Büning Hildegard, Perabo Luca, Coutelle Oliver, Quadt-Humme Sibille, Hallek Michael
Clinic I for Internal Medicine, University of Cologne, Cologne, Germany.
J Gene Med. 2008 Jul;10(7):717-33. doi: 10.1002/jgm.1205.
Adeno-associated virus (AAV), a single-stranded DNA parvovirus, is emerging as one of the leading gene therapy vectors owing to its nonpathogenicity and low immunogenicity, stability and the potential to integrate site-specifically without known side-effects. A portfolio of recombinant AAV vector types has been developed with the aim of optimizing efficiency, specificity and thereby also the safety of in vitro and in vivo gene transfer. More and more information is now becoming available about the mechanism of AAV/host cell interaction improving the efficacy of recombinant AAV vector (rAAV) mediated gene delivery. This review summarizes the current knowledge of the infectious biology of AAV, provides an overview of the latest developments in the field of AAV vector technology and discusses remaining challenges.
腺相关病毒（AAV）是一种单链DNA细小病毒，由于其无致病性、低免疫原性、稳定性以及能够位点特异性整合且无已知副作用的潜力，正逐渐成为主要的基因治疗载体之一。为了优化体外和体内基因转移的效率、特异性以及安全性，已经开发了一系列重组AAV载体类型。现在，关于AAV/宿主细胞相互作用机制的信息越来越多，这有助于提高重组AAV载体（rAAV）介导的基因递送效率。本综述总结了目前关于AAV感染生物学的知识，概述了AAV载体技术领域的最新进展，并讨论了尚存的挑战。
