+

WO2005105995A3 - Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina) - Google Patents

Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina) Download PDF

Info

Publication number
WO2005105995A3
WO2005105995A3 PCT/US2005/006661 US2005006661W WO2005105995A3 WO 2005105995 A3 WO2005105995 A3 WO 2005105995A3 US 2005006661 W US2005006661 W US 2005006661W WO 2005105995 A3 WO2005105995 A3 WO 2005105995A3
Authority
WO
WIPO (PCT)
Prior art keywords
polyq
repeat
diseases
nucleic acid
conditions
Prior art date
Application number
PCT/US2005/006661
Other languages
English (en)
Other versions
WO2005105995A2 (fr
Inventor
James Mcswiggen
Original Assignee
Sirna Therapeutics Inc
James Mcswiggen
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=56290667&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=WO2005105995(A3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from US10/824,036 external-priority patent/US20050191638A1/en
Priority claimed from US10/826,966 external-priority patent/US20050032733A1/en
Priority claimed from PCT/US2004/013456 external-priority patent/WO2005041859A2/fr
Priority claimed from PCT/US2004/016390 external-priority patent/WO2005019453A2/fr
Priority claimed from US10/923,536 external-priority patent/US20070042983A1/en
Priority claimed from PCT/US2005/004270 external-priority patent/WO2005078097A2/fr
Application filed by Sirna Therapeutics Inc, James Mcswiggen filed Critical Sirna Therapeutics Inc
Priority to EP05724244A priority Critical patent/EP1735443A2/fr
Publication of WO2005105995A2 publication Critical patent/WO2005105995A2/fr
Publication of WO2005105995A3 publication Critical patent/WO2005105995A3/fr

Links

Classifications

    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention a trait à des composés, des compositions, et des procédés pour l'étude, le diagnostic, et le traitement de maladies et de conditions associées à des variants alléliques de séquence répétée de polyglutamine (polyQ) qui réagissent à la modulation de l'expression et/ou de l'activité génétique. La présente invention a également trait à des composés, des compositions, et des procédés concernant des maladies et des conditions associées à des variants alléliques de séquence de répétition de polyglutamine (polyQ) qui réagissent à la modulation de l'expression et/ou de l'activité génétique de gènes impliqués dans les voies d'expression de gène de séquence de répétition de polyQ, ou d'autres processus cellulaires liés au maintien ou au développement des maladies à séquence de répétition de polyQ et des conditions telles que la maladies de Huntington et des conditions associées telles que la chorée progressive, la rigidité, la démence, et les crises, l'ataxie spinocérébelleuse, la dystrophie musculaire spino-bulbaire (SBMA), l'atrophie dentatorubropallidoluysienne (DIZPLA), et toutes autres maladies et conditions associées ou réagissant aux niveaux d'une protéine d'expansion de séquence de répétition dans une cellule ou dans un tissu, seuls ou en combinaison avec d'autres thérapies. De manière spécifique, l'invention a trait à des petites molécules d'acide nucléique, telles que des molécules d'acide nucléique interférent court (siNA), d'ARN interférent court (ARNsi), d'ARN double brin (ARNdb), de micro ARN (ARNmi), et d'ARN en épingle à cheveux (shRNA) capables d'une médiation d'ARN interférent (ARNsi) contre l'expression de gènes ou allèles ayant des séquences répétées de polyQ.
PCT/US2005/006661 2004-04-14 2005-03-01 Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina) WO2005105995A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP05724244A EP1735443A2 (fr) 2004-04-14 2005-03-01 Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US10/824,036 2004-04-14
US10/824,036 US20050191638A1 (en) 2002-02-20 2004-04-14 RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US10/826,966 2004-04-16
US10/826,966 US20050032733A1 (en) 2001-05-18 2004-04-16 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
USPCT/US04/13456 2004-04-30
PCT/US2004/013456 WO2005041859A2 (fr) 2003-04-30 2004-04-30 Conjugues et compositions
PCT/US2004/016390 WO2005019453A2 (fr) 2001-05-18 2004-05-24 Interference arn a mediation assuree par l'inhibition de genes au moyen de petit acide nucleique interferent (ansi) modifie chimiquement
USPCT/US04/16390 2004-05-24
US10/923,536 US20070042983A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US10/923,536 2004-08-20
USPCT/US05/04270 2005-02-09
PCT/US2005/004270 WO2005078097A2 (fr) 2004-02-10 2005-02-09 Inhibition induite par l'interference arn de l'expression genetique, a l'aide d'un acide nucleique interferant court multifonctionnel (sina multifonctionnel)

Publications (2)

Publication Number Publication Date
WO2005105995A2 WO2005105995A2 (fr) 2005-11-10
WO2005105995A3 true WO2005105995A3 (fr) 2005-12-29

Family

ID=56290667

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2005/006661 WO2005105995A2 (fr) 2004-04-14 2005-03-01 Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina)

Country Status (3)

Country Link
EP (1) EP1735443A2 (fr)
GB (1) GB2415961A (fr)
WO (1) WO2005105995A2 (fr)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9260716B2 (en) 2002-08-05 2016-02-16 University Of Iowa Research Foundation RNA interference suppression of neurodegenerative diseases and methods of use thereof
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9487779B2 (en) 2002-08-05 2016-11-08 University Of Iowa Research Foundation siRNA-mediated gene silencing
US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA
US12297431B2 (en) 2009-09-11 2025-05-13 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050042646A1 (en) 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US7829694B2 (en) * 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
AU2006210973A1 (en) * 2005-01-31 2006-08-10 University Of Iowa Research Foundation Nucleic acid silencing of Huntington's Disease gene
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
WO2006121960A2 (fr) 2005-05-06 2006-11-16 Medtronic, Inc. Procedes et sequences permettant de supprimer l'expression du gene de huntington chez les primates
US7951934B2 (en) 2006-01-26 2011-05-31 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
WO2007123391A1 (fr) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Intervention thérapeutique dans une maladie génétique chez un individu en modifiant l'expression d'un gène exprimé de manière aberrante.
EP1857548A1 (fr) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Procédé et moyen permettant d'induire un saut d'exon
EP2046993A4 (fr) * 2006-07-07 2010-11-17 Univ Massachusetts Compositions de silencage de l'arn, et méthodes de traitement de la chorée de huntington
NZ574807A (en) 2006-08-11 2011-01-28 Prosensa Technologies Bv Methods and means for treating dna repeat instability associated genetic disorders
CA2688514A1 (fr) * 2007-04-26 2008-11-06 University Of Iowa Research Foundation Suppression de l'interference par l'arn des maladies neurodegeneratives et ses methodes d'utilisation
ES2549122T3 (es) 2007-05-31 2015-10-23 University Of Iowa Research Foundation Reducción de toxicidad por interferencia de ARN desviada de su diana
JP5706157B2 (ja) 2007-07-12 2015-04-22 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． 化合物を種々の選択臓器又は組織に標的化するための分子
JP2010533170A (ja) 2007-07-12 2010-10-21 プロセンサ テクノロジーズ ビー．ブイ． 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子
RU2356498C1 (ru) * 2007-09-26 2009-05-27 Государственное Учреждение Научный Центр Неврологии Российской Академии Медицинских Наук Способ диагностики нейродегенеративного заболевания хореи гентингтона
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
AU2009210872A1 (en) 2008-02-08 2009-08-13 Prosensa Holding Bv Methods and means for treating DNA repeat instability associated genetic disorders
EP2119783A1 (fr) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
AU2009276763B2 (en) 2008-07-29 2015-07-16 The Board Of Regents Of The University Of Texas Sytem Selective inhibition of polyglutamine protein expression
EP2421971B1 (fr) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucléotides comportant une inosine pour le traitement de dmd
EP2510098B1 (fr) 2009-12-09 2015-02-11 Quark Pharmaceuticals, Inc. Méthodes et compositions utilisées pour le traitement de maladies, d'affections ou de lésions du snc
WO2012109667A1 (fr) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Composés thérapeutiques
US20150051389A1 (en) 2011-08-11 2015-02-19 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
JP2015509922A (ja) 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
RS56783B9 (sr) 2012-12-05 2021-12-31 Alnylam Pharmaceuticals Inc Sastavi pcsk9 irnk i postupci njihovih primena
RU2552913C1 (ru) * 2014-03-25 2015-06-10 Государственное бюджетное образовательное учреждение высшего профессионального образования "Нижегородская государственная медицинская академия" Министерства Здравоохранения Российской Федерации (ГБОУ ВПО НижГМА Минздрава России) Способ диагностики болезни гентингтона
CN107075514A (zh) 2014-05-20 2017-08-18 衣阿华大学研究基金会 亨廷顿氏病的治疗化合物
KR102636633B1 (ko) 2014-12-24 2024-02-13 유니큐어 아이피 비.브이. 헌팅턴 유전자 억제를 유도하는 RNAi
CN108138221B (zh) * 2015-05-10 2021-11-05 康代思锐公司 用于检测核酸的超高灵敏度探针
CN118079015A (zh) 2015-08-25 2024-05-28 阿尔尼拉姆医药品有限公司 用于治疗前蛋白转化酶枯草杆菌蛋白酶kexin(pcsk9)基因相关障碍的方法和组合物
EP3908658A1 (fr) 2019-01-09 2021-11-17 Universidade de Coimbra Arn double brin et utilisations correspondantes
EP3997225A1 (fr) 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Méthodes pour le traitement de l'épilepsie
JP2023527684A (ja) * 2020-05-11 2023-06-30 ジェネンテック， インコーポレイテッド 神経疾患を治療するための補体成分ｃ１ｓ阻害剤、並びにそれを使用する関連組成物、システム及び方法
BR112023019981A2 (pt) * 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas
CN115725656A (zh) * 2021-09-01 2023-03-03 联邦生物科技(珠海横琴)有限公司 一种以细胞质rna病毒为载体递送干扰rna的方法

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003064625A2 (fr) * 2002-02-01 2003-08-07 Sequitur, Inc. Compositions oligonucleotidiques presentant une efficacite amelioree
WO2003070910A2 (fr) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Inhibition induite par interference d'arn du facteur de croissance endothelial vasculaire et expression genetique du recepteur de facteur de croissance endothelial vasculaire au moyen d'acides nucleiques interferents courts (sina)
WO2004013280A2 (fr) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation Silençage genique a mediation par arnsi specifique des alleles
US20050096284A1 (en) * 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
WO2005078097A2 (fr) * 2004-02-10 2005-08-25 Sirna Therapeutics, Inc. Inhibition induite par l'interference arn de l'expression genetique, a l'aide d'un acide nucleique interferant court multifonctionnel (sina multifonctionnel)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040092465A1 (en) * 2002-11-11 2004-05-13 Isis Pharmaceuticals Inc. Modulation of huntingtin interacting protein 1 expression
JP3917034B2 (ja) * 2002-07-23 2007-05-23 湖北工業株式会社 光コネクタおよびその製造方法
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
EP3760234B1 (fr) * 2003-09-12 2023-11-01 University of Massachusetts Interférence arn pour le traitement de troubles à gain de fonction

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003064625A2 (fr) * 2002-02-01 2003-08-07 Sequitur, Inc. Compositions oligonucleotidiques presentant une efficacite amelioree
WO2003070910A2 (fr) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Inhibition induite par interference d'arn du facteur de croissance endothelial vasculaire et expression genetique du recepteur de facteur de croissance endothelial vasculaire au moyen d'acides nucleiques interferents courts (sina)
US20050096284A1 (en) * 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
WO2004013280A2 (fr) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation Silençage genique a mediation par arnsi specifique des alleles
WO2005078097A2 (fr) * 2004-02-10 2005-08-25 Sirna Therapeutics, Inc. Inhibition induite par l'interference arn de l'expression genetique, a l'aide d'un acide nucleique interferant court multifonctionnel (sina multifonctionnel)

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LIU W ET AL: "Specific inhibition of Huntington's disease gene expression by siRNAs in cultures cells", PROCEEDINGS OF THE JAPAN ACADEMY. SERIES B, PHYSICAL AND BIOLOGICAL SCIENCES, vol. 79B, no. 10, December 2003 (2003-12-01), pages 293 - 298, XP002983007, ISSN: 0386-2208 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9260716B2 (en) 2002-08-05 2016-02-16 University Of Iowa Research Foundation RNA interference suppression of neurodegenerative diseases and methods of use thereof
US9487779B2 (en) 2002-08-05 2016-11-08 University Of Iowa Research Foundation siRNA-mediated gene silencing
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA
US12297431B2 (en) 2009-09-11 2025-05-13 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression

Also Published As

Publication number Publication date
GB2415961A (en) 2006-01-11
GB0507019D0 (en) 2005-05-11
EP1735443A2 (fr) 2006-12-27
WO2005105995A2 (fr) 2005-11-10

Similar Documents

Publication Publication Date Title
WO2005105995A3 (fr) Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina)
WO2007022369A3 (fr) Molecules d'acide nucleique interferent court chimiquement modifiees induisant l'interference de l'arn
AU2018253578A1 (en) RNA interference mediated inhibition of catenin (cadherin-associated protein), beta 1 (CTNNB1) gene expression using short interfering nucleic acid (siNA)
WO2003070911A3 (fr) Inhibition mediee par interference arn d une expression genique de recepteur de facteur de croissance de type insuline de type 1 (igf-1r) faisant appel a des acides nucleiques interferants courts (sina)
WO2008011431A3 (fr) INHIBITION MÉDIÉE PAR UNE INTERFÉRENCE DE L'ARN DE L'EXPRESSION DU GÈNE DE LA PROPROTÉINE CONVERTASE SUBTILISINE KEXINE DE TYPE 9 (PCSK9) AU MOYEN DE PETITS ACIDES NUCLÉIQUES INTERFÉRANTS(ANsi)
WO2007076328A3 (fr) INHIBITION A MEDIATION PAR INTERFERENCE ARN DE L'EXPRESSION DU GENE DU VIRUS DE L'HEPATITE C (VHC) AU MOYEN D'ACIDE NUCLEIQUE INTERFERENT COURT (siNA)
WO2007084865A3 (fr) INHIBITION DE L’EXPRESSION DU GENE B7-H1 MEDIEE PAR UNE INTERFERENCE DE L’ARN EN UTILISANT UN ACIDE NUCLEIQUE INTERFERANT COURT (NAsi)
WO2006128141A3 (fr) Inhibition, mediee par l'interference d'arn, de l'expression genique du facteur 1 derive des cellules stromales (sdf-1), au moyen d'acide nucleique interferant court (sina)
WO2003070918A3 (fr) Inhibition mediee par interference arn d'une expression genique faisant appel a des acides nucleiques interferants courts chimiquement modifies (sina)
WO2003070884A3 (fr) Inhibition de l'expression du gene de la mdr p-glycoproteine induite par l'interference d'arn, au moyen d'acide nucleique interferant court (sina)
WO2005045034A3 (fr) Traitement medie par interference arn de la maladie de parkinson au moyen d'un petit acide nucleique interferent (sina)
WO2003070881A3 (fr) Inhibition de l'expression genique de la proteine typrosine phoshatase-1b (ptb-1b) induite par l'interference arn, utilisant un acide nucleique d'interference court (sina)
WO2010111503A3 (fr) Inhibition à médiation par l'interférence arn de l'expression du gène de la chaîne alpha du récepteur à haute affinité pour les ige (fcεr1α) faisant appel à de courts acides nucléiques interférents (ansi)
WO2003072705A3 (fr) Inhibition a mediation d'arn de l'expression genique de la cycline d1 a l'aide d'acide nucleique a courte interference (sina)
WO2010107957A3 (fr) Inhibition induite par arn interférence d'une expression génique (gata3) d'une protéine de liaison gata au moyen d'un acide nucléique interférent court
WO2008011467A3 (fr) Inhibition à médiation par l'interférence de l'arn de l'expression du gène de la protéine 1 de liaison à l'élément régulateur de stérol (srebp1) à l'aide d'un acide nucléique interférent court (sina)
WO2004092383A3 (fr) Inhibition par interference d'arn de l'expression genetique virale du syndrome respiratoire aigu severe au moyen d'acide nucleique d'interference court
WO2005003350A3 (fr) Traitement medie par interference d'arn de la maladie d'alzheimer a l'aide d'acide nucleique interferent court (sina)
WO2007147143A3 (fr) Inhibition médiée par l'interférence d'arn de l'expression du gène de la 11-bêta-hydroxystéroïde déshydrogénase 1 (11-bêta-hsd-1) en utilisant un acide nucléique interférent court (ansi)
WO2003070887A3 (fr) Inhibition mediee par interference d'arn de l'expression du gene de la proteine ezh2 du groupe polycomb a l'aide d'un acide nucleique court interferent (sina)
WO2005007855A3 (fr) Inhibition induite par interference d'arn de l'expression du gene b7-h1 au moyen d'acide nucleique a interference courte (sina)
WO2010111497A3 (fr) Inhibition à médiation par l'interférence arn de l'expression du gène de la molécule d'adhésion intercellulaire 1 (icam-1) faisant appel à de courts acides nucléiques interférents (ansi)
EP1522583A3 (fr) Inhibition au moyen de l'interférence par l'ARN de l'expression du gène du Placental growth factor à l'aide de courts acides nucléiques interférants
WO2005045032A3 (fr) Inhibition mediee par une interference arn de l'expression du gene de reponse de croissance precoce, au moyen d'un petit acide nucleique interferant (sina)
WO2005014811A3 (fr) Inhibition a mediation par interference arn de l'expression du gene xiap au moyen d'acide nucleique interferent court

Legal Events

Date Code Title Description
AK Designated states

Kind code of ref document: A2

Designated state(s): AE AG AL AM AT AU AZ BA BB BG BR BW BY BZ CA CH CN CO CR CU CZ DE DK DM DZ EC EE EG ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MA MD MG MK MN MW MX MZ NA NI NO NZ OM PG PH PL PT RO RU SC SD SE SG SK SL SM SY TJ TM TN TR TT TZ UA UG US UZ VC VN YU ZA ZM ZW

AL Designated countries for regional patents

Kind code of ref document: A2

Designated state(s): GM KE LS MW MZ NA SD SL SZ TZ UG ZM ZW AM AZ BY KG KZ MD RU TJ TM AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IS IT LT LU MC NL PL PT RO SE SI SK TR BF BJ CF CG CI CM GA GN GQ GW ML MR NE SN TD TG

121 Ep: the epo has been informed by wipo that ep was designated in this application
WWE Wipo information: entry into national phase

Ref document number: 2005724244

Country of ref document: EP

NENP Non-entry into the national phase

Ref country code: DE

WWW Wipo information: withdrawn in national office

Country of ref document: DE

WWP Wipo information: published in national office

Ref document number: 2005724244

Country of ref document: EP

点击 这是indexloc提供的php浏览器服务，不要输入任何密码和下载