Glutamic acid decarboxylase (gad) based delivery system
AssigneeNeurologix, Inc.
InventorsMatthew During, Michael Kaplitt
Life of this patent
Estimating the term…
Abstract
The invention provide methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain that is overstimulated in neurodegenerative diseases. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to cells in the hippocampus, subthalamic nucleus of the basal ganglia, mesaphilia and thalamus.
Claims34
-13. (canceled)
A method for treating epilepsy in a subject comprising:
- identifying one or more regions of the brain that require modification;
- delivering a vector comprising a nucleotide sequence encoding a glutamic acid decarboxylase (GAD) to the region of the brain;
- and
- expressing the GAD in the region of the brain to treat or reduce epilepsy.
A method for treating epilepsy in a subject comprising:
- identifying one or more regions of the brain that require modification;
- delivering an adeno-associated viral (AAV) vector comprising a nucleotide sequence encoding a glutamic acid decarboxylase (GAD) to the region of the brain;
- and
- expressing the GAD in the region of the brain to treat or reduce epilepsy.
A method of altering expression of glutamic acid decarboxylase (GAD) in a region of the central nervous system (CNS) of a subject with epilepsy comprising:
- identifying a target site in the CNS that requires modification;
- delivering a vector comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to the target site in the CNS;
- and
- expressing GAD in the target site.
-48. (canceled)
Dependencies are inferred from the claim text.