腺相关病毒介导的基因治疗在遗传性癫痫中的应用前景与挑战

孙博; 王晓乐; 彭镜

doi:10.7499/j.issn.1008-8830.2507145

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中国当代儿科杂志 ›› 2026, Vol. 28 ›› Issue (3) : 358-364. DOI: 10.7499/j.issn.1008-8830.2507145

综述

腺相关病毒介导的基因治疗在遗传性癫痫中的应用前景与挑战

孙博 ,
王晓乐 (综述) ,
彭镜 (审校)

作者信息 +

Adeno-associated virus-mediated gene therapy for genetic epilepsy: prospects and challenges

Author information +

文章历史 +

摘要

随着抗癫痫发作药物不断研发上市，部分遗传性癫痫已有精准治疗，但仍有多数遗传性癫痫患者为药物难治性癫痫，严重影响患者的生活质量及生命健康。基因治疗技术的发展改变了遗传性疾病的传统诊疗模式并提高了该疾病的诊断率，为患者带来了新的希望。腺相关病毒是基因治疗的“明星载体”，在基因治疗中应用广泛。该文旨在对腺相关病毒介导的基因治疗在遗传性癫痫中的应用进行综述，以期为临床提供参考。

Abstract

With the development and launch of anti-seizure medications, some genetic epilepsies have precision treatments; however, many patients remain drug-resistant, which severely affects quality of life and overall health. Advances in gene therapy have changed traditional diagnostic and therapeutic approaches for hereditary diseases, improved diagnostic accuracy, and brought new hope to patients. Adeno-associated virus is a leading vector in gene therapy and is widely used. This review summarizes the application prospects and challenges of adeno-associated virus-mediated gene therapy for genetic epilepsy to inform clinical practice.

导出引用

孙博, 王晓乐, 彭镜. 腺相关病毒介导的基因治疗在遗传性癫痫中的应用前景与挑战[J]. 中国当代儿科杂志. 2026, 28(3): 358-364 https://doi.org/10.7499/j.issn.1008-8830.2507145

Bo SUN, Xiao-Le WANG, Jing PENG. Adeno-associated virus-mediated gene therapy for genetic epilepsy: prospects and challenges[J]. Chinese Journal of Contemporary Pediatrics. 2026, 28(3): 358-364 https://doi.org/10.7499/j.issn.1008-8830.2507145

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