腺相关病毒作为基因治疗载体在生殖系统中的分布及影响的研究进展

腺相关病毒(adeno-associated virus,AAV)作为一种基因治疗载体被广泛应用于医学相关的各个领域。大量以AAV为载体的基因治疗被应用于药物研究,但目前国内外没有相关的基因药物应用于临床,原因在于其安全性问题有待进一步研究。AAV具有器官靶向性,易于分布在靶器官,但在非靶器官也会分布。AAV在生殖系统的短暂分布降低了对其功能的影响,但不排除潜在影响。就目前的研究结果来看,AAV不会整合在生殖细胞,从而不会对子代产生影响。本文将综述目前以腺相关病毒介导的基因治疗中对生殖系统安全性的研究进展,从载体在生殖系统的分布、对生殖系统功能的影响以及对子代影响的三个方面进行阐述。

Progress in the Distribution and Influence of Adeno-associated Virus as a Gene Therapy Vector in the Reproductive System

ABSTRACT: Adeno associated virus as a vector for gene therapy has been widely used in various fields related to medicine, its biological characteristics have been thoroughly understood. At present a large number of recombinant adeno-associated virus vectors for gene therapy is applied in drug research, no relevant gene drugs in clinical application at home and abroad, reason is the safety problems still need to be further studied. AAV has the target of tissue and organ, easy to distribute in target organs, but also distributes in non target organs. The transient distribution of AAV in the reproductive system reduces its impact on its function, but does not exclude potential effects. As far as the present study is concerned, AAV does not integrate in the germ cells, and does not affect the offspring. This paper will research progress on safety of the reproductive system in the review of current gene therapy mediated by adeno-associated virus, three effect of biodistribution in the reproductive system, impact on reproductive function and offspring effects are discussed.