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Life sciences · Journal article
Molecular Medicine Reports · November 3, 2026
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Genetic epilepsy, for which the molecular mechanisms are relatively well defined in numerous monogenic forms, represents an attractive model for the transition from symptomatic seizure control to mechanism-driven correction.However, gene therapy strategies differ in their mechanisms of action, adaptability to specific mutations and translational maturity, making rational selection of interventions based on variant-specific functional consequences an important clinical challenge.The present review examined five nucleic acid-based therapeutic strategies, namely gene supplementation, enhancement of endogenous gene expression, gene silencing, dna editing and rna editing, for major causative genes, using mutation-specific functional consequences [loss-of-function (loF) vs. gain-of-function] as the primary framework for therapeutic decision-making.The mechanisms and suitability of each approach are analysed, and their performance in terms of cell-type specificity, delivery vectors and safety is evaluated by integrating evidence from animal models, induced pluripotent stem cell-derived neurons and organoids and clinical trials.The present review also summarised key translational barriers, including vector cargo constraints, blood-brain barrier penetration, off-target and immune-associated risks, therapeutic dosing windows and the timing of developmental intervention.antisense oligonucleotides and regulatory adeno-associated viral approaches, supported by mature delivery platforms and manageable safety profiles, have progressed furthest towards clinical validation.endogenous gene activation strategies, such as criSPr activation, offer a gene replacement-free approach for loF disorders but remain preclinical.dna and rna editing, while potentially capable of durable disease modification, require further evidence on delivery efficiency and long-term homeostatic safety.Gene therapy for genetic epilepsy is undergoing a key transition from proof-of-concept studies to individualised clinical application.Future progress will depend on integrating personalised intervention design, multi-target therapeutic combinations, artificial intelligence-assisted sequence optimisation and standardised translational models, with the potential to drive a paradigm shift from seizure suppression to mechanistic correction.Contents 1. introduction 2. Therapeutic strategies oriented to loF mutations: expression enhancement 3. Therapeutic strategies oriented towards GoF mutations: Gene silencing 4. Precision editing strategies: dna editing and rna editing 5. Translational research progress in representative epilepsy genes 6. challenges and limitations 7. Future directions 8. conclusions