Allergy Asthma Proc. 2024 Sep 1;45(5):384-388. doi: 10.2500/aap.2024.45.240054.
Current gene therapy for inborn errors of immunity have involved the use of gene addition approaches with viral delivery. This main strategy has had demonstrated success mainly in severe combined immune deficiency, Wiskott-Aldrich syndrome, and chronic granulomatous disease. Despite the increasing success of gene therapy, there are limitations of gene therapy, and, therefore, hematopoietic stem cell transplantation continues to be the preferred option. With improvements in viral delivery through next-generation lentiviral vectors and the advent of gene editing with CRISPR-Cas9, the efficacy and safety of gene therapy may soon surpass hematopoietic stem cell transplantation. Furthermore, these advances improve the viability of gene therapy for inborn errors of immunity primarily through decreased risk of transplantation-related complications. Therefore, despite current limitations, gene therapy for inborn errors of immunity is poised to continue to expand to more patients and indications.
目前针对先天性免疫缺陷的基因治疗涉及使用病毒载体的基因添加方法。这种主要策略在严重联合免疫缺陷、Wiskott-Aldrich 综合征和慢性肉芽肿病中已被证明是成功的。尽管基因治疗的成功率不断提高，但仍存在一些局限性，因此造血干细胞移植仍然是首选。随着下一代慢病毒载体在病毒传递方面的改进以及 CRISPR-Cas9 基因编辑的出现，基因治疗的疗效和安全性可能很快超过造血干细胞移植。此外，这些进展通过降低与移植相关的并发症风险，提高了基因治疗对先天性免疫缺陷的可行性。因此，尽管存在当前的局限性，针对先天性免疫缺陷的基因治疗有望继续扩展到更多的患者和适应症。
