Gene and Stem Cell Therapy Program Centenary Institute, The University of Sydney, Camperdown, NSW 2050, Australia; Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia.
Gene and Stem Cell Therapy Program Centenary Institute, The University of Sydney, Camperdown, NSW 2050, Australia; Faculty of Medicine and Health, The University of Sydney, Sydney, NSW 2006, Australia; Cell and Molecular Therapies, Royal Prince Alfred Hospital, Missenden Road, Camperdown, NSW 2050, Australia.
Trends Mol Med. 2021 Feb;27(2):172-184. doi: 10.1016/j.molmed.2020.09.010. Epub 2020 Oct 15.
As adeno-associated virus (AAV)-based gene therapies are being increasingly approved for use in humans, it is important that we understand vector-host interactions in detail. With the advances in genome-wide genetic screening tools, a clear picture of AAV-host interactions is beginning to emerge. Understanding these interactions can provide insights into the viral life cycle. Accordingly, novel strategies to circumvent the current limitations of AAV-based vectors may be explored. Here, we summarize our current understanding of the various stages in the journey of the vector from the cell surface to the nucleus and contextualize the roles of recently identified host factors.
随着腺相关病毒(AAV)为基础的基因治疗方法在人类中越来越被批准使用,详细了解载体-宿主相互作用非常重要。随着全基因组遗传筛选工具的进步,AAV-宿主相互作用的清晰图景开始显现。了解这些相互作用可以深入了解病毒的生命周期。因此,可以探索新的策略来规避基于 AAV 载体的当前局限性。在这里,我们总结了我们目前对载体从细胞表面到细胞核的各个阶段的理解,并将最近确定的宿主因子的作用置于上下文中。