化学修饰的重组腺相关病毒载体

Chemical Modification of Recombinant Adenovirus-associated Virus Vectors

重组腺相关病毒(Recombinant adenovirus-associated virus,rAAV)载体源于腺相关病毒(Adeno-associated virus,AAV),以其无致病性、低免疫原性、可定点整合及在宿主细胞内长期表达等优势,广泛应用于肿瘤和遗传疾病等基因治疗研究,被视为最有前途的基因治疗载体之一。但是人体内广泛存在AAV中和抗体,以及rAAV对体内特定组织或细胞的靶向性欠理想,限制了其在临床上的应用。经化学修饰的rAAV可以抵御血清中和抗体,提高转导靶向性,还可实现rAAV体内动态监测,这为解决当前rAAV载体的问题提供了新的思路和方法。本文对化学修饰的rAAV展开系统阐述,并对其未来发展趋势作出展望。

Recombinant adenovirus-associated virus （rAAV） vectors mediated gene delivery system has been widely used in the treatments of cancer or other genetic diseases. It is considered to be one of the most promising vector owing to its non-pathogenicicity, low immune response, potential to integrate site-specif- ically, persistent and stable gene expression. However, it was limited in clinical applications because it is easy to be neutralized in serum and non-selection to the target site. Chemical modifications of rAAV have been studied to overcome those problems. This article reviewed the progress of chemical modifications of rAAV by various compounds and different modification methods, and predicted what the researches needed to do in the future.