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EA201790642A1 - Аллель-специфическая терапия для гаплотипов болезни хантингтона - Google Patents

Аллель-специфическая терапия для гаплотипов болезни хантингтона

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Publication number
EA201790642A1
EA201790642A1 EA201790642A EA201790642A EA201790642A1 EA 201790642 A1 EA201790642 A1 EA 201790642A1 EA 201790642 A EA201790642 A EA 201790642A EA 201790642 A EA201790642 A EA 201790642A EA 201790642 A1 EA201790642 A1 EA 201790642A1
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EA
Eurasian Patent Office
Prior art keywords
hantington
allel
haplotypes
disease
specific therapy
Prior art date
Application number
EA201790642A
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English (en)
Inventor
Эмбер Л. Саутвэлл
Кристофер Кей
Майкл Р. Хейден
Никола С. Карон
Original Assignee
Ти Юниверсити Оф Бритиш Коламбиа
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Application filed by Ти Юниверсити Оф Бритиш Коламбиа filed Critical Ти Юниверсити Оф Бритиш Коламбиа
Publication of EA201790642A1 publication Critical patent/EA201790642A1/ru

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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
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  • Hospice & Palliative Care (AREA)
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Abstract

Настоящее изобретение относится к композициям и способам их применения для ингибирования транскрипции мРНК мутантного НТТ или экспрессии CAG-удлиненного белка НТТ в клетке, включающим приведение в контакт клетки с эффективным количеством олигомера, нацеленного на дифференцирующий полиморфизм, при этом дифференцирующий полиморфизм выбран из rs72239206, rs363107, rs362313, rs2530595, rs113407847. Также предлагаются специфические последовательности олигомеров.
EA201790642A 2014-09-18 2015-09-18 Аллель-специфическая терапия для гаплотипов болезни хантингтона EA201790642A1 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462052282P 2014-09-18 2014-09-18
US201562191144P 2015-07-10 2015-07-10
PCT/CA2015/000501 WO2016041058A1 (en) 2014-09-18 2015-09-18 Allele-specific therapy for huntington disease haplotypes

Publications (1)

Publication Number Publication Date
EA201790642A1 true EA201790642A1 (ru) 2017-09-29

Family

ID=55532376

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201790642A EA201790642A1 (ru) 2014-09-18 2015-09-18 Аллель-специфическая терапия для гаплотипов болезни хантингтона

Country Status (9)

Country Link
US (2) US10533172B2 (ru)
EP (1) EP3194597B1 (ru)
JP (1) JP2017529082A (ru)
KR (1) KR20170058979A (ru)
AU (1) AU2015318782A1 (ru)
CA (1) CA2960728C (ru)
EA (1) EA201790642A1 (ru)
IL (1) IL251065A0 (ru)
WO (1) WO2016041058A1 (ru)

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JP2024512635A (ja) 2021-03-29 2024-03-19 アルナイラム ファーマシューティカルズ， インコーポレイテッド ハンチンチン（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法

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Also Published As

Publication number Publication date
KR20170058979A (ko) 2017-05-29
EP3194597A1 (en) 2017-07-26
US20170253877A1 (en) 2017-09-07
WO2016041058A1 (en) 2016-03-24
IL251065A0 (en) 2017-04-30
EP3194597A4 (en) 2018-05-16
US10533172B2 (en) 2020-01-14
JP2017529082A (ja) 2017-10-05
EP3194597B1 (en) 2021-06-30
US20200199595A1 (en) 2020-06-25
AU2015318782A1 (en) 2017-04-06
CA2960728C (en) 2023-06-06
CA2960728A1 (en) 2016-03-24
US10731160B2 (en) 2020-08-04

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