实现基因治疗的关键在于目的基因的高效转移并适度表达 ,这将直接影响其治疗的效率和安全性。因此 ,探寻理想的基因转移载体显得尤为重要。腺病毒相关病毒 (adeno- associated virus,AAV)是一种缺陷型的单链DNA病毒 ,既可以转染分裂细胞又可以转染非分裂细胞。 AAV在宿主体内以定向整合的方式存在。 AAV重组体在细胞内能长期稳定地表达 ,且在体内不引起明显的病理变化 ,表明 AAV作为基因治疗的载体是安全、有效和可行的 The key to achieve gene therapy is the efficient transfer of gene of interest and moderate expression, which will directly affect the efficiency and safety of its treatment. Therefore, to explore the ideal gene transfer vector is particularly important. Adeno-associated virus (AAV) is a defective single-stranded DNA virus that can be transfected into both dividing and non-dividing cells. AAV exists in a targeted integration in the host. AAV recombinant can be stably expressed in the cell for a long time without any significant pathological changes in vivo, suggesting that AAV is safe, effective and feasible as a gene therapy vector