Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna
AssigneeMedtronic, Inc.
InventorWilliam Kaemmerer
Life of this patent
Estimating the term…
Abstract
The present invention provides devices, small interfering RNAs, 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, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient.
Claims31
A medical system for treatment of Huntington's disease, comprising:
- a. an intracranial access device;
- b. a mapping means locating a predetermined location in the brain, said predetermined location comprising at least one cell expressing huntingtin;
- c. a deliverable amount of a small interfering RNA, said small interfering RNA comprising a first strand comprising at least 19 contiguous nucleotides encoded by the group consisting of SEQ. ID. NO:
- 24 or SEQ. ID. NO:
- 25, or a vector encoding said small interfering RNA.
- d. a delivery means for delivering said small interfering RNA to said location of the brain from said intracranial access device.
A method of treating Huntington's disease in a patient comprising:
- a. locating a predetermined location in the brain, said predetermined location comprising at least one cell expressing huntingtin;
- b. positioning an intracranial access delivery device to provide access to the predetermined location in the brain;
- c. infusing a small interfering RNA a small interfering RNA, said small interfering RNA comprising a first strand comprising at least 19 contiguous nucleotides encoded by the group consisting of SEQ. ID. NO:
- 24 or SEQ. ID. NO:
- 25, or a vector encoding said small interfering RNA.
- wherein at least one attribute of Huntington's disease is reduced or its progression slowed or arrested.
Dependencies are inferred from the claim text.