Title:Gene Therapy Repairs for the Epileptic Brain: Potential for Treatment and Future Directions
Volume: 19
Issue: 6
关键词:
通道病，癫痫，基因治疗，神经保护，癫痫持续状态，颞叶癫痫，非病毒载体的递送，病毒载体的递送。
摘要: Epilepsy is a syndrome specified by frequent seizures and is one of the most prevalent neurological
conditions, and that one-third of people of epilepsy are resistant to available drugs. Surgery
is supposed to be the main treatment for the remedy of multiple drug-resistant epilepsy, but it is a
drastic procedure. Advancement in genomic technologies indicates that gene therapy can make such
surgery unnecessary. The considerable number of new studies show the significance of mutation in
mammalian target of rapamycin pathway, NMDA receptors, GABA receptors, potassium channels and
G-protein coupled receptors. Illustration of the meticulous drug in epilepsy targeting new expression
of mutations in SCN8A, GRIN2A, GRIN2D and KCNT1 are conferred. Various methods are utilized
to express a gene in a precise area of the brain; Transplantation of cells in an ex vivo approach (fetal
cells, fibroblasts, immortalized cells), nonviral vector delivery and viral vector delivery like retrovirus,
herpes simplex virus adenovirus and adeno-related virus. Gene therapy has thus been explored to generate
anti-epileptogenic, anti-seizure and disease-modifying effects. Specific targeting of the epileptogenic
region is facilitated by gene therapy, hence sparing the adjacent healthy tissue and decreasing
the adverse effects that frequently go hand in hand with antiepileptic medication.
