腺相关病毒(AAV)载体在感染细胞中的基因组整合

Q4 Medicine
Yukihiko Hirai
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引用次数: 0

摘要

利用腺相关病毒(AAV)载体的基因治疗产品的开发正在取得进展,基因治疗市场正在迅速扩大。AAV在人体中没有致病性,具有极低的细胞毒性,并且与慢病毒和逆转录病毒载体不同,很少整合到染色体中。因此,与AAV相关的风险被认为是低的。然而,近年来,随着病例在实验动物和临床试验中积累,肝毒性等不良事件已经变得明显。在本文中,我们介绍了AAV载体整合到感染细胞基因组的概述,这被认为是不良事件的原因。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Genome Integration of Adeno-associated Virus (AAV) Vectors into Infected Cells
The development of gene therapy products using adeno-associated virus (AAV) vectors is progressing, and the gene therapy market is rapidly expanding. AAV shows no pathogenicity in the human body, has extremely low cytotoxicity and, unlike lentiviral and retroviral vectors, rarely integrates into chromosomes. Consequently, risk associated with AAV was thought to be low. In recent years, however, adverse events such as hepatotoxicity have become apparent as cases accumulate among laboratory animals and in clinical trials. In this paper, we present an overview of the integration of AAV vectors into the genome of infected cells, which is thought to be the cause of adverse events.
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Journal of Tokyo Medical University
Journal of Tokyo Medical University Medicine-Medicine (all)
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