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Nov 12, 2012

Gene Therapies Target Epilepsy

  • A pair of new gene therapy techniques may benefit people with epilepsy by targeting the part of the brain that generates seizures.

    Rob Wykes, Ph.D., a senior research associate focused on clinical and experimental epilepsy on the faculty of Brain Sciences at University College London’s Institute of Neurology, and colleagues injected extra copies of a potassium ion channel gene into a specific region of rodents’ brains. The gene allowed potassium ions to flow out of cells, reducing the number of neurons able to fire together in order for a seizure to occur. That, in turn, stopped epilepsy in the rodents, seemingly without side effects.

    Dr. Wykes and colleagues also used a lentivirus vector to deliver into rodent brains a gene expressing the light-sensitive halorhodospin protein. Using optic fibers, they shined laser light on a specific region of the brain to activate the halorhodospin and observed a decrease in electric seizure activity in the animals. Halorhodopsin pumps negatively charged chloride ions into cells when exposed to light, making it more difficult for neurons to fire.

    The study, “Optogenetic Gene Therapy in a Rodent Model of Focal Neocortical Epilepsy,” is to be published today in Science Translational Medicine.


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