During M J, Leone P
University of Auckland, New Zealand.
Clin Neurosci. 1995;3(5):292-300.
Adeno-associated virus (AAV) shows significant potential as a gene delivery system. Although it is ubiquitous in its distribution, with approximately 85% of the adult population in the United States seropositive for the virus, it has never been associated with clinical disease. Not only is AAV non-pathogenic, but it can infect with high efficiency both dividing and terminally-differentiated cells, moreover the wild-type virus integrates into a specific chromosomal site. It also has a broad host range and the virus is extremely resistant to environmental extremes. These characteristics make it particularly attractive as a gene delivery vehicle. As the enthusiasm driving the proliferation of clinical gene transfer protocols has dampened recently due to the lack of clinical success often reflecting immunogenicity and inefficiency of the gene transfer methods used in these trials, AAV with minimal (if any) toxicity and high efficiency in a wide range of cells and tissues, may become the vector-of-choice for many applications. There have been a number of comprehensive recent reviews of AAV biology and this article specifically discusses recent advances in the use of AAV vectors with particular emphasis on AAV vector-mediated in vivo gene transfer in the mammalian central nervous system.
腺相关病毒（AAV）作为一种基因传递系统显示出巨大潜力。尽管它分布广泛，在美国约85%的成年人口对该病毒血清学呈阳性，但它从未与临床疾病相关联。AAV不仅无致病性，而且能高效感染分裂细胞和终末分化细胞，此外野生型病毒会整合到特定的染色体位点。它还具有广泛的宿主范围，并且该病毒对极端环境具有极强的抵抗力。这些特性使其作为一种基因传递载体特别具有吸引力。由于缺乏临床成功案例，往往反映出这些试验中使用的基因传递方法具有免疫原性和低效性，最近推动临床基因转移方案激增的热情有所降温，而AAV在广泛的细胞和组织中具有最小（如果有的话）毒性和高效性，可能会成为许多应用的首选载体。最近有许多关于AAV生物学的全面综述，本文特别讨论了AAV载体使用方面的最新进展，尤其着重于AAV载体介导的哺乳动物中枢神经系统体内基因转移。
