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
University of Texas System
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Ionis Pharmaceuticals Inc
University of Texas System
Isis Pharmaceuticals Inc
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    • 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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    • A—HUMAN NECESSITIES
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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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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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    • C12N2310/30—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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    • C12N2310/30—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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  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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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
US61/302,454 2010-02-08
US61/302,450 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

Publications (2)

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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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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
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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
JP2014513946A (ja) * 2011-04-22 2014-06-19 プロセンサ テクノロジーズ ビー．ブイ． 筋強直性ジストロフィー１型（ｄｍ１）などのヒト遺伝性疾患の治療、遅延及び／又は予防のための新規の化合物
US9976138B2 (en) 2011-08-29 2018-05-22 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
BR112014018427B1 (pt) 2012-01-27 2021-11-03 Biomarin Technologies B.V. Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker
US20140378533A1 (en) 2012-02-08 2014-12-25 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
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KR20160074368A (ko) 2012-05-16 2016-06-28 라나 테라퓨틱스, 인크. Utrn 발현을 조절하기 위한 조성물 및 방법
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EA201492120A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии atp2a2
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CN110257379B (zh) 2012-07-03 2023-08-11 马林生物科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
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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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TW201536329A (zh) * 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
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Cited By (3)

* 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

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

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