The Responsible Development of Adeno-Associated Virus Gene Therapies.

IF 3.9 3区医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Human gene therapy Pub Date : 2025-07-11 DOI:10.1177/10430342251359668

James M Wilson, Arthur L Caplan

引用次数: 0

Abstract

Several unexpected fatalities in patients who received adeno-associated virus (AAV)-based gene therapies have recently occurred. These tragic events have cast a pall over the entire sector with some stakeholders suggesting that AAV is patently unsafe as a gene delivery platform and ought not to be pursued. This conclusion is not warranted.

查看原文本刊更多论文

腺相关病毒基因疗法的负责任发展

最近发生了几例接受腺相关病毒（AAV）基因治疗的患者意外死亡。这些悲剧性事件给整个行业蒙上了一层阴影，一些利益相关者认为，作为基因传递平台，AAV显然是不安全的，不应该继续发展。这个结论是站不住脚的。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

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.