Treatment of neurodegenerative disease through intracranial delivery of sirna
AssigneeMedtronic, Inc.
InventorWilliam F. Kaemmerer
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Abstract
The present invention provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, and methods for treating neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease, Huntington's disease, Spinocerebellar Ataxia Type 1, Type 2, Type 3, and/or dentatorubral-pallidoluysian atrophy.
Claims84
A medical system for treating a neurodegenerative disorder comprising:
- a. an intracranial access device;
- b. a mapping means for locating a predetermined location in the brain;
- c. a deliverable amount of a small interfering RNA or vector encoding said small interfering RNA;
- and
- d. a delivery means for delivering said small interfering RNA or vector encoding said small interfering RNA to said location of the brain from said intracranial access device.
A method for treating a neurodegenerative disorder comprised of modulating the expression or production of a protein in neurons by intracranial delivery of a small interfering RNA that reduces said expression of production of said protein, in a pharmaceutically acceptable carrier.
A method of delivering a small interfering RNA to a location in the brain comprising the steps of:
- a. surgically implanting an intracranial access delivery device;
- and
- b. infusing a small interfering RNA and/or a vector encoding said small interfering RNA at a predetermined site in the brain.
A method of delivering a small interfering RNA to a location in the brain comprising the steps of:
- a. surgically implanting an intracranial access delivery device;
- and
- b. infusing a small interfering RNA and/or a vector encoding said small interfering RNA at a predetermined site in the brain;
- wherein at least one attribute of said neurodegenerative diseases is reduced or its progression slowed or arrested.
A small interfering RNA containing sequences according to SEQ ID Nos 1-4, or a partial sequence thereof, or a base sequence hybridizable to a complementary strand of RNA encoding a protein associated with a neurodegenerative disease.
A small interfering RNA comprising an RNA sequence hybridizable to the RNA sequence encoding a protein associated with a neurodegenerative disease to cause cleavage of said protein-encoding RNA sequence.
A small interfering RNA expression sequence comprising the DNA sequence encoding an RNA sequence hybridizable to the RNA sequence encoding a protein associated with a neurodegenerative disease to cause cleavage of said protein-encoding RNA sequence.
Dependencies are inferred from the claim text.