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 (https=
Inventor
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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    • C12N15/09—Recombinant DNA-technology
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    • 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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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • C12N2310/30—Chemical structure
    • 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
    • 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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (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
US30245410P 2010-02-08 2010-02-08
US30245010P 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

Publications (2)

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

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US (1) US20130059902A1 (https=)
EP (1) EP2536738A4 (https=)
JP (1) JP2013518603A (https=)
WO (1) WO2011097641A1 (https=)

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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
EP2595663A4 (en) 2010-07-19 2014-03-05 Isis Pharmaceuticals Inc MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION
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US9920317B2 (en) 2010-11-12 2018-03-20 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)
WO2013033223A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
NZ627896A (en) 2012-01-27 2016-11-25 Biomarin Technologies B V Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
WO2013120003A1 (en) 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
LT2841578T (lt) * 2012-04-23 2017-09-25 Biomarin Technologies B.V. Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui
AU2013262699A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating ATP2A2 expression
EA201492122A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии utrn
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
BR112014028631A2 (pt) 2012-05-16 2017-10-17 Rana Therapeutics Inc composições e métodos para modulação da expressão da família de genes da hemoglobina
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
EP2870246B1 (en) 2012-07-03 2019-09-11 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
RS54944B1 (sr) 2012-07-12 2016-11-30 Proqr Therapeutics Ii Bv Oligonukleotidi za pravljenje promena u sekvencama ciljanih rnk molekula prisutnih u živoj ćeliji
CA2917229A1 (en) * 2013-07-02 2015-01-08 Isis Pharmaceuticals, Inc. Modulators of growth hormone receptor
US10435430B2 (en) 2013-07-31 2019-10-08 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
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
EP3119888B1 (en) 2014-03-19 2021-07-28 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
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BR112016022742B1 (pt) 2014-05-01 2022-06-14 Ionis Pharmaceuticals, Inc Composto químico, composição compreendendo o composto e uso dos mesmos
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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
EP2536738A4 (en) 2014-09-17
EP2536738A1 (en) 2012-12-26
WO2011097641A1 (en) 2011-08-11

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