Gene Editing for the Treatment of Primary Immunodeficiency Diseases.
Gene Editing for the Treatment of Primary Immunodeficiency Diseases.
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DOI：
10.1089/hum.2020.185
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发表时间：
2021-01
影响因子：
4.2
通讯作者：
Cavazza A
中科院分区：
文献类型：
--
作者：
Rai R;Thrasher AJ;Cavazza A
由于原发性免疫缺陷疾病（PID）的常规治疗，如同种异体干细胞移植或自体基因治疗，仍然面临着重要的挑战，基因组编辑技术的快速发展，以更准确地纠正遗传性疾病发病的突变，为治疗此类疾病提供了新的替代方案，但有希望的平台。更有效和特异性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，但目前正在临床前研究中解决对基因组编辑安全性和有效性的挥之不去的担忧。本综述总结了基因编辑技术在治疗PID方面的进展，以及仍需实施的优化，以将基因组编辑转化为罕见单基因危及生命疾病的下一代治疗方法。
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.
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影响因子：
4.2
作者：
Gutierrez-Guerrero, Alejandra;Sanchez-Hernandez, Sabina;Martin, Francisco
通讯作者：
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影响因子：
17.3
作者：
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