用于基因治疗的工程化腺相关病毒受体足迹
Engineering AAV receptor footprints for gene therapy.
作者信息
Madigan Victoria J, Asokan Aravind
机构信息
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC, United States; Curriculum in Genetics and Molecular Biology, The University of North Carolina at Chapel Hill, Chapel Hill, NC, United States.
Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC, United States; Department of Genetics, The University of North Carolina at Chapel Hill, Chapel Hill, NC, United States; Department of Biochemistry & Biophysics, The University of North Carolina at Chapel Hill, Chapel Hill, NC, United States.
出版信息
Curr Opin Virol. 2016 Jun;18:89-96. doi: 10.1016/j.coviro.2016.05.001. Epub 2016 Jun 2.
Adeno-associated viruses (AAV) are currently at the forefront of human gene therapy clinical trials as recombinant vectors. Significant progress has been made in elucidating the structure, biology and tropisms of different naturally occurring AAV isolates in the past decade. In particular, a spectrum of AAV capsid interactions with host receptors have been identified and characterized. These studies have enabled a better understanding of key determinants of AAV cell recognition and entry in different hosts. This knowledge is now being applied toward engineering new, lab-derived AAV capsids with favorable transduction profiles. The current review conveys a structural perspective of capsid-glycan interactions and provides a roadmap for generating synthetic strains by engineering AAV receptor footprints.
腺相关病毒（AAV）作为重组载体，目前处于人类基因治疗临床试验的前沿。在过去十年中，在阐明不同天然存在的AAV分离株的结构、生物学特性和嗜性方面取得了重大进展。特别是，已经鉴定并表征了一系列AAV衣壳与宿主受体的相互作用。这些研究有助于更好地理解AAV在不同宿主中细胞识别和进入的关键决定因素。这一知识目前正被应用于设计具有良好转导特性的新型实验室衍生AAV衣壳。本综述从结构角度阐述了衣壳-聚糖相互作用，并为通过改造AAV受体足迹来生成合成菌株提供了路线图。
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