COMBINED USE OF A VECTOR ENCODING A MODIFIED RECEPTOR AND ITS EXOGENOUS AGONIST IN THE TREATMENT OF SEIZURES

摘要

The invention provides methods and materials for treating a seizure disorder such as epilepsy in a patient which employ a vector encoding a modified receptor, the so-called “DREADD” receptor being characterised by (i) a decreased responsiveness to its endogenous activating ligand (ii) a retained or enhanced responsiveness to an exogenous agonist. The modified receptor is expressed in neurons of a seizure focus in brain of the patient, and an exogenous agonist is administered which activates the modified receptor to reversibly alters the excitability of the neurons in the seizure focus leading to synaptic silencing or other inhibition.

主权项

1. A method of treating a seizure disorder in a patient suffering from said disorder, which method comprises:
(a) administering to said patient a vector encoding a modified receptor, the modified receptor being characterised by (i) a decreased responsiveness to its endogenous activating ligand (ii) a retained or enhanced responsiveness to an exogenous agonist,
wherein said modified receptor is expressed neurons of a seizure focus in brain of the patient; (b) administering to said patient said exogenous agonist, whereby the presence of said agonist in the brain of the patient activates said modified receptor, whereby activation of said modified receptor reversibly alters the excitability of the neurons in the seizure focus.