Abstract: Vectors used to carry foreign genes play an important role in gene therapy,among which, the adeno-associated virus(AAV) has many advantages, such as nonpathogenicity, low immunogenicity, stable and long-term expression and multiple-tissue-type infection, ect. AAV is one of the hottest and most hopeful vectors in current days and has been extensively used in clinical research. With the research and development of AAV vector, the new vector systems will play a more important role in gene therapy. This review describes the recent development of AAV vector including the serum type, the new vector systems, the production processes and the scale up, the AAV-mediated clinical trials, the problems remained and the possible solutions. With the development of AAV vector, new vector system will play more important role in gene therapy.

Key words: adeno-associated virus(AAV), vector, gene therapy, serum type

摘要： 基因治疗的载体在基因治疗过程中起着至关重要的作用,其中腺相关病毒(AAV)载体具有低致病性、对宿主免疫原性弱、长期稳定表达、广泛的细胞和组织亲嗜性等优点,是目前发展最热,也是最有希望的载体之一,已被广泛应用于临床研究。本文综述了腺相关病毒载体的研究进展,包括AAV的血清型、新型载体的开发、生产工艺及其放大及临床研究及讨论了AAV载体还存在的问题及可能的解决方案。随着腺相关病毒载体研究的进展,新的载体系统必将在基因治疗中发挥更重要的作用。

关键词: 腺相关病毒, AAV, 载体, 基因治疗, 血清型