Carter P J, Samulski R J
Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599-7352, USA.
Int J Mol Med. 2000 Jul;6(1):17-27. doi: 10.3892/ijmm.6.1.17.
Adeno-associated virus (AAV), a non-pathogenic human parvovirus, is gaining attention for its potential use as a human gene therapy vector. One of the most attractive features of recombinant AAV vectors is the ability to be stably maintained in host cells as integrated proviruses. This property is particularly desireable for therapies requiring long-term correction of a genetic defect. This review highlights recent advances made in the AAV field and will discuss some limitations of rAAV vector integration. A novel method for enhancing the integration efficiency of these vectors will be presented.
腺相关病毒(AAV)是一种非致病性人类细小病毒,因其作为人类基因治疗载体的潜在用途而受到关注。重组腺相关病毒载体最吸引人的特点之一是能够作为整合型原病毒稳定地维持在宿主细胞中。对于需要长期纠正基因缺陷的治疗方法来说,这一特性尤为可取。本综述重点介绍了腺相关病毒领域的最新进展,并将讨论重组腺相关病毒载体整合的一些局限性。还将介绍一种提高这些载体整合效率的新方法。