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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 originate from Adeno-associated virus (AAV), which has no pathogenicity, low immunogenicity, integration and long-term expression in host cells And other advantages, is widely used in gene therapy research such as cancer and genetic diseases and is regarded as one of the most promising gene therapy vectors. However, the widespread presence of AAV neutralizing antibodies in the human body and the lack of targeting of rAAV to specific tissues or cells in the body have limited their clinical application. Chemically modified rAAV can resist serum neutralizing antibodies, improve transduction targeting, and achieve dynamic monitoring of rAAV in vivo, which provides a new idea and method for solving the current problems of rAAV vector. This article systematically elaborates the chemical modification of rAAV and gives a prospect of its future development.