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Recent advances and current status of gene therapy for epilepsy
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Recent advances and current status of gene therapy for epilepsy
Background Epilepsy is a common neurological disorder with complex pathogenic mechanisms,and refractory epilepsy often lacks effective treatments.Gene therapy is a promising therapeutic option,with various preclinical experiments achieving positive results,some of which have progressed to clinical studies.Data sources This narrative review was conducted by searching for papers published in PubMed/MEDLINE with the fol-lowing single and/or combination keywords:epilepsy,children,neurodevelopmental disorders,genetics,gene therapy,vectors,transgenes,receptors,ion channels,micro RNAs(miRNAs),clustered regularly interspaced short palindromic repeats(CRISPR)/CRISPR-associated protein(Cas)9(CRISPR/Cas9),expression regulation,optogenetics,chemical genet-ics,mitochondrial epilepsy,challenges,ethics,and disease models.Results Currently,gene therapy research in epilepsy primarily focuses on symptoms attenuation mediated by viral vectors such as adeno-associated virus and other types.Advances in gene therapy technologies,such as CRISPR/Cas9,have provided a new direction for epilepsy treatment.However,the clinical application still faces several challenges,including issues related to vectors,models,expression controllability,and ethical considerations.Conclusions Here,we summarize the relevant research and clinical advances in gene therapy for epilepsy and outline the challenges facing its clinical application.In addition to the shortcomings inherent in gene therapy components,the recon-figuration of excitatory and inhibitory properties in epilepsy treatment is a delicate process.On-demand,cell-autonomous treatments and multidisciplinary collaborations may be crucial in addressing these issues.Understanding gene therapy for epilepsy will help clinicians gain a clearer perception of the research progress and challenges,guiding the design of future clinical protocols and research decisions.
ChallengesCRISPREpilepsyGene therapyStrategy
Ao-Jie Cai、Kai Gao、Fan Zhang、Yu-Wu Jiang
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Department of Pediatrics,Peking University First Hospital,Beijing,China
Department of Pediatrics,The First Affiliated Hospital of Zhengzhou University,Henan Province,Zhengzhou 450052,China
Beijing Key Laboratory of Molecular Diagnosis and Study on Pediatric Genetic Diseases,Beijing,China
Children Epilepsy Center,Peking University First Hospital,Beijing,China
Key Laboratory for Neuroscience,Ministry of Education/National Health and Family Planning Commission,Peking University,Beijing,China
Center of Epilepsy,Beijing Institute for Brain Disorders,Beijing,China
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