基于腺相关病毒的阿尔茨海默病小鼠模型及潜在的新治疗途径。
Adeno-associated virus-based Alzheimer's disease mouse models and potential new therapeutic avenues.
作者信息
Ittner Lars M, Klugmann Matthias, Ke Yazi D
机构信息
Dementia Research Centre and Department of Biomedical Sciences, Faculty of Medicine and Health Sciences, Macquarie University, Sydney, NSW, Australia.
School of Medical Sciences, University of New South Wales, Sydney, NSW, Australia.
出版信息
Br J Pharmacol. 2019 Sep;176(18):3649-3665. doi: 10.1111/bph.14637. Epub 2019 Apr 23.
Abstract
Alzheimer's disease (AD) is a highly prevalent neurodegenerative condition that presents with cognitive decline. The current understanding of underlying disease mechanisms remains incomplete. Genetically modified mouse models have been instrumental in deciphering pathomechanisms in AD. While these models were typically generated by classical transgenesis and genome editing, the use of adeno-associated viruses (AAVs) to model and investigate AD in mice, as well as to develop novel gene-therapy approaches, is emerging. Here, we reviewed literature that used AAVs to study and model AD and discuss potential gene therapy strategies. LINKED ARTICLES: This article is part of a themed section on Therapeutics for Dementia and Alzheimer's Disease: New Directions for Precision Medicine. To view the other articles in this section visit http://onlinelibrary.wiley.com/doi/10.1111/bph.v176.18/issuetoc.
摘要
阿尔茨海默病（AD）是一种高度常见的神经退行性疾病，表现为认知功能下降。目前对其潜在疾病机制的理解仍不完整。基因工程小鼠模型在阐明AD的发病机制方面发挥了重要作用。虽然这些模型通常是通过经典转基因和基因组编辑构建的，但利用腺相关病毒（AAV）在小鼠中模拟和研究AD以及开发新的基因治疗方法正逐渐兴起。在此，我们综述了利用AAV研究和模拟AD的文献，并讨论了潜在的基因治疗策略。相关文章：本文是关于痴呆症和阿尔茨海默病治疗：精准医学新方向主题部分的一部分。要查看本部分的其他文章，请访问http://onlinelibrary.wiley.com/doi/10.1111/bph.v176.18/issuetoc。
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