Hormel Institute, University of Minnesota, Austin, Minnesota, USA.
Hum Gene Ther. 2020 Oct;31(19-20):1124-1131. doi: 10.1089/hum.2020.074. Epub 2020 Jul 17.
Adeno-associated virus (AAV)-based gene therapy is undergoing major expansion into clinical practice, with two treatments currently being granted Food and Drug Administration (FDA) approval. However, the presence of pre-existing neutralizing antibodies (NAB) is one of the significant hurdles for the clinical application of AAV vectors that significantly limits the patient population, which benefits from the treatment. A reliable diagnostic to evaluate the patient's seropositivity is required to ensure the effectiveness of the AAV-mediated therapeutic. Here, we describe a simple method for the determination of AAV NAB activity based on our finding that Compound C makes HEK293 cell highly permissive for infection by 10 commonly used AAV serotypes.
腺相关病毒（AAV）为基础的基因治疗正在向临床实践中进行重大拓展，目前已有两种治疗方法获得了美国食品和药物管理局（FDA）的批准。然而，预先存在的中和抗体（NAB）的存在是 AAV 载体临床应用的一个重大障碍，这显著限制了受益于治疗的患者人群。需要一种可靠的诊断方法来评估患者的血清阳性，以确保 AAV 介导的治疗的有效性。在这里，我们描述了一种基于我们的发现的简单方法来确定 AAV NAB 活性，我们发现化合物 C 使 HEK293 细胞对 10 种常用的 AAV 血清型的感染具有高度易感性。
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