The state of the art of adeno-associated virus-based vectors in gene therapy.
The state of the art of adeno-associated virus-based vectors in gene therapy.
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DOI：
10.1186/1743-422x-4-99
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发表时间：
2007-10-16
期刊：
影响因子：
4.8
通讯作者：
Nardi NB
中科院分区：
文献类型：
--
作者：
Coura Rdos S;Nardi NB
自从1982年第一个AAV2感染性克隆建立以来，腺相关病毒(AAV)因其缺乏致病性、广泛的感染性和建立长期转基因表达的能力等特点而在基因治疗中迅速流行起来。值得注意的是，在过去的十年里，这种病毒作为一种基因治疗载体引起了相当大的兴趣，目前可获得的2041篇关于腺相关病毒的PubMed参考文献中，约有85%是在此期间发表的。AAV病毒的病毒学和生物学知识的进步使得AAV载体的指数式发展成为可能，这使得AAV载体的构建得到了极大的改进，并使人们对其操作有了更好的理解。此外，随着最近新的AAV血清型的发现，几乎每个器官或组织都有一种首选的血清型作为靶标。因此，基于AAV的载体已经成功地克服了转基因维持、安全性和宿主免疫反应等主要的基因治疗挑战，并从潜在的应用角度满足了高安全性、临床有效性和通用性的理想载体系统的特点。因此，AAV正日益成为广泛基因治疗方法的选择载体。这份报告将重点介绍基于AAV载体的研究现状以及AAV载体在几种基因治疗方法中的应用进展。
The adeno-associated virus (AAV) has rapidly gained popularity in gene therapy since the establishment of the first AAV2 infectious clone, in 1982, due to some of their distinguishing characteristics such as lack of pathogenicity, wide range of infectivity, and ability to establish long-term transgene expression. Notably over the past decade, this virus has attracted considerable interest as a gene therapy vector, and about 85% of the currently available 2,041 PubMed references on adeno-associated viruses have been published during this time. The exponential progress of AAV-based vectors has been made possible by the advances in the knowledge of the virology and biology of this virus, which allows great improvement in AAV vectors construction and a better comprehension of their operation. Moreover, with the recent discovery of novel AAV serotypes, there is virtually one preferred serotype for nearly every organ or tissue to target. Thus, AAV-based vectors have been successfully overcoming the main gene therapy challenges such as transgene maintenance, safety and host immune response, and meeting the desirable vector system features of high level of safety combined with clinical efficacy and versatility in terms of potential applications. Consequently, AAV is increasingly becoming the vector of choice for a wide range of gene therapy approaches. This report will highlight the state of the art of AAV-based vectors studies and the advances on the use of AAV vectors for several gene therapy approaches.