MODULATION OF HUNTINGTIN EXPRESSION

PROBLEM TO BE SOLVED: To provide methods for modulating expression of huntingtin and treating, preventing, delaying or mitigating Huntington's disease and/or a symptom thereof.SOLUTION: A method for reducing expression of huntingtin mRNA and protein in an animal (human) comprises administering...

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Main Authors BENNETT C FRANK, LAMYA SHIHABUDDIN, SENG H CHENG, GENE HUNG, SUSAN M FREIER, HOLLY KORDASIEWICZ, LISA STANEK, DON W CLEVELAND
Format Patent
LanguageEnglish
Japanese
Published 12.10.2017
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
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Summary:PROBLEM TO BE SOLVED: To provide methods for modulating expression of huntingtin and treating, preventing, delaying or mitigating Huntington's disease and/or a symptom thereof.SOLUTION: A method for reducing expression of huntingtin mRNA and protein in an animal (human) comprises administering a compound or composition comprising a modified oligonucleotide consisting of 12 to 30 linked nucleosides comprising at least 8 contiguous nucleobases of a specific sequence.SELECTED DRAWING: None 【課題】ハンチンチンの発現を修飾し、そしてハンチントン病および／またはその症状を治療し、予防し、遅らせ、または軽減するための方法の提供。【解決手段】特定の配列の少なくとも８個の隣接した核酸塩基を含む、１２個〜３０個の連結した連結したヌクレオシドからなる修飾されたオリゴヌクレオチドを含む化合物または組成物を投与して、動物（ヒト）におけるハンチンチンｍＲＮＡまたはタンパク質の発現を減少させるための方法。【選択図】なし
Bibliography:Application Number: JP20170106418