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

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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
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polyq
repeat
diseases
nucleic acid
conditions
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PCT/US2005/006661
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WO2005105995A2 (fr
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James Mcswiggen
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Sirna Therapeutics Inc
James Mcswiggen
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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

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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 US20050032733A1 (en) 2001-05-18 2004-04-16 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
US10/826,966 2004-04-16
PCT/US2004/013456 WO2005041859A2 (fr) 2003-04-30 2004-04-30 Conjugues et compositions
USPCT/US04/13456 2004-04-30
USPCT/US04/16390 2004-05-24
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
US10/923,536 2004-08-20
US10/923,536 US20070042983A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
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

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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)

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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

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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
WO2008005562A2 (fr) * 2006-07-07 2008-01-10 University Of Massachusetts Compositions de silençage de l'arn, et méthodes de traitement de la chorée de huntington
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
EP1885854B1 (fr) 2005-05-06 2012-10-17 Medtronic, Inc. Procedes et sequences permettant de supprimer l'expression du gene de huntington chez les primates
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
US20070299027A1 (en) 2006-01-26 2007-12-27 Gene Hung 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
EP2049664B1 (fr) 2006-08-11 2011-09-14 Prosensa Technologies B.V. Oligonucléotides monocaténaires complémentaires des elements repetitifs pour le traitement de troubles génétiques associés a l'instabilité des répétitions de l'ADN
US20090036395A1 (en) * 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
ES2549122T3 (es) 2007-05-31 2015-10-23 University Of Iowa Research Foundation Reducción de toxicidad por interferencia de ARN desviada de su diana
WO2009008725A2 (fr) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molécules pour cibler des composés vers divers organes, tissus ou cellules tumorales sélectionnés
NZ582521A (en) 2007-07-12 2011-09-30 Prosensa Technologies Bv A conjugate comprising the amino acid sequence LGAQSNF for targeting compounds to muscle tissue
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
NZ587178A (en) 2008-02-08 2011-11-25 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
EP2317847B1 (fr) 2008-07-29 2019-04-17 The Board of Regents of The University of Texas System Inhibition sélective d'expression de protéine de polyglutamine
WO2010123369A1 (fr) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucléotide comprenant une inosine pour traiter une dystrophie musculaire de duchenne (dmd)
KR102173836B1 (ko) 2009-09-11 2020-11-05 아이오니스 파마수티컬즈, 인코포레이티드 헌팅틴 발현의 조절
US8778904B2 (en) 2009-12-09 2014-07-15 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the CNS
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US9752142B2 (en) 2011-08-11 2017-09-05 Ionis Pharmaceuticals, Inc. Gapped oligomeric compounds comprising 5′-modified deoxyribonucleosides in the gap and uses thereof
CN104203289B (zh) 2012-01-27 2020-11-03 比奥马林技术公司 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
JP6574383B2 (ja) 2012-12-05 2019-09-11 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． ＰＣＳＫ９ｉＲＮＡ組成物及びその使用方法
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
EP3146051B8 (fr) 2014-05-20 2019-11-27 University of Iowa Research Foundation Composés thérapeutiques pour la maladie de huntington
LT3237618T (lt) 2014-12-24 2019-07-10 Uniqure Ip B.V. Rnri sukeltas hantingtino geno slopinimas
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
CN114469984A (zh) 2015-08-25 2022-05-13 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
WO2020144611A1 (fr) 2019-01-09 2020-07-16 Universidade De Coimbra Arn double brin et utilisations correspondantes
US20220251567A1 (en) 2019-07-10 2022-08-11 Inserm (Institut National De La Santè Et De La Recherche Médicale) Methods for the treatment of epilepsy
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
AR125230A1 (es) * 2021-03-29 2023-06-28 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO

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Cited By (4)

* Cited by examiner, † Cited by third party
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US9260716B2 (en) 2002-08-05 2016-02-16 University Of Iowa Research Foundation RNA interference suppression of neurodegenerative diseases and methods of use thereof
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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

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