JP2010510807A - 高コレステロール血症を治療するための方法 - Google Patents

高コレステロール血症を治療するための方法 Download PDF

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Publication number
JP2010510807A
JP2010510807A JP2009539274A JP2009539274A JP2010510807A JP 2010510807 A JP2010510807 A JP 2010510807A JP 2009539274 A JP2009539274 A JP 2009539274A JP 2009539274 A JP2009539274 A JP 2009539274A JP 2010510807 A JP2010510807 A JP 2010510807A
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seq
compound
certain embodiments
cholesterol
nucleotides
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JP2010510807A5 (enExample
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フレイヤー,スーザン,エム.
クローク,ロザンヌ,エム.
クラハム,マーク,ジェイ.
タルベット,クリスティーナ,レモニディス
バーノット,サンジャイ
トライブル,ダイアン
ワット,アンドリュー,ティー.
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
Isis Pharmaceuticals Inc
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JP2009539274A 2006-11-27 2007-11-27 高コレステロール血症を治療するための方法 Pending JP2010510807A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US86739506P 2006-11-27 2006-11-27
US91289207P 2007-04-19 2007-04-19
US98628607P 2007-11-07 2007-11-07
US98807407P 2007-11-14 2007-11-14
PCT/US2007/024369 WO2008066776A2 (en) 2006-11-27 2007-11-27 Methods for treating hypercholesterolemia

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JP2010510807A true JP2010510807A (ja) 2010-04-08
JP2010510807A5 JP2010510807A5 (enExample) 2011-01-20

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EP (3) EP2453016A1 (enExample)
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KR (1) KR20090103894A (enExample)
AU (1) AU2007325767A1 (enExample)
CA (1) CA2670563A1 (enExample)
CO (1) CO6241169A2 (enExample)
EA (1) EA200900741A1 (enExample)
IL (1) IL198851A0 (enExample)
MX (1) MX2009005527A (enExample)
NO (1) NO20092150L (enExample)
TW (1) TW200838551A (enExample)
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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014007305A1 (ja) * 2012-07-04 2014-01-09 国立大学法人大阪大学 オリゴヌクレオチド、およびオリゴヌクレオチドを有効成分として含有する脂質異常症治療剤
JP2016520616A (ja) * 2013-05-30 2016-07-14 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. プロタンパク質変換酵素スブチリシンケキシン−9(pcsk9)の阻害剤を投与することによるレムナントコレステロールおよび他のリポタンパク質画分を減少させるための方法

Families Citing this family (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2194128B1 (en) 2006-05-11 2012-08-01 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
EP2453016A1 (en) 2006-11-27 2012-05-16 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
JOP20080381B1 (ar) 2007-08-23 2023-03-28 Amgen Inc بروتينات مرتبطة بمولدات مضادات تتفاعل مع بروبروتين كونفيرتاز سيتيليزين ككسين من النوع 9 (pcsk9)
US10131904B2 (en) 2008-02-11 2018-11-20 Rxi Pharmaceuticals Corporation Modified RNAi polynucleotides and uses thereof
WO2009148605A2 (en) * 2008-06-04 2009-12-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
HRP20171969T1 (hr) * 2008-10-15 2018-04-06 Ionis Pharmaceuticals, Inc. Modulacija ekspresije faktora 11
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
JO3672B1 (ar) 2008-12-15 2020-08-27 Regeneron Pharma أجسام مضادة بشرية عالية التفاعل الكيماوي بالنسبة لإنزيم سبتيليسين كنفرتيز بروبروتين / كيكسين نوع 9 (pcsk9).
US9493774B2 (en) * 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
NZ594995A (en) * 2009-03-12 2013-06-28 Alnylam Pharmaceuticals Inc LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF HUMAN KINESIN FAMILY MEMBER 11 (Eg5) AND VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) GENES
NZ595891A (en) * 2009-04-15 2013-06-28 Isis Pharmaceuticals Inc Modulation of inflammatory responses by factor xi
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
KR20120050429A (ko) 2009-06-15 2012-05-18 알닐람 파마슈티칼스 인코포레이티드 Pcsk9 유전자를 표적으로 하는 지질 제형된 dsrna
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
EP2456870A1 (en) 2009-07-21 2012-05-30 Santaris Pharma A/S Antisense oligomers targeting pcsk9
EP3561060A1 (en) * 2010-02-08 2019-10-30 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2011097644A2 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
GB2481373A (en) * 2010-06-21 2011-12-28 Weiming Xu Treatment of hypercholesterolaemia by ubiquitination of PCSK9
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
WO2012058693A2 (en) * 2010-10-29 2012-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of pcsk9 genes
BR112013018740A2 (pt) 2011-01-28 2019-01-08 Sanofi Sa anticorpos humanos para pcsk9 para uso em métodos de tratamento de grupos específicos de indivíduos
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
AR087305A1 (es) 2011-07-28 2014-03-12 Regeneron Pharma Formulaciones estabilizadas que contienen anticuerpos anti-pcsk9, metodo de preparacion y kit
EP2742056B2 (en) 2011-08-11 2020-06-10 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
CN103930444B (zh) 2011-09-16 2020-08-04 瑞泽恩制药公司 用前蛋白转化酶枯草溶菌素-9(PCSK9)抑制剂降低脂蛋白(a)水平的方法
JP2015530867A (ja) 2012-06-15 2015-10-29 ジェネンテック, インコーポレイテッド 抗pcsk9抗体、製剤、投薬、及び使用方法
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
CA2887884A1 (en) 2012-10-12 2014-04-17 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
JP6452614B2 (ja) 2012-11-15 2019-01-16 ロシュ イノベーション センター コペンハーゲン エーエス オリゴヌクレオチドコンジュゲート
PT2929031T (pt) 2012-12-05 2018-01-19 Alnylam Pharmaceuticals Inc Composições de irna de pcsk9 e métodos de seu uso
DK2951305T3 (en) 2013-01-30 2018-10-29 Hoffmann La Roche LNA oligonucleotide KULHYDRATKONJUGATER
GB2523527A (en) * 2013-04-05 2015-09-02 Weiming Xu Screen compounds for the modulation of proprotein convertase subtilisin/kexin type 9(PCSK9)
BR112015026513A2 (pt) 2013-04-17 2017-07-25 Pfizer derivados de n-piperidin-3-ilbenzamida para tratar as doenças cardiovasculares
PE20152002A1 (es) 2013-05-01 2016-01-21 Isis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de ttr y vhb
SG10201908122XA (en) 2013-06-27 2019-10-30 Roche Innovation Ct Copenhagen As Antisense oligomers and conjugates targeting pcsk9
WO2015002971A2 (en) * 2013-07-02 2015-01-08 Isis Pharmaceuticals, Inc. Modulators of growth hormone receptor
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
MY192997A (en) * 2015-07-10 2022-09-20 Ionis Pharmaceuticals Inc Modulators of diacyglycerol acyltransferase 2 (dgat2)
JP2018523684A (ja) 2015-08-18 2018-08-23 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. リポタンパク質アフェレーシスを受けている高脂血症の患者を治療するための抗pcsk9阻害抗体
IL293355B2 (en) 2015-08-25 2024-07-01 Alnylam Pharmaceuticals Inc Methods and compositions for treating a proprotein convertase subtilisin kexin (pcsk9) gene-associated disorder
KR20190065341A (ko) 2016-10-06 2019-06-11 아이오니스 파마수티컬즈, 인코포레이티드 올리고머 화합물들의 접합 방법
WO2018102397A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
EP3765611A4 (en) 2018-04-18 2022-05-04 Dicerna Pharmaceuticals, Inc. PCSK9 TARGETED OLIGONUCLEOTIDES FOR THE TREATMENT OF HYPERCHOLESTEROLEMIA AND RELATED DISORDERS
SG11202010012PA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing fxi expression
KR20180087217A (ko) 2018-07-20 2018-08-01 주식회사 유진바이오텍 귀리산화 베타-글루칸을 유효성분으로 포함하는 비만, 이상지방혈증(dyslipidemia) 또는 지방간의 예방 또는 치료용 조성물
WO2024229020A2 (en) * 2023-05-01 2024-11-07 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of pcsk9 expression

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004357715A (ja) * 2003-01-21 2004-12-24 Research Association For Biotechnology 全長cDNA
WO2007134161A2 (en) * 2006-05-11 2007-11-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the pcsk9 gene
WO2008011431A2 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)
WO2008043753A2 (en) * 2006-10-09 2008-04-17 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9

Family Cites Families (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US604360A (en) * 1898-05-24 Safety device for elevators
US5023243A (en) * 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US5118800A (en) * 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) * 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5591722A (en) * 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
EP0497875B1 (en) * 1989-10-24 2000-03-22 Isis Pharmaceuticals, Inc. 2' modified oligonucleotides
US5646265A (en) * 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5681941A (en) * 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US6005087A (en) * 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
DE69032425T2 (de) * 1990-05-11 1998-11-26 Microprobe Corp., Bothell, Wash. Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden
US5677437A (en) * 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
ES2083593T3 (es) * 1990-08-03 1996-04-16 Sterling Winthrop Inc Compuestos y metodos para inhibir la expresion de genes.
US6582908B2 (en) * 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
ES2103918T3 (es) * 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
TW393513B (en) * 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5359044A (en) * 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
FR2687679B1 (fr) * 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
US5633360A (en) * 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
EP0577558A2 (de) * 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
WO1994001093A1 (en) 1992-07-09 1994-01-20 Merck & Co., Inc. Controlled porosity osmotic enalapril pump
US5652355A (en) * 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
AU6449394A (en) 1993-03-30 1994-10-24 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
AU6412794A (en) * 1993-03-31 1994-10-24 Sterling Winthrop Inc. Oligonucleotides with amide linkages replacing phosphodiester linkages
HU9501994D0 (en) 1993-03-31 1995-09-28 Sterling Winthrop Inc Novel 5'-substituted nucleosides and oligomers produced therefrom
DE4311944A1 (de) * 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
US5801154A (en) * 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5457187A (en) * 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) * 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) * 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5627053A (en) * 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) * 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5597909A (en) * 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5792747A (en) * 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
US5567427A (en) * 1995-03-17 1996-10-22 Helene Curtis, Inc. Emulsified, low ph cosmetic compositions having improved stability
US5673633A (en) * 1995-05-31 1997-10-07 Pfister; Joel W. Table leg system
ATE232880T1 (de) 1996-11-18 2003-03-15 Takeshi Imanishi Neue nucleotidanaloga
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) * 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
US6794499B2 (en) * 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US20030228597A1 (en) * 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6043352A (en) * 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
US6410323B1 (en) * 1999-08-31 2002-06-25 Isis Pharmaceuticals, Inc. Antisense modulation of human Rho family gene expression
KR100573231B1 (ko) * 1999-02-12 2006-04-24 상꾜 가부시키가이샤 신규 뉴클레오시드 및 올리고뉴클레오티드 유사체
US7084125B2 (en) * 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
AU776362B2 (en) * 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6525191B1 (en) * 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP4151751B2 (ja) * 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
AU2001282522A1 (en) * 2000-08-29 2002-03-13 Takeshi Imanishi Novel nucleoside analogs and oligonucleotide derivatives containing these analogs
US6426220B1 (en) * 2000-10-30 2002-07-30 Isis Pharmaceuticals, Inc. Antisense modulation of calreticulin expression
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US7407943B2 (en) * 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
CA2459347C (en) * 2001-09-04 2012-10-09 Exiqon A/S Locked nucleic acid (lna) compositions and uses thereof
WO2003085110A2 (en) * 2002-04-05 2003-10-16 Santaris Pharma A/S Oligomeric compounds for the modulation hif-1alpha expression
US7569575B2 (en) * 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US20040219565A1 (en) * 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
EP1562971B1 (en) * 2002-11-05 2014-02-12 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US7511131B2 (en) * 2002-11-13 2009-03-31 Genzyme Corporation Antisense modulation of apolipoprotein B expression
US7655785B1 (en) * 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
US8090542B2 (en) * 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
EP2752488B1 (en) * 2002-11-18 2020-02-12 Roche Innovation Center Copenhagen A/S Antisense design
EP1471152A1 (en) * 2003-04-25 2004-10-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Mutations in the human PCSK9 gene associated to hypercholesterolemia
JP2005060664A (ja) 2003-07-31 2005-03-10 Asahi Glass Co Ltd 含フッ素化合物、含フッ素ポリマーとその製造方法およびそれを含むレジスト組成物
DK1661905T3 (da) * 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
CA2569419A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
WO2006085973A2 (en) * 2004-07-02 2006-08-17 Avi Biopharma, Inc. Antisense antibacterial method and compound
WO2006126040A1 (en) 2005-05-25 2006-11-30 Rosetta Genomics Ltd. Bacterial and bacterial associated mirnas and uses thereof
US20070068404A1 (en) 2005-09-29 2007-03-29 Edwin Hirahara Systems and methods for additive deposition of materials onto a substrate
JP5128796B2 (ja) 2006-01-20 2013-01-23 G&Gサイエンス株式会社 脳血管障害の遺伝的リスク検出法
CA2640171C (en) 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
CA3044969A1 (en) 2006-05-05 2007-12-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gene expression
US7572618B2 (en) * 2006-06-30 2009-08-11 Bristol-Myers Squibb Company Polynucleotides encoding novel PCSK9 variants
US20110150897A1 (en) 2006-10-11 2011-06-23 Meyer Thomas F Influenza targets
EP2453016A1 (en) 2006-11-27 2012-05-16 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
US8093222B2 (en) * 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
US20100105134A1 (en) * 2007-03-02 2010-04-29 Mdrna, Inc. Nucleic acid compounds for inhibiting gene expression and uses thereof
WO2008109369A2 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting tnf gene expression and uses thereof
WO2009148605A2 (en) 2008-06-04 2009-12-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2004357715A (ja) * 2003-01-21 2004-12-24 Research Association For Biotechnology 全長cDNA
WO2007134161A2 (en) * 2006-05-11 2007-11-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the pcsk9 gene
WO2008011431A2 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)
WO2008043753A2 (en) * 2006-10-09 2008-04-17 Santaris Pharma A/S Rna antagonist compounds for the modulation of pcsk9

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
JPN6012057060; J. Lipid Res. Vol. 46, 2005, p. 1312-1319 *
JPN6012057062; Proc. Natl. Acad. Sci. USA. Vol. 102, No. 15, 2005, p. 5374-5379 *
JPN6012057064; J. Lipid. Res. Vol. 46, 2005, p. 872-884 *
JPN6012057067; Hepatology Vol. 42, No. 2, 2005, p. 362-371 *
JPN6012057070; Database GenBank [online], Accession No. NM_174936, *
JPN6012057071; Database GenBank [online], Accession No. AL589790 *
JPN6012057074; J. Neurochem. Vol. 98, 200608, p. 838-850 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014007305A1 (ja) * 2012-07-04 2014-01-09 国立大学法人大阪大学 オリゴヌクレオチド、およびオリゴヌクレオチドを有効成分として含有する脂質異常症治療剤
JP2016520616A (ja) * 2013-05-30 2016-07-14 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. プロタンパク質変換酵素スブチリシンケキシン−9(pcsk9)の阻害剤を投与することによるレムナントコレステロールおよび他のリポタンパク質画分を減少させるための方法

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