JP2013518603A - 反復伸張に関連する疾患または病態の治療に有用な方法および組成物 - Google Patents

反復伸張に関連する疾患または病態の治療に有用な方法および組成物 Download PDF

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JP2013518603A
JP2013518603A JP2012552931A JP2012552931A JP2013518603A JP 2013518603 A JP2013518603 A JP 2013518603A JP 2012552931 A JP2012552931 A JP 2012552931A JP 2012552931 A JP2012552931 A JP 2012552931A JP 2013518603 A JP2013518603 A JP 2013518603A
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substituted
chemically modified
independently
modified oligonucleotide
nucleoside
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JP2013518603A5 (enExample
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コーリー，デヴィッド
ベネット，シー・フランク
スウェイジ，エリック・イー
ガニョン，キース
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Ionis Pharmaceuticals Inc
The University of Texas System
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Ionis Pharmaceuticals Inc
The University of Texas System
Isis Pharmaceuticals Inc
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2012552931A 2010-02-08 2011-02-08 反復伸張に関連する疾患または病態の治療に有用な方法および組成物 Pending JP2013518603A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US30245010P 2010-02-08 2010-02-08
US30245410P 2010-02-08 2010-02-08
US61/302,450 2010-02-08
US61/302,454 2010-02-08
US40515710P 2010-10-20 2010-10-20
US61/405,157 2010-10-20
PCT/US2011/024099 WO2011097641A1 (en) 2010-02-08 2011-02-08 Methods and compositions useful in treatment of diseases or conditions related to repeat expansion

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JP2013518603A true JP2013518603A (ja) 2013-05-23
JP2013518603A5 JP2013518603A5 (enExample) 2014-03-20

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US (1) US20130059902A1 (enExample)
EP (1) EP2536738A4 (enExample)
JP (1) JP2013518603A (enExample)
WO (1) WO2011097641A1 (enExample)

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US11968787B2 (en) 2012-06-22 2024-04-23 C3 Nano, Inc. Metal nanowire networks and transparent conductive material
JP2024102041A (ja) * 2016-07-05 2024-07-30 バイオマリン テクノロジーズ ベー．フェー． 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド
US12407349B2 (en) 2013-02-26 2025-09-02 Ekc Technology, Inc. Fused metal nanostructured networks, fusing solutions with reducing agents and methods for forming metal networks

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NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP3702460A1 (en) 2010-11-12 2020-09-02 The General Hospital Corporation Polycomb-associated non-coding rnas
US8569254B2 (en) 2010-12-10 2013-10-29 National Yang Ming University Methods for modulating the expression and aggregation of CAG-expanded gene product in cells and methods for identifying agents useful for doing the same
AU2012246822B2 (en) * 2011-04-22 2017-05-25 Biomarin Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (DM1)
EP2751269B1 (en) 2011-08-29 2016-03-23 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
EP3330278A1 (en) * 2012-02-08 2018-06-06 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
PT2841578T (pt) * 2012-04-23 2017-08-10 Biomarin Tech Bv Resumo
EA201492120A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии atp2a2
JP2015523854A (ja) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法
CN104583402A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节mecp2表达的组合物和方法
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
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US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
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KR20150037968A (ko) 2012-07-12 2015-04-08 프로큐알 테라퓨틱스 엔.브이. 살아있는 세포 내에 존재하는 목적 rna 분자의 서열을 변화시키기 위한 올리고뉴클레오타이드
JP6487913B2 (ja) * 2013-07-02 2019-03-20 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 成長ホルモン受容体のモジュレータ
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SI3119888T1 (sl) 2014-03-19 2021-11-30 Ionis Pharmaceuticals, Inc. Sestavki za moduliranje izražanja ataksina 2
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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11968787B2 (en) 2012-06-22 2024-04-23 C3 Nano, Inc. Metal nanowire networks and transparent conductive material
US12407349B2 (en) 2013-02-26 2025-09-02 Ekc Technology, Inc. Fused metal nanostructured networks, fusing solutions with reducing agents and methods for forming metal networks
JP2024102041A (ja) * 2016-07-05 2024-07-30 バイオマリン テクノロジーズ ベー．フェー． 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド
JP7817791B2 (ja) 2016-07-05 2026-02-19 バイオマリン テクノロジーズ ベー．フェー． 遺伝性障害の治療のための改善された特徴を有する二環式スキャフォールド部分を含むプレｍＲＮＡスプライススイッチング又は調節オリゴヌクレオチド

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US20130059902A1 (en) 2013-03-07
WO2011097641A1 (en) 2011-08-11
EP2536738A4 (en) 2014-09-17
EP2536738A1 (en) 2012-12-26

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