JP2014514921A - 機能性核酸分子、及びその利用 - Google Patents

機能性核酸分子、及びその利用 Download PDF

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JP2014514921A
JP2014514921A JP2014501947A JP2014501947A JP2014514921A JP 2014514921 A JP2014514921 A JP 2014514921A JP 2014501947 A JP2014501947 A JP 2014501947A JP 2014501947 A JP2014501947 A JP 2014501947A JP 2014514921 A JP2014514921 A JP 2014514921A
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nucleic acid
protein
acid molecule
rna
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ピエロ カルニンチ
アリステア フォレスト
ステファノ グスティンチック
クラウディア カッリエーリ
シルヴィア ズックリ
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RIKEN
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • 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
    • 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
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid

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  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Organic Chemistry (AREA)
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  • Biochemistry (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Developing Agents For Electrophotography (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2014501947A 2011-03-30 2012-03-30 機能性核酸分子、及びその利用 Pending JP2014514921A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161469399P 2011-03-30 2011-03-30
US61/469,399 2011-03-30
PCT/JP2012/059430 WO2012133947A1 (en) 2011-03-30 2012-03-30 Functional nucleic acid molecule and use thereof

Related Child Applications (1)

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JP2017083227A Division JP6770921B2 (ja) 2011-03-30 2017-04-19 機能性核酸分子、及びその利用

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JP2014514921A true JP2014514921A (ja) 2014-06-26
JP2014514921A5 JP2014514921A5 (enExample) 2015-05-14

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JP2014501947A Pending JP2014514921A (ja) 2011-03-30 2012-03-30 機能性核酸分子、及びその利用
JP2017083227A Active JP6770921B2 (ja) 2011-03-30 2017-04-19 機能性核酸分子、及びその利用
JP2020162110A Active JP7229487B2 (ja) 2011-03-30 2020-09-28 機能性ｒｎａ分子、及びその利用

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JP2020162110A Active JP7229487B2 (ja) 2011-03-30 2020-09-28 機能性ｒｎａ分子、及びその利用

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US (1) US9353370B2 (enExample)
EP (2) EP2691522B1 (enExample)
JP (3) JP2014514921A (enExample)
ES (1) ES2855577T3 (enExample)
WO (1) WO2012133947A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021512646A (ja) * 2018-02-05 2021-05-20 スクオラ インターナジオナレ スペリオレ ディ ステュディ アバンザティ‐シッサ 翻訳を上方調節するアンチセンスｒｎａ分子の構造ドメイン
JP2023542529A (ja) * 2020-09-24 2023-10-10 フォンダジオネ イスティツト イタリアノ ディ テクノロジア 修飾された機能性核酸分子
JP2023545083A (ja) * 2020-10-08 2023-10-26 トランシネ セラピューティクス リミテッド 機能性核酸分子

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* Cited by examiner, † Cited by third party
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CN105624156B (zh) * 2014-11-04 2021-07-16 清华大学 含有反向sineb2重复序列的人工非编码rna及其在增强靶蛋白翻译中的用途
IT201700105372A1 (it) * 2017-09-20 2019-03-20 Fondazione St Italiano Tecnologia Molecola di acido nucleico funzionale e relativo uso
IT201900011490A1 (it) 2019-07-11 2021-01-11 Scuola Int Superiore Di Studi Avanzati Sissa MOLECOLE DI ACIDO NUCLEICO FUNZIONALI CHE AUMENTANO LA TRADUZIONE DI UN mRNA DELLA FRATASSINA
WO2021046243A2 (en) 2019-09-03 2021-03-11 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
EP3992289A1 (en) 2020-10-30 2022-05-04 Transine Therapeutics Limited Functional nucleic acid molecules incorporating protein binding domain
WO2022154107A1 (ja) 2021-01-18 2022-07-21 イビデン株式会社 組電池用熱伝達抑制シート及び組電池
EP4063505A1 (en) 2021-03-22 2022-09-28 Fondazione Istituto Italiano di Tecnologia Functional nucleic acid molecules directed to targets for nervous system disorders
WO2022214635A1 (en) 2021-04-08 2022-10-13 Stichting Vu Nucleic acid molecules for compensation of stxbp1 haploinsufficiency and their use in the treatment of stxbp1-related disorders
WO2022241029A1 (en) 2021-05-11 2022-11-17 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
GB202205423D0 (en) 2022-04-12 2022-05-25 Transine Therapeutics Ltd Functional nucleic acid molecule
GB202207796D0 (en) 2022-05-26 2022-07-13 Fondazione St Italiano Tecnologia Functional nucleic acid molecule
GB202207795D0 (en) 2022-05-26 2022-07-13 Fondazione St Italiano Tecnologia Functional nucleic acid molecule
IT202200011546A1 (it) 2022-06-01 2022-09-01 Univ Degli Studi Di Trento Sequenze di RNA non codificanti in grado di aumentare l'espressione delle proteine CHD8 e CHD2
WO2025133858A1 (en) 2023-12-18 2025-06-26 Fondazione Istituto Italiano Di Tecnologia Functional nucleic acid molecule capable of increasing bdnf production
WO2026115437A1 (en) 2024-11-26 2026-06-04 Universita' Degli Studi Del Piemonte Orientale "A. Avogadro" Nucleic acid molecules upregulating the translation of prpf31 mrna

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WO2008113773A2 (en) * 2007-03-16 2008-09-25 Biorigen S.R.L. Gene expression regulation technology and noncoding rnas for diagnosis and therapy

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US5807715A (en) 1984-08-27 1998-09-15 The Board Of Trustees Of The Leland Stanford Junior University Methods and transformed mammalian lymphocyte cells for producing functional antigen-binding protein including chimeric immunoglobulin
ES2534300T3 (es) * 2004-11-12 2015-04-21 Asuragen, Inc. Procedimientos y composiciones que implican miARN y moléculas inhibidoras de miARN
EP2206724A1 (en) 2005-12-13 2010-07-14 Kyoto University Nuclear reprogramming factor
ATE532868T1 (de) * 2007-12-28 2011-11-15 Qiagen Sciences Inc Apoptose induzierende positivkontrolle für expressionsmodulationsexperimente
JP2010273680A (ja) 2009-05-29 2010-12-09 Kyoto Univ 初期化因子が除去された人工多能性幹細胞の作製方法
JP5696282B2 (ja) 2009-06-10 2015-04-08 国立大学法人京都大学 人工多能性幹細胞樹立効率改善剤
JP2011004674A (ja) 2009-06-26 2011-01-13 Fujitsu Ltd 誘導多能性幹細胞（ｉＰＳ細胞）の製造方法
JP5751548B2 (ja) 2009-08-07 2015-07-22 国立大学法人京都大学 イヌｉＰＳ細胞及びその製造方法

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WO2008113773A2 (en) * 2007-03-16 2008-09-25 Biorigen S.R.L. Gene expression regulation technology and noncoding rnas for diagnosis and therapy

Non-Patent Citations (9)

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JPN6016007269; ＣＡＲＮＩＮＣＩ　Ｐ．　ｅｔ　ａｌ．　'Ｍｕｓ　ｍｕｓｃｕｌｕｓ　ａｄｕｌｔ　ｍａｌｅ　ｏｌｆａｃｔｏｒｙ　ｂｒａｉｎ　ｃＤＮＡ，　ＲＩＫＥＮｆｕｌｌ-ｌｅｎｇｔｈ　ｅｎｒｉｃｈｅｄ　ｌｉｂｒａｒｙ， *
JPN6016007271; ＣＡＲＮＩＮＣＩ　Ｐ．　ｅｔ　ａｌ．　'Ｍｕｓ　ｍｕｓｃｕｌｕｓ　０　ｄａｙ　ｎｅｏｎａｔｅ　ｈｅａｄ　ｃＤＮＡ，　ＲＩＫＥＮｆｕｌｌ-ｌｅｎｇｔｈ　ｅｎｒｉｃｈｅｄ　ｌｉｂｒａｒｙ，　ｃｌｏｎｅ：４ *
JPN6016007273; Ｎａｔｕｒｅ，　２００２，　ｖｏｌ．４２０，　ｐ．５６３-７３ *
JPN6016007276; Ｇｅｎｏｍｅ　Ｒｅｓ．，　２０００，　ｖｏｌ．１０，　ｐ．１７５７-７１ *
JPN6016007277; Ｇｅｎｏｍｅ　Ｒｅｓ．，　２０００，　ｖｏｌ．１０，　ｐ．１６１７-３０ *
JPN6016007279; Ｎａｔｕｒｅ，　２００１，　ｖｏｌ．４０９，　ｐ．６８５-９０ *
JPN6016007281; Ｓｃｉｅｎｃｅ，　２００５，　ｖｏｌ．３０９，　ｐ．１５５９-６３ *
JPN6016007282; Ｓｃｉｅｎｃｅ，　２００５，　ｖｏｌ．３０９，　ｐ．１５６４-６ *
JPN6016007285; Ｐｒｏｃ．　Ｎａｔｌ．　Ａｃａｄ．　Ｓｃｉ．　Ｕ．Ｓ．Ａ．，　２００８，　ｖｏｌ．１０５，　ｐ．４２２０-５ *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021512646A (ja) * 2018-02-05 2021-05-20 スクオラ インターナジオナレ スペリオレ ディ ステュディ アバンザティ‐シッサ 翻訳を上方調節するアンチセンスｒｎａ分子の構造ドメイン
JP2023542529A (ja) * 2020-09-24 2023-10-10 フォンダジオネ イスティツト イタリアノ ディ テクノロジア 修飾された機能性核酸分子
JP7900371B2 (ja) 2020-09-24 2026-08-04 フォンダジオネ イスティツト イタリアノ ディ テクノロジア 修飾された機能性核酸分子
JP2023545083A (ja) * 2020-10-08 2023-10-26 トランシネ セラピューティクス リミテッド 機能性核酸分子
JP7811941B2 (ja) 2020-10-08 2026-02-06 フォンダツィオーネ・イスティテュート・イタリアーノ・ディ・テクノロジア 機能性核酸分子

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EP2691522A1 (en) 2014-02-05
EP2691522B1 (en) 2020-11-25
JP7229487B2 (ja) 2023-02-28
JP2021006047A (ja) 2021-01-21
EP2691522A4 (en) 2014-11-05
JP2017169573A (ja) 2017-09-28
JP6770921B2 (ja) 2020-10-21
EP3792359A1 (en) 2021-03-17
WO2012133947A1 (en) 2012-10-04
ES2855577T3 (es) 2021-09-23
US9353370B2 (en) 2016-05-31
US20140107187A1 (en) 2014-04-17

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