Immunodeficiency: Gene therapy for primary immune deficiency.

Sarah Y Afzal,Matthew S MacDougall,Sean A McGhee
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Abstract

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.
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免疫缺陷:原发性免疫缺陷的基因治疗。
目前,针对先天性免疫错误的基因疗法包括使用病毒递送的基因添加方法。这种主要策略主要在严重联合免疫缺陷症、威斯科特-阿尔德里奇综合征和慢性肉芽肿病方面取得了成功。尽管基因治疗的成功率越来越高,但基因治疗也有其局限性,因此造血干细胞移植仍是首选。随着新一代慢病毒载体在病毒递送方面的改进,以及CRISPR-Cas9基因编辑技术的出现,基因治疗的有效性和安全性可能很快就会超过造血干细胞移植。此外,这些进展主要通过降低移植相关并发症的风险,提高了基因疗法治疗先天性免疫错误的可行性。因此,尽管目前存在局限性,先天性免疫错误基因疗法仍将继续向更多患者和适应症扩展。
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Real-world outcomes in patients with hereditary angioedema prescribed lanadelumab versus other prophylaxis. Protective antibody concentrations in primary immunodeficiency following infusion with 5% or 10% intravenous immunoglobulin. Immunodeficiency: Overview of primary immune regulatory disorders (PIRDs). Essentials of an immunodeficiency primer: A practical reference for the allergist/immunologist and the allergy-immunology fellow-in-training (FIT). Immunodeficiency: Gene therapy for primary immune deficiency.
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