Date : 2024-07-06 DOI:10.1002/iub.2896
Fu-Hsuan Shih, Hsiung-Hao Chang, Yi-Ching Wang
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引用次数: 0

摘要

二十多年来,3000 多名患者接受了治疗,这些临床数据表明,腺相关病毒(AAV)基因疗法是一种安全、有效、耐受性良好的治疗方法。利用 AAV 介导的基因传递到可触及组织的临床试验已成功治疗了许多单基因疾病,并推动了组织工程学的发展。尽管美国食品和药物管理局(FDA)已批准将 AAV 用于临床,但全身给药仍是一项重大挑战。在本综述中,我们将深入研究 AAV 生物学,重点关注当前的制造技术和转基因工程策略。我们研究了 AAV 在眼科、神经和血液疾病以及癌症临床试验中的应用。通过讨论该领域的最新进展和当前面临的挑战,我们旨在为研究人员和临床医生提供有价值的见解,帮助他们了解基于 AAV 的基因疗法不断发展的现状。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Utilizing adeno-associated virus as a vector in treating genetic disorders or human cancers

Clinical data from over two decades, involving more than 3000 treated patients, demonstrate that adeno-associated virus (AAV) gene therapy is a safe, effective, and well-tolerated therapeutic method. Clinical trials using AAV-mediated gene delivery to accessible tissues have led to successful treatments for numerous monogenic disorders and advancements in tissue engineering. Although the US Food and Drug Administration (FDA) has approved AAV for clinical use, systemic administration remains a significant challenge. In this review, we delve into AAV biology, focusing on current manufacturing technologies and transgene engineering strategies. We examine the use of AAVs in ongoing clinical trials for ocular, neurological, and hematological disorders, as well as cancers. By discussing recent advancements and current challenges in the field, we aim to provide valuable insights for researchers and clinicians navigating the evolving landscape of AAV-based gene therapy.

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来源期刊
IUBMB Life
IUBMB Life 生物-生化与分子生物学
CiteScore
10.60
自引率
0.00%
发文量
109
审稿时长
4-8 weeks
期刊介绍: IUBMB Life is the flagship journal of the International Union of Biochemistry and Molecular Biology and is devoted to the rapid publication of the most novel and significant original research articles, reviews, and hypotheses in the broadly defined fields of biochemistry, molecular biology, cell biology, and molecular medicine.
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