JP5570806B2 - Pcsk9遺伝子の発現を阻害するための組成物および方法 - Google Patents

Pcsk9遺伝子の発現を阻害するための組成物および方法 Download PDF

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Publication number
JP5570806B2
JP5570806B2 JP2009510173A JP2009510173A JP5570806B2 JP 5570806 B2 JP5570806 B2 JP 5570806B2 JP 2009510173 A JP2009510173 A JP 2009510173A JP 2009510173 A JP2009510173 A JP 2009510173A JP 5570806 B2 JP5570806 B2 JP 5570806B2
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dsrna
pcsk9
expression
nucleotide
pcsk9 gene
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JP2009536827A (ja
JP2009536827A5 (enExample
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パメラ タン,
バージット ブラムラーゲ,
マリア フランク−カーメネツスキ,
ケビン フィッツジェラルド,
アキン アキニック,
ビクター イー. コテリャンスキ,
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Alnylam Pharmaceuticals Inc
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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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P9/00—Drugs for disorders of the cardiovascular system
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
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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]
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
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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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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Cosmetics (AREA)
JP2009510173A 2006-05-11 2007-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法 Expired - Fee Related JP5570806B2 (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US79945806P 2006-05-11 2006-05-11
US60/799,458 2006-05-11
US81720306P 2006-06-27 2006-06-27
US60/817,203 2006-06-27
US84008906P 2006-08-25 2006-08-25
US60/840,089 2006-08-25
US82991406P 2006-10-18 2006-10-18
US60/829,914 2006-10-18
US90113407P 2007-02-13 2007-02-13
US60/901,134 2007-02-13
PCT/US2007/068655 WO2007134161A2 (en) 2006-05-11 2007-05-10 Compositions and methods for inhibiting expression of the pcsk9 gene

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2010108836A Division JP2010246544A (ja) 2006-05-11 2010-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2014018817A Division JP6034316B2 (ja) 2006-05-11 2014-02-03 Pcsk9遺伝子の発現を阻害するための組成物および方法

Publications (3)

Publication Number Publication Date
JP2009536827A JP2009536827A (ja) 2009-10-22
JP2009536827A5 JP2009536827A5 (enExample) 2010-07-01
JP5570806B2 true JP5570806B2 (ja) 2014-08-13

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Application Number Title Priority Date Filing Date
JP2009510173A Expired - Fee Related JP5570806B2 (ja) 2006-05-11 2007-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2010108836A Pending JP2010246544A (ja) 2006-05-11 2010-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2014018817A Expired - Fee Related JP6034316B2 (ja) 2006-05-11 2014-02-03 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2015226712A Pending JP2016027830A (ja) 2006-05-11 2015-11-19 Pcsk9遺伝子の発現を阻害するための組成物および方法

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JP2010108836A Pending JP2010246544A (ja) 2006-05-11 2010-05-10 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2014018817A Expired - Fee Related JP6034316B2 (ja) 2006-05-11 2014-02-03 Pcsk9遺伝子の発現を阻害するための組成物および方法
JP2015226712A Pending JP2016027830A (ja) 2006-05-11 2015-11-19 Pcsk9遺伝子の発現を阻害するための組成物および方法

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Country Link
US (10) US7605251B2 (enExample)
EP (8) EP2835429B1 (enExample)
JP (4) JP5570806B2 (enExample)
KR (2) KR101036126B1 (enExample)
CN (2) CN103614375A (enExample)
AU (2) AU2007249329C1 (enExample)
CA (2) CA2651839C (enExample)
EA (2) EA015676B1 (enExample)
ES (2) ES2392478T3 (enExample)
IL (3) IL195181A (enExample)
NZ (2) NZ587616A (enExample)
PL (2) PL2194128T3 (enExample)
SG (1) SG171676A1 (enExample)
WO (1) WO2007134161A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12702719B2 (en) 2019-03-06 2026-08-11 Northwestern University Hairpin-like oligonucleotide-conjugated spherical nucleic acid

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