Gene therapy is a promising approach for treating genetic disorders by delivering therapeutic genes to replace or correct malfunctioning genes. However, the introduced gene therapy vector can trigger an immune response, leading to reduced efficacy and potential harm to the patient. To improve the efficiency and safety of gene therapy, preventing the immune response to the vector is crucial. This can be achieved through the use of immunosuppressive drugs, vector engineering to evade the immune system, or delivery methods that bypass the immune system altogether. By reducing the immune response, gene therapy can deliver therapeutic genes more effectively and potentially cure genetic diseases. In this study, a novel molecular imprinting technique, combined with mass-spectrometry and bioinformatics, was used to identify four antigen-binding fragments (Fab) sequences of Adeno-Associated Virus (AAV) - neutralising antibodies capable of binding to AAV. The identified Fab peptides were shown to prevent AAV8's binding to antibodies, demonstrating their potential to improve gene therapy efficiency by preventing the immune response.

基因治疗是一种很有前途的治疗遗传性疾病的方法，通过提供治疗性基因来替换或纠正故障基因。然而，引入的基因治疗载体可能会引发免疫反应，导致疗效降低并对患者造成潜在伤害。为了提高基因治疗的效率和安全性，防止对载体的免疫反应至关重要。这可以通过使用免疫抑制药物、逃避免疫系统的载体工程或完全绕过免疫系统的递送方法来实现。通过减少免疫反应，基因疗法可以更有效地传递治疗基因，并有可能治愈遗传性疾病。在这项研究中，一种新颖的分子印迹技术结合了质谱和生物信息学，用于鉴定腺相关病毒 (AAV) 的四个抗原结合片段 (Fab) 序列 - 能够与 AAV 结合的中和抗体。已鉴定的 Fab 肽被证明可以阻止 AAV8 与抗体的结合，这证明了它们通过阻止免疫反应来提高基因治疗效率的潜力。