US2024009324A1PendingUtilityA1

Using dreadd for neuronal modulation in treating neuronal diseases

Assignee: ASSAF FADIPriority: Mar 9, 2016Filed: Sep 19, 2023Published: Jan 11, 2024
Est. expiryMar 9, 2036(~9.6 yrs left)· nominal 20-yr term from priority
A61K 48/0016C07K 14/705A61P 25/14A61K 48/005A61K 31/5513A61P 25/16A61K 48/0058A61K 48/0075C12N 15/113C12N 15/62C12N 15/8509A61K 38/00C12N 2750/14143
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Claims

Abstract

A method for treating a patient suffering from a neuronal hypo-kinetic disease or a neuronal hyper-kinetic disease by modulating neuronal activity in the: internal globus pallidus (GPi), in the anterior motor thalamus and/or in the external globus pallidum (GPe) and/or in the subthalamic nucleus (STN) by utilizing suppressor and/or enhancer DREADDs is provided.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A method for treating a subject afflicted with a neuronal hyper-kinetic disease or disorder, comprising:
 (a) enhancing neuronal activity in the internal globus pallidus (GPi); and   (b) suppressing neuronal activity in the: anterior motor thalamus, external globus  pallidum  (GPe), the subthalamic nucleus (STN), or any combination thereof, wherein said enhancing neuronal activity comprises transfecting said GPi neuron with an excitatory DREADD and activating said excitatory DREADD, wherein said suppressing neuronal activity comprises transfecting a neuron in: the anterior motor thalamus, the external globus  pallidum  (GPe), or the subthalamic nucleus (STN) with an inhibitory DREADD and activating said inhibitory DREADD, thereby treating a subject afflicted with a neuronal hyper-kinetic disease or disorder.   
     
     
         2 . The method of  claim 1 , wherein said transfecting said GPi neuron with an excitatory DREADD is injecting AAV viral vector comprising the: Gq DREAD gene, Gs DREAD gene, or both. 
     
     
         3 . The method of  claim 1 , wherein said transfecting a neuron in: the anterior motor thalamus, the external globus  pallidum  (GPe), or the subthalamic nucleus (STN) with an inhibitory DREADD is injecting AAV viral vector comprising the Gi DREAD gene. 
     
     
         4 . The method of  claim 1 , wherein said enhancing neuronal activity in the internal globus pallidus (GPi) and said suppressing neuronal activity in: the anterior motor thalamus, the external globus  pallidum  (GPe), or the subthalamic nucleus (STN) are preformed concomitantly. 
     
     
         5 . The method of  claim 1 , wherein said activating said inhibitory DREADD and activating said excitatory DREADD is achieved by a single ligand. 
     
     
         6 . The method of  claim 1 , wherein said activating said inhibitory DREADD, activating said excitatory DREADD, or both is contacting: a GPi neuron, an anterior motor thalamus neuron, a GPe neuron, a STN neuron or any combination thereof with CNO. 
     
     
         7 . The method of  claim 1 , wherein said treating a subject afflicted with a neuronal hyper-kinetic disease or disorder is improving motor activities in said subject. 
     
     
         8 . The method of  claim 1 , wherein said treating a subject afflicted with a neuronal hyper-kinetic disease or disorder is alleviating non-motor symptoms. 
     
     
         9 . The method of  claim 1 , wherein said treating a subject afflicted with a neuronal hyper-kinetic disease or disorder is without inducing permanent damage to the brain. 
     
     
         10 . The method of  claim 1 , wherein said neuronal hyper-kinetic disease or disorder is chorea, dystonia, tick-disorder, Tourette syndrome, or any combination thereof.

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