With conventional treatments for primary immunodeficiency diseases (PIDs), such as allogeneic stem cell transplantation or autologous gene therapy, still facing important challenges, the rapid development of genome editing technologies to more accurately correct the mutations underlying the onset of genetic disorders has provided a new alternative, yet promising platform for the treatment of such diseases. The prospect of a more efficient and specific therapeutic tool has pushed many researchers to apply these editing tools to correct genetic, phenotypic, and functional defects of numerous devastating PIDs with extremely promising results to date. Despite these achievements, lingering concerns about the safety and efficacy of genome editing are currently being addressed in preclinical studies. This review summarizes the progress made toward the development of gene editing technologies to treat PIDs and the optimizations that still need to be implemented to turn genome editing into a next-generation treatment for rare monogenic life-threatening disorders.

中文翻译：

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用于治疗原发性免疫缺陷病的基因编辑。

同种异体干细胞移植或自体基因治疗等原发性免疫缺陷病 (PID) 的常规治疗方法仍面临重大挑战，基因组编辑技术的快速发展可以更准确地纠正遗传病发病的潜在突变，这为新疗法提供了新的思路。治疗此类疾病的替代但有前途的平台。更有效和更具体的治疗工具的前景促使许多研究人员应用这些编辑工具来纠正许多破坏性 PID 的遗传、表型和功能缺陷，迄今为止取得了非常有希望的结果。尽管取得了这些成就，但临床前研究目前正在解决对基因组编辑的安全性和有效性的挥之不去的担忧。