Looking for breakthrough ideas for innovation challenges? Try Patsnap Eureka!

Methods and sequences to suppress primate huntington gene expression

a technology of primate huntington and gene expression, applied in the field of inhibitory nucleic acid molecules, can solve the problems of slow destruction of the affected individual's ability to walk, think, talk and reason, and reduce the cell's ability to create the protein for which it encodes, so as to suppress the expression of the hd gene, and reduce the level of huntingtin within the cell

Inactive Publication Date: 2006-11-16
MEDTRONIC INC
View PDF13 Cites 85 Cited by
  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

The present invention describes methods, nucleic acid sequences, and molecules that use RNA interference to suppress the expression of the HD gene and reduce the levels of huntingtin protein in cells. This suppression can be useful in studying HD pathogenesis and preventing or treating symptoms of the disease. The invention includes nucleic acid duplexes that target specific parts of the HD gene and can be introduced into cells to reduce huntingtin protein levels. Overall, the invention provides a tool for research and potential treatment for HD.

Problems solved by technology

It slowly destroys an affected individual's ability to walk, think, talk and reason.
If the mRNA is degraded quickly within the cell (such as before it reaches a ribosome), it is unable to serve as a template for new protein translation, thus reducing the cell's ability to create the protein for which it encoded.

Method used

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
View more

Image

Smart Image Click on the blue labels to locate them in the text.
Viewing Examples
Smart Image
  • Methods and sequences to suppress primate huntington gene expression
  • Methods and sequences to suppress primate huntington gene expression
  • Methods and sequences to suppress primate huntington gene expression

Examples

Experimental program
Comparison scheme
Effect test

##ic example 1

PROPHETIC EXAMPLE 1

Pre-Clinical Test of the Safety of Suppressing Huntingtin Protein Expression in the Rhesus Monkey Brain using shNA of the Invention

[0089] A pre-clinical study of the safety of suppressing huntingtin protein expression in the normal adult rhesus monkey brain is conducted over a 12-month period. During the first three months of the study, six rhesus macaques (Macaca mulatta) are trained to perform the tasks of a commercially available computerized behavioral test battery (Monkey CANTAB, Model 80650, Lafayette Instruments, Lafayette, Ind.) that is based upon human neuropsychological tests (CANTAB, CeNeS, Cambridge, UK). The performance of these six monkeys on the test battery is compared to previously published performance norms for rhesus monkeys to verify that the monkey's cognitive abilities are within normal limits. See, for example, Weed et al., Brain Research: Cognitive Brain Research, Oct. 25, 1999; 8(3) 185-201.

[0090] Next, the monkeys are randomly assigned...

##ic example 2

PROPHETIC EXAMPLE 2

Treatment of Huntington's Disease (“HD”) in Human Patients using shNA of the Invention

[0093] Once the safety of suppressing huntingtin protein in the caudate and putamen of the adult primate brain is established in rhesus monkeys, the same shNA expression cassette that is used in the pre-clinical safety tests in rhesus monkeys is engineered into an AAV vector using methods well-known to those skilled in the art. This AAV vector comprises an expression cassette containing an RNA polymerase III promoter driving expression of a short hairpin RNA transcript encoding for the shNA of SEQ ID NO: 17. The AAV vector optionally does not contain a second expression cassette encoding for a green fluorescent protein reporter gene, because this expression cassette is not needed for the treatment of HD in patients.

[0094] It is to be especially understood that because the shNA sequence encoded by SEQ ID NO:17 contains the siNA target region of SEQ ID NO: 4, and the sequence of ...

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to View More

PUM

PropertyMeasurementUnit
thickaaaaaaaaaa
lengthaaaaaaaaaa
nucleic acidaaaaaaaaaa
Login to View More

Abstract

Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatta and Homo sapiens. These sequences, molecules and methods aid in the study of the pathogenesis of HD and can also provide a treatment for this disease.

Description

CROSS REFERENCE TO RELATED APPLICATIONS [0001] This application claims priority under 35 U.S.C. §119(e) to U.S. Provisional Patent Application No. 60 / 678,729 filed May 6, 2005.FIELD OF THE INVENTION [0002] The present invention relates to inhibitory nucleic acid molecules that suppress the expression of the Huntington's disease gene in primates, including rhesus monkeys (Macaca mulatta) and humans (Homo sapiens). BACKGROUND OF THE INVENTION [0003] Huntington's disease (“HD”) is a neurodegenerative brain disorder with a juvenile or adult onset. It slowly destroys an affected individual's ability to walk, think, talk and reason. Symptoms include changes in cognitive ability, such as impaired short-term memory and a decreased ability to concentrate; changes in mood, such as the development of mood swings, depression and irritability; and changes in coordination and physical movement such as clumsiness, involuntary movements and twitching. These symptoms gradually worsen until HD patien...

Claims

the structure of the environmentally friendly knitted fabric provided by the present invention; figure 2 Flow chart of the yarn wrapping machine for environmentally friendly knitted fabrics and storage devices; image 3 Is the parameter map of the yarn covering machine
Login to View More

Application Information

Patent Timeline
no application Login to View More
Patent Type & Authority Applications(United States)
IPC IPC(8): A61K48/00C12Q1/68C07H21/04C12P21/06C07K14/705C12N15/113
CPCC12N2310/14C12N15/113
Inventor KAEMMERER, WILLIAMKAYTOR, MICHAEL
Owner MEDTRONIC INC
Who we serve
  • R&D Engineer
  • R&D Manager
  • IP Professional
Why Patsnap Eureka
  • Industry Leading Data Capabilities
  • Powerful AI technology
  • Patent DNA Extraction
Social media
Patsnap Eureka Blog
Learn More
PatSnap group products

Browse by: Latest US Patents, China's latest patents, Technical Efficacy Thesaurus, Application Domain, Technology Topic, Popular Technical Reports.

© 2024 PatSnap. All rights reserved.Legal|Privacy policy|Modern Slavery Act Transparency Statement|Sitemap|About US| Contact US: help@patsnap.com