Gene Therapy To Treat Epilepsy With Dr. Eric Wengert

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July 21, 2022

In this Teach Me in 10 episode, we are joined by Dr. Eric Wengert, postdoctoral fellow at The Children’s Hospital of Philadelphia. Dr. Wengert will be discussing epilepsy, the neurological disorder caused by hyperexcitability and synchrony in the brain, which manifest as behavioral seizures. While there are antiseizure medications available, many patients become refractory to available treatments and even those in which seizures are controlled have to deal with significant adverse side effects. For this reason, scientists are actively investigating new ways to better prevent seizures with fewer and less severe side effects. Gene therapy, which seeks to modify brain activity by altering gene expression directly rather than protein function, represents a more targeted way of intervening in the brain. One of these approaches, Targeted Augmentation of Nuclear Gene Output (TANGO), seeks to boost gene expression of the SCN1A gene to treat a severe genetic epilepsy syndrome called Dravet Syndrome. In a mouse model of Dravet Syndrome, a single treatment with TANGO rescues the impaired excitability of inhibitory interneurons, reduces seizure frequency and prolongs survival. Gene therapy approaches like TANGO hold great potential for treating epilepsy and improving the lives of patients, as Dr. Wengert explores in this video.

Drug DiscoveryNeurosciencePersonalized Medicine

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