The emergence of adeno-associated virus (AAV)-based gene therapy has brought hope to patients with severe monogenic disorders. However, immune responses to AAV vectors and transgene products present challenges that require effective immunosuppressive strategies. This systematic review focuses on the immunosuppressive protocols used in 38 clinical trials and 35 real-world studies, considering a range of monogenic diseases, AAV serotypes, and administration routes. The review underscores the need for a deeper understanding of immunosuppressive regimens to enhance the safety and effectiveness of AAV-based gene therapy. Characterizing the immunological responses associated with various gene therapy treatments is crucial for optimizing treatment protocols and ensuring the safety and efficacy of forthcoming gene therapy interventions. Further research and understanding of the impact of immunosuppression on disease, therapy, and route of administration will contribute to the development of more effective and safer gene therapy approaches in the future.

中文翻译：

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单基因疾病 AAV 基因治疗中使用的免疫抑制方案的系统评价


基于腺相关病毒（AAV）的基因治疗的出现给患有严重单基因疾病的患者带来了希望。然而，对 AAV 载体和转基因产品的免疫反应提出了挑战，需要有效的免疫抑制策略。本系统综述重点关注 38 项临床试验和 35 项真实世界研究中使用的免疫抑制方案，考虑了一系列单基因疾病、AAV 血清型和给药途径。该综述强调需要更深入地了解免疫抑制方案，以提高基于 AAV 的基因治疗的安全性和有效性。表征与各种基因治疗相关的免疫反应对于优化治疗方案和确保即将进行的基因治疗干预措施的安全性和有效性至关重要。进一步研究和了解免疫抑制对疾病、治疗和给药途径的影响将有助于未来开发更有效、更安全的基因治疗方法。