Spinal subpial gene delivery system
AssigneeThe Regents of the University of California
InventorMartin Marsala
Life of this patent
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Abstract
Delivery devices, systems, and methods related thereto may be used in humans for spinal delivery of cells, drugs or vectors. Thus, the system enables subpial delivery, which leads to a near complete spinal parenchymal AAV9-mediated gene expression or ASO distribution in both white and grey matter.
Claims26
A method of spinal trans-parenchymal infection of a nucleic acid molecule in a subject comprising administering a nucleic acid molecule to the subpial space of a subject.
A gene delivery system comprising:
- (a) an L-shaped guide tube configured to puncture the pia of a subject;
- (b) a catheter slidingly disposed within the guide tube and configured to be advanced into subpial space of a spinal segment of a vertebra of the subject;
- and
- (c) a reservoir in fluid communication with the catheter and containing a composition comprising a nucleic acid molecule.
A method of treating a neurodegenerative disorder in a subject in need thereof comprising administering a vector or an antisense oligonucleotide (ASO) to the subpial space of the subject.
Dependencies are inferred from the claim text.