JP2009502138A - ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法 - Google Patents

ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法 Download PDF

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JP2009502138A
JP2009502138A JP2008523010A JP2008523010A JP2009502138A JP 2009502138 A JP2009502138 A JP 2009502138A JP 2008523010 A JP2008523010 A JP 2008523010A JP 2008523010 A JP2008523010 A JP 2008523010A JP 2009502138 A JP2009502138 A JP 2009502138A
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irna agent
nucleotides
rhoa
nucleotide
modified
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JP2009502138A5 (https=
Inventor
ストゥシェク、ユルゲン
タン、パメラ
ガイック、アンク
フォンロハー、ハンス−ピーター
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Alnylam Pharmaceuticals Inc
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    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
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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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
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  • Heart & Thoracic Surgery (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
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  • Hospice & Palliative Care (AREA)
  • Oncology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2008523010A 2005-07-21 2006-07-21 ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法 Pending JP2009502138A (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US70147005P 2005-07-21 2005-07-21
US72683805P 2005-10-14 2005-10-14
US74831605P 2005-12-07 2005-12-07
PCT/US2006/028488 WO2007014077A2 (en) 2005-07-21 2006-07-21 Rnai modulation of the rho-a gene and uses thereof

Related Child Applications (1)

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JP2011170509A Division JP2011250796A (ja) 2005-07-21 2011-08-03 ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法

Publications (2)

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JP2009502138A true JP2009502138A (ja) 2009-01-29
JP2009502138A5 JP2009502138A5 (https=) 2011-03-24

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JP2008523010A Pending JP2009502138A (ja) 2005-07-21 2006-07-21 ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法
JP2011170509A Pending JP2011250796A (ja) 2005-07-21 2011-08-03 ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法
JP2011282081A Withdrawn JP2012061007A (ja) 2005-07-21 2011-12-22 ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法

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JP2011170509A Pending JP2011250796A (ja) 2005-07-21 2011-08-03 ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法
JP2011282081A Withdrawn JP2012061007A (ja) 2005-07-21 2011-12-22 ＲｈｏＡ遺伝子のＲＮＡｉ調節及びその使用法

Country Status (6)

Country Link
US (3) US20070044161A1 (https=)
EP (2) EP2230305A1 (https=)
JP (3) JP2009502138A (https=)
AU (2) AU2006272808C1 (https=)
CA (1) CA2607668A1 (https=)
WO (2) WO2007014077A2 (https=)

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EP2230305A1 (en) * 2005-07-21 2010-09-22 Alnylam Pharmaceuticals Inc. Rnai modulation of the rho-a gene and uses thereof
AU2008306455C1 (en) * 2007-10-03 2014-04-17 Quark Pharmaceuticals, Inc. Novel siRNA structures
JP5524189B2 (ja) 2008-06-06 2014-06-18 クォーク ファーマシューティカルズ インコーポレーティッド 耳障害治療のための組成物および方法
ES2804764T3 (es) 2009-06-01 2021-02-09 Halo Bio Rnai Therapeutics Inc Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos
EP2456456A4 (en) * 2009-07-24 2013-11-20 California Inst Of Techn METHOD AND COMPOSITIONS FOR TREATING NEURAL INJURY OR DEGENERATION
WO2011021221A2 (en) * 2009-08-17 2011-02-24 Reliance Life Sciences Pvt. Ltd. Compositions for spinal cord injury
US8901097B2 (en) 2009-11-08 2014-12-02 Quark Pharmaceuticals, Inc. Methods for delivery of siRNA to the spinal cord and therapies arising therefrom
KR101553753B1 (ko) * 2010-06-24 2015-09-16 쿠아크 파마수티칼스 인코퍼레이티드 Ｒｈｏａ에 대한 이중 가닥 ｒｎａ 및 그의 용도
DK2631291T3 (da) 2010-10-22 2019-06-11 Olix Pharmaceuticals Inc Nukleinsyremolekyler, der inducerer rna-interferens, og anvendelser deraf
CN108148838A (zh) 2012-05-22 2018-06-12 奥利克斯医药有限公司 具有细胞内穿透能力的诱导rna干扰的核酸分子及用途
US9770461B2 (en) 2013-08-02 2017-09-26 California Institute Of Technology Tailored glycopolymers as anticoagulant heparin mimetics
US10227370B2 (en) 2013-08-02 2019-03-12 California Institute Of Technology Heparan sulfate/heparin mimetics with anti-chemokine and anti-inflammatory activity
JP2018525037A (ja) 2015-08-24 2018-09-06 ハロー−バイオ・アールエヌエーアイ・セラピューティックス、インコーポレイテッド 遺伝子発現の調節のためのポリヌクレオチドナノ粒子及びその使用
US11071791B2 (en) 2018-01-26 2021-07-27 Wisconsin Alumni Research Foundation Vector for gene silencing and replacement and methods of use thereof
GB201903804D0 (en) * 2019-03-20 2019-05-01 Univ Leicester Methods and kits for detecting rhoa mutations
WO2025212926A1 (en) * 2024-04-05 2025-10-09 University Of Massachusetts Aav-mediated rhoa knockdown

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WO2007014075A2 (en) 2007-02-01
JP2012061007A (ja) 2012-03-29
AU2010206007B2 (en) 2011-12-08
EP2230305A1 (en) 2010-09-22
AU2006272808A1 (en) 2007-02-01
WO2007014075A3 (en) 2009-04-23
AU2006272808C1 (en) 2010-10-21
US20070042984A1 (en) 2007-02-22
AU2006272808B2 (en) 2010-04-29
EP1909850A2 (en) 2008-04-16
CA2607668A1 (en) 2007-02-01
WO2007014077A3 (en) 2007-12-06
US20070044161A1 (en) 2007-02-22
AU2010206007A1 (en) 2010-08-19
EP1909850A4 (en) 2010-05-12
JP2011250796A (ja) 2011-12-15
WO2007014077A2 (en) 2007-02-01
US20110071209A1 (en) 2011-03-24
US7772200B2 (en) 2010-08-10

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