Ding L, Lu S, Munshi N C
Department of Medicine, University of Arkansas for Medical Sciences, Little Rock, Arkansas 72205, USA.
Gene Ther. 1997 Nov;4(11):1167-72. doi: 10.1038/sj.gt.3300514.
Adeno-associated virus 2 (AAV), a human parvovirus, has properties such as stable chromosomal integration, high infectivity and lack of known human pathogenicity, making it a potentially useful vector for human gene therapy. AAV requires a helper virus, such as an adenovirus, for optimal replication and packaging in mammalian cells. Although replication of the wild-type AAV genome has been demonstrated in vitro, packaging of infectious viral particles was not documented until now. In this study, we produced in vitro infectious recombinant AAV virions containing the neomycin resistance (NeoR) and the human CD-16 (FC gamma RIIIa) gene. Our in vitro packaging of AAV has the same characteristics as AAV produced in vivo and demonstrates the feasibility of developing a safe packaging method for AAV to be used in gene therapy.
腺相关病毒2型（AAV）是一种人类细小病毒，具有染色体整合稳定、感染性高以及未知人类致病性等特性，使其成为人类基因治疗中一种潜在有用的载体。AAV需要一种辅助病毒，如腺病毒，以便在哺乳动物细胞中进行最佳复制和包装。尽管野生型AAV基因组的复制已在体外得到证实，但传染性病毒颗粒的包装直到现在才被记录。在本研究中，我们在体外产生了含有新霉素抗性（NeoR）和人类CD-16（FCγRIIIa）基因的传染性重组AAV病毒粒子。我们对AAV的体外包装具有与体内产生的AAV相同的特性，并证明了开发一种用于基因治疗的AAV安全包装方法的可行性。
