[미국특허]
Devices, systems and methods for improving memory and/or cognitive function through brain delivery of siRNA
원문보기
IPC분류정보
국가/구분
United States(US) Patent
등록
국제특허분류(IPC7판)
C07H-021/02
C07H-021/04
A61K-031/70
C12Q-001/68
출원번호
US-0575752
(2009-10-08)
등록번호
US-8618069
(2013-12-31)
발명자
/ 주소
Kaemmerer, William F
출원인 / 주소
Medtronic, Inc.
인용정보
피인용 횟수 :
0인용 특허 :
56
초록▼
The present invention relates to devices, systems, and methods for improving memory and/or cognitive function by brain delivery of compositions of small interfering RNA or vectors containing the DNA encoding for small interfering RNA. Such compositions can be administered using devices, systems and
The present invention relates to devices, systems, and methods for improving memory and/or cognitive function by brain delivery of compositions of small interfering RNA or vectors containing the DNA encoding for small interfering RNA. Such compositions can be administered using devices, systems and methods for direct delivery of the compositions to the brain, or using devices, systems, methods of delivery, and compositions that deliver small interfering RNA or vectors containing the DNA encoding the small interfering RNA across the blood-brain barrier. The present invention also provides valuable small interfering RNA vectors, and methods for reduction of BACE1 levels in the hippocampus, cerebral cortex, or other regions of the brain that have beneficial effects on improving memory and/or cognitive function in a subject.
대표청구항▼
1. A medical system for improving memory or cognitive function in a subject 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 selected fro
1. A medical system for improving memory or cognitive function in a subject 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 selected from one or more sequences coded from SEQ ID NOS:24-40; andd. 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. 2. The medical system of claim 1, wherein said intracranial access device is selected from the group consisting of an intracranial catheter, an intracranial access port, an infusion pump an electromechanical pump, and an osmotic pump. 3. The medical system of claim 1, wherein the predetermined location is the nucleus basalis of Meynert or the cerebral cortex or the hippocampus. 4. The medical system of claim 1, wherein the delivery means is injection from an external syringe into an intracranial access port. 5. A double-stranded RNA molecule, having a first strand and a second strand, wherein the first and the second strand each is between 19 and about 30 nucleotides long, and wherein the first strand comprises a sequence selected from the group consisting of SEQ ID NO: 28, SEQ ID NO: 29, and SEQ ID NO: 30. 6. The double-stranded RNA molecule of claim 5 wherein the first strand and the second strand are connected by means of a loop. 7. The double-stranded RNA of claim 5, comprising one or more modified nucleotides. 8. A vector comprising a nucleotide sequence encoding the double-stranded RNA molecule of claim 5. 9. The vector of claim 8, further comprising a promoter. 10. The vector of claim 9, wherein the promoter is a polIII promoter. 11. The vector of claim 8, which is a viral vector. 12. The vector of claim 11, which is an adeno-associated viral vector. 13. A double-stranded cassette comprising SEQ ID NOs 37-40 and encoding a short hairpin RNA. 14. A vector comprising a nucleic acid sequence encoding the double-stranded cassette of claim 13. 15. The vector of claim 14, further comprising a promoter. 16. The vector of claim 15, wherein the promoter is a polIII promoter. 17. The vector of claim 14, which is a viral vector. 18. A method of decreasing the amount of BACE-1 mRNA comprising intracranially administering to the subject in need thereof a nucleic acid sequence comprising the double-stranded RNA molecule of claim 5. 19. The method of claim 18, wherein the double-stranded RNA molecule is included within a vector. 20. The method of claim 19, wherein said vector is a viral vector. 21. The method of claim 18, wherein the first and the second strands of the double-stranded RNA molecule are connected by means of a loop. 22. The method of claim 18, wherein the double-stranded RNA molecule comprises one or more modified nucleotides. 23. A method of decreasing the amount of BACE-1 mRNA comprising intracranially administering to the subject in need thereof a double-stranded cassette of claim 13. 24. The method of claim 23, wherein the double-stranded cassette is included within a vector. 25. The method of claim 24, wherein the vector is a viral vector.
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