Ding W, Zhang L, Yan Z, Engelhardt J F
Department of Anatomy and Cell Biology, University of Iowa School of Medicine, Iowa City, 52242, USA.
Gene Ther. 2005 Jun;12(11):873-80. doi: 10.1038/sj.gt.3302527.
Adeno-associated virus (AAV) has attracted considerable interest as a gene therapy vector over the past decade. In all, 85% of the current 2052 PubMed references on AAV (as of December 2004) have been published in the last 10 years. As researchers have moved forward with using this vector system for gene delivery, an increased appreciation for the complexities of AAV biology has emerged. The biology of recombinant AAV (rAAV) transduction has demonstrated considerable diversity in different cell types and target tissues. This review will summarize the current understanding of events that control rAAV transduction following receptor binding and leading to nuclear uptake. These stages are broadly classified as intracellular trafficking and have been found to be a major rate-limiting step in rAAV transduction for many cell types. Advances in understanding this area of rAAV biology will help to improve the efficacy of this vector system for the treatment of inherited and acquired diseases.
在过去十年中，腺相关病毒（AAV）作为一种基因治疗载体引起了广泛关注。截至2004年12月，在当前2052篇关于AAV的PubMed参考文献中，有85%是在过去10年发表的。随着研究人员推进使用这种载体系统进行基因递送，人们对AAV生物学复杂性的认识不断加深。重组腺相关病毒（rAAV）转导的生物学特性在不同细胞类型和靶组织中表现出相当大的差异。本综述将总结目前对受体结合后控制rAAV转导并导致核摄取的事件的理解。这些阶段大致归类为细胞内运输，并且已发现它们是许多细胞类型中rAAV转导的主要限速步骤。在理解rAAV生物学这一领域取得的进展将有助于提高该载体系统治疗遗传性和获得性疾病的疗效。
