Fisher K J, Kelley W M, Burda J F, Wilson J M
Institute for Human Gene Therapy, University of Pennsylvania Medical Center, Philadelphia, USA.
Hum Gene Ther. 1996 Nov 10;7(17):2079-87. doi: 10.1089/hum.1996.7.17-2079.
Adenovirus and adeno-associated virus (AAV) are eukaryotic DNA viruses being developed as vectors for human gene therapy. The strengths of each system have been exploited in a novel vector that is based on an adenovirus-AAV hybrid virus incorporated into a plasmid-based molecular conjugate. Efficient rescue and replication of the recombinant AAV genome in this hybrid required transient expression of rep. This feature was incorporated into the transducing particle by conjugating a rep expression plasmid to the hybrid virus through a polylysine bridge. The resulting particle is an attractive vehicle for gene therapy because it is easily manufactured and capable of efficiently transducing cells with the end result being rescue and replication of the recombinant AAV genome. This particle is also useful in the production of recombinant AAV resulting in yields 10-fold greater than that achieved with transfection-based protocols.
腺病毒和腺相关病毒（AAV）是正在被开发用作人类基因治疗载体的真核DNA病毒。每个系统的优势都被应用于一种新型载体中，该载体基于整合到基于质粒的分子共轭物中的腺病毒-AAV杂交病毒。在这种杂交体中，重组AAV基因组的有效拯救和复制需要rep的瞬时表达。通过聚赖氨酸桥将rep表达质粒与杂交病毒共轭，这一特性被整合到转导颗粒中。所得颗粒是一种有吸引力的基因治疗载体，因为它易于制备，能够有效地转导细胞，最终实现重组AAV基因组的拯救和复制。这种颗粒在重组AAV的生产中也很有用，其产量比基于转染的方案高出10倍。
