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Related Concept Videos

Epilepsy and Seizures: Overview01:24

Epilepsy and Seizures: Overview

Epilepsy is a chronic neurological disease marked by recurrent, unpredictable seizures. These seizures are caused by abnormal electrical discharges in the brain, leading to behavior, sensation, or consciousness alterations. They can also cause transient impairment of awareness, interfering with daily activities.
Various factors can trigger epilepsy, including genetic factors, brain damage, metabolic causes, and unknown etiology. Diagnosis of epilepsy involves electroencephalography (EEG), which...
Antiepileptic Drugs: GABAergic Pathway Potentiators01:18

Antiepileptic Drugs: GABAergic Pathway Potentiators

γ-aminobutyric acid or GABA, plays a pivotal role as an inhibitory neurotransmitter in the brain. GABA pathway potentiators, also known as GABAergic drugs, are a class of pharmaceutical agents designed to enhance the functioning of the GABAergic system. These medications primarily treat epilepsy, a neurological disorder characterized by recurrent seizures.
The key GABA pathway potentiators used in epilepsy management are as follows.
Benzodiazepines are a well-known class of drugs used for their...
Antiepileptic Drugs: Glutamate Antagonists01:14

Antiepileptic Drugs: Glutamate Antagonists

Glutamate is a fundamental neurotransmitter in the central nervous system, playing a vital role in neuronal communication and various cognitive processes. Glutamate stands as the principal excitatory neurotransmitter in the brain. Its presence is crucial for the communication between neurons, underpinning essential processes such as synaptic transmission, neuronal excitability, and plasticity. These functions are vital for higher-order cognitive processes, including learning and memory. The...
Antiepileptic Drugs: Potassium Channel Activators01:20

Antiepileptic Drugs: Potassium Channel Activators

Ezocgabine or retigabine, an antiepileptic drug of remarkable efficacy, has revolutionized the management of seizures. It is a potassium channel activator, explicitly targeting the family of Q subtype potassium channels. It enhances the transmembrane potassium currents, regulating neuronal excitability. This action stabilizes the resting membrane potential, a pivotal factor in mitigating the hyperexcitability that characterizes epilepsy.
Ezogabine has gained approval as an adjunctive treatment...
Antiepileptic Drugs: Modulators of Neurotransmitter Release Mediated by SV2A Protein01:20

Antiepileptic Drugs: Modulators of Neurotransmitter Release Mediated by SV2A Protein

Antiepileptic drugs, such as levetiracetam (Keppra) and brivaracetam (Briviact), have emerged as crucial tools in managing epilepsy. These medications exert their therapeutic effects by targeting the synaptic vesicle protein SV2A, a transmembrane glycoprotein primarily found in the brain.
SV2A is a transmembrane glycoprotein located predominantly in the brain, modulating the release of neurotransmitters for neuronal communication. Both levetiracetam and brivaracetam exhibit a high affinity for...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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Updated: Jul 2, 2026

Electrophoretic Delivery of &#x3B3;-aminobutyric Acid (GABA) into Epileptic Focus Prevents Seizures in Mice
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Electrophoretic Delivery of γ-aminobutyric Acid (GABA) into Epileptic Focus Prevents Seizures in Mice

Published on: May 16, 2019

Gene therapy in epilepsy.

Véronique Riban1, Helen L Fitzsimons, Matthew J During

  • 1Department of Molecular Virology, The Ohio State University, Biological Research Tower, Columbus, Ohio, USA.

Epilepsia
|August 23, 2008
PubMed
Summary
This summary is machine-generated.

Gene therapy shows promise for epilepsy treatment, utilizing candidate genes like neuropeptide Y. Recombinant adeno-associated viral (rAAV) vectors offer efficient and safe delivery for potential new epilepsy therapies.

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Last Updated: Jul 2, 2026

Electrophoretic Delivery of &#x3B3;-aminobutyric Acid (GABA) into Epileptic Focus Prevents Seizures in Mice
07:01

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Preparation and Implantation of Electrodes for Electrically Kindling VGAT-Cre Mice to Generate a Model for Temporal Lobe Epilepsy
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Preparation and Implantation of Electrodes for Electrically Kindling VGAT-Cre Mice to Generate a Model for Temporal Lobe Epilepsy

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Area of Science:

  • Neuroscience
  • Gene Therapy
  • Epilepsy Research

Background:

  • Epilepsy treatment remains a challenge, necessitating novel therapeutic strategies.
  • Animal models indicate gene therapy's potential for managing seizure activity.
  • Key candidate genes like neuropeptide Y and galanin show therapeutic promise.

Purpose of the Study:

  • To evaluate the potential of gene therapy for epilepsy treatment.
  • To highlight the importance of efficient transgene delivery to target neurons.
  • To discuss the suitability of recombinant adeno-associated viral (rAAV) vectors for neurological gene therapy.

Main Methods:

  • Review of preclinical studies on candidate genes (neuropeptide Y, galanin) in animal models.
  • Examination of gene delivery advancements, including cell transplantation and viral vectors.
  • Focus on recombinant adeno-associated viral (rAAV) vector characteristics for neurological applications.

Main Results:

  • Preclinical studies demonstrate positive effects of candidate genes on seizure activity.
  • Recombinant adeno-associated viral (rAAV) vectors exhibit neuronal tropism, low toxicity, and stable transgene expression.
  • rAAV vectors have shown a favorable safety profile in Phase I clinical trials for Parkinson's disease.

Conclusions:

  • Gene therapy, particularly using rAAV vectors, is a promising approach for epilepsy treatment.
  • Further preclinical evaluation in chronic epilepsy models and toxicological studies are necessary before human trials.
  • Efficient and safe gene delivery is critical for successful gene therapy interventions in epilepsy.