JP2009509548A - 対立遺伝子特異的ｒｎａ干渉 - Google Patents

対立遺伝子特異的ｒｎａ干渉 Download PDF

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JP2009509548A
JP2009509548A JP2008533785A JP2008533785A JP2009509548A JP 2009509548 A JP2009509548 A JP 2009509548A JP 2008533785 A JP2008533785 A JP 2008533785A JP 2008533785 A JP2008533785 A JP 2008533785A JP 2009509548 A JP2009509548 A JP 2009509548A
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shrna
sirna
base pair
rna
target
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JP2009509548A5 (enExample
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ツオシャン シュー，
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University of Massachusetts Amherst
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University of Massachusetts Boston
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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
    • C12N15/1137—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 against enzymes
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    • C12N15/09—Recombinant DNA-technology
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    • C12Y115/00—Oxidoreductases acting on superoxide as acceptor (1.15)
    • C12Y115/01—Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001—Superoxide dismutase (1.15.1.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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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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    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2008533785A 2005-09-30 2006-10-02 対立遺伝子特異的ｒｎａ干渉 Pending JP2009509548A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11/241,873 US7892793B2 (en) 2002-11-04 2005-09-30 Allele-specific RNA interference
PCT/US2006/038704 WO2007044362A2 (en) 2005-09-30 2006-10-02 Allele-specific rna interference

Publications (2)

Publication Number Publication Date
JP2009509548A true JP2009509548A (ja) 2009-03-12
JP2009509548A5 JP2009509548A5 (enExample) 2009-11-26

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JP2008533785A Pending JP2009509548A (ja) 2005-09-30 2006-10-02 対立遺伝子特異的ｒｎａ干渉

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US (2) US7892793B2 (enExample)
EP (1) EP1940862A4 (enExample)
JP (1) JP2009509548A (enExample)
AU (1) AU2006302534A1 (enExample)
CA (1) CA2624673A1 (enExample)
WO (1) WO2007044362A2 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
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JP2012525852A (ja) * 2009-05-08 2012-10-25 クルナ・インコーポレーテッド Ｄｍｄファミリーに対する天然アンチセンス転写物の抑制によるジストロフィンファミリー関連疾患の治療
JP2020500518A (ja) * 2016-11-29 2020-01-16 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル ダイナミン２関連疾患のためのアレル特異的サイレンシング療法

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US7892793B2 (en) * 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
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US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
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US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
JP2009504782A (ja) 2005-08-18 2009-02-05 アルナイラム ファーマシューティカルズ インコーポレイテッド 神経疾患を治療するための方法および組成物
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