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

Bo SUN; Xiao-Le WANG; Jing PENG

doi:10.7499/j.issn.1008-8830.2507145

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Chinese Journal of Contemporary Pediatrics ›› 2026, Vol. 28 ›› Issue (3) : 358-364. DOI: 10.7499/j.issn.1008-8830.2507145

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

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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.

Key words

Genetic epilepsy / Adeno-associated virus / Gene therapy

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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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