Louis Jeune Vedell, Joergensen Jakob A, Hajjar Roger J, Weber Thomas
Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Hum Gene Ther Methods. 2013 Apr;24(2):59-67. doi: 10.1089/hgtb.2012.243. Epub 2013 Apr 3.
Adeno-associated virus (AAV)-based vectors are promising tools for gene therapeutic applications, in part because AAVs are nonpathogenic viruses, and vectors derived from them can drive long-term transgene expression without integration of the vector DNA into the host genome. AAVs are not strongly immunogenic, but they can, nonetheless, give rise to both a cellular and humoral immune response. As a result, a significant fraction of potential patients for AAV-based gene therapy harbors pre-existing antibodies against AAV. Because even very low levels of antibodies can prevent successful transduction, antecedent anti-AAV antibodies pose a serious obstacle to the universal application of AAV gene therapy. In this review, we discuss the current knowledge of the role of anti-AAV antibodies in AAV-based gene therapy with a particular emphasis on approaches to overcome the hurdle that they pose.
基于腺相关病毒(AAV)的载体是基因治疗应用中很有前景的工具,部分原因是AAV是非致病性病毒,由它们衍生的载体可以驱动转基因长期表达,而无需将载体DNA整合到宿主基因组中。AAV的免疫原性不强,但仍可引发细胞免疫和体液免疫反应。因此,很大一部分潜在的基于AAV的基因治疗患者体内存在针对AAV的预先存在的抗体。由于即使是非常低水平的抗体也会阻碍成功转导,所以预先存在的抗AAV抗体对AAV基因治疗的广泛应用构成了严重障碍。在这篇综述中,我们讨论了抗AAV抗体在基于AAV的基因治疗中的作用的当前知识,特别强调了克服它们所带来障碍的方法。