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Hot topics in adeno-associated virus as a gene transfer vector

机译:腺相关病毒作为基因转移载体的热点话题

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Adeno-associated virus (AAV) is a proising viral vector in treating many kinds of hereditary diseases.The broad host range,low level of imuneresponse,and longevity of gene expression observed with this vector have enabled theinitiatio of a number of clinical trials using this gene delivery system.Another potential benefit of AAV vectors is their ability to integrate site-specifically in the presence of Rep proteins.However,this virus is not well characterized.To obtain high level,persistent expressionf the foreign gene,some problems should be solved.In this article,we will describe the advances in some fields of recombinant AAV technology that overcome certain limitations of te vector as a gene delivery system,such as the transduction efficiency,the production,the package capacity,and elimination of immeune responses,as well as the applications involving these recombinant vectors for the treatmet of some diseases.
机译:腺伴随病毒(AAV)是一种正在治疗多种遗传性疾病的新型病毒载体。使用该载体观察到的宿主范围广,免疫应答水平低以及基因表达的寿命长,因此可以启动许多使用该病毒的临床试验。 AAV载体的另一个潜在好处是在Rep蛋白存在下它们能够特异性地整合位点。但是,这种病毒的特征并不十分清楚。要获得高水平,持续表达的外源基因,必须解决一些问题在本文中,我们将描述重组AAV技术在某些领域的进展,这些技术克服了te载体作为基因传递系统的某些局限性,例如转导效率,产量,包装能力以及消除免疫反应等。以及涉及这些重组载体的某些疾病的治疗应用。

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