Shimada T
Department of Biochemistry and Molecular Biology, Nippon Medical School.
Nihon Rinsho. 1997 Aug;55(8):2156-9.
Adeno-associated virus (AAV) is a non-pathogenic, replication defective parvovirus. In the absence of helper adenovirus, AAV stably integrates into a defined region of human chromosome 19. Because of these unique properties, recombinant AAV is considered to be an attractive vector for human gene therapy. It has been demonstrated that AAV vectors are capable of efficient transduction of various types of cells including hematopoietic cells and post mitotic neuronal cells. We have recently developed a new strategy for large scale preparation of high titer AAV vectors by using packaging cell lines and sulfonated cellulose column chromatography. This new strategy may be important for further development of gene therapy using AAV vectors.
腺相关病毒(AAV)是一种无致病性、复制缺陷型细小病毒。在没有辅助腺病毒的情况下,AAV可稳定整合到人类19号染色体的一个特定区域。由于这些独特的特性,重组AAV被认为是一种有吸引力的人类基因治疗载体。已经证明,AAV载体能够有效地转导包括造血细胞和有丝分裂后神经元细胞在内的各种类型的细胞。我们最近开发了一种新策略,通过使用包装细胞系和磺化纤维素柱色谱法大规模制备高滴度AAV载体。这种新策略对于使用AAV载体的基因治疗的进一步发展可能很重要。