Targeted Gene and Genome-Editing Strategies for Epilepsy: Experimental Advances and Translational Challenges

Area of Science:

• Neuroscience
• Genetics
• Biotechnology

Background:

• Epilepsy impacts over 50 million globally, with a significant portion unresponsive to standard treatments.
• Drug-resistant epilepsy presents a major unmet clinical need, driving research into novel therapeutic strategies.

Purpose of the Study:

• To review current gene therapy and genome-editing approaches for epilepsy.
• To highlight technological and biological challenges in translating these therapies to clinical practice.
• To discuss emerging strategies for durable, targeted epilepsy treatments.

Main Methods:

• Review of recent advancements in gene therapy and genome editing for central nervous system disorders.
• Analysis of preclinical models utilizing viral and non-viral delivery systems.
• Examination of CRISPR-derived technologies like transcriptional modulation, base editing, and prime editing.

Main Results:

• Gene therapy strategies include gene restoration (e.g., SCN1A for Dravet syndrome) and modulation of neuronal excitability.
• Genome editing platforms enable precise gene regulation in neurons without DNA breaks.
• Progress in delivery systems and editing tools facilitates manipulation of neuronal and glial function.

Conclusions:

• Gene therapy and genome editing show promise for treating drug-resistant epilepsies.
• Efficient, cell-specific delivery and long-term safety are critical translational hurdles.
• Emerging strategies are paving the way for targeted and durable epilepsy interventions.