WO2005045032A3 - 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) - Google Patents

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) Download PDF

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Publication number
WO2005045032A3
WO2005045032A3 PCT/US2004/026941 US2004026941W WO2005045032A3 WO 2005045032 A3 WO2005045032 A3 WO 2005045032A3 US 2004026941 W US2004026941 W US 2004026941W WO 2005045032 A3 WO2005045032 A3 WO 2005045032A3
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WIPO (PCT)
Prior art keywords
nucleic acid
short interfering
sina
gene expression
rna
Prior art date
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PCT/US2004/026941
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English (en)
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WO2005045032A2 (fr
Inventor
Nassim Usman
James Mcswiggen
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Sirna Therapeutics Inc
Nassim Usman
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
Priority claimed from US10/693,059 external-priority patent/US20080039414A1/en
Priority claimed from US10/720,448 external-priority patent/US8273866B2/en
Priority claimed from US10/727,780 external-priority patent/US20050233329A1/en
Priority claimed from US10/757,803 external-priority patent/US20050020525A1/en
Priority claimed from US10/780,447 external-priority patent/US7491805B2/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
Application filed by Sirna Therapeutics Inc, Nassim Usman, James Mcswiggen filed Critical Sirna Therapeutics Inc
Publication of WO2005045032A2 publication Critical patent/WO2005045032A2/fr
Publication of WO2005045032A3 publication Critical patent/WO2005045032A3/fr

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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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
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    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
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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
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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
    • 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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    • 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
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    • C12N2310/121—Hammerhead
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
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  • Gastroenterology & Hepatology (AREA)
  • Pathology (AREA)
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Abstract

L'invention concerne des composés, des compositions et des méthodes utiles pour moduler l'expression du gène de réponse de croissance précoce (Egr-1), au moyen de molécules de petit acide nucléique interférent (siNA). Cette invention concerne également des composés, des compositions et des méthodes utiles pour moduler l'expression et l'activité d'autres gènes impliqués dans les voies de l'expression et/ou de l'activité du gène Egr-1, par une interférence ARN (RNAi), au moyen de molécules de petit acide nucléique. En particulier, l'invention concerne des molécules de petit acide nucléique, notamment un petit acide nucléique interférent (siNA), un petit ARN interférent (siRNA), un ARN bicaténaire (dsRNA), un micro-ARN (miRNA), et des molécules de ARN en épingle (shRNA), ainsi que des méthodes utilisées pour moduler l'expression du gène de réponse de croissance précoce Egr-1.
PCT/US2004/026941 2003-10-20 2004-08-19 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) WO2005045032A2 (fr)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US51270103P 2003-10-20 2003-10-20
US60/512,701 2003-10-20
US10/693,059 2003-10-23
US10/693,059 US20080039414A1 (en) 2002-02-20 2003-10-23 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US10/720,448 2003-11-24
US10/720,448 US8273866B2 (en) 2002-02-20 2003-11-24 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US10/727,780 US20050233329A1 (en) 2002-02-20 2003-12-03 Inhibition of gene expression using duplex forming oligonucleotides
US10/727,780 2003-12-03
US10/757,803 US20050020525A1 (en) 2002-02-20 2004-01-14 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US10/757,803 2004-01-14
US54348004P 2004-02-10 2004-02-10
US60/543,480 2004-02-10
US10/780,447 2004-02-13
US10/780,447 US7491805B2 (en) 2001-05-18 2004-02-13 Conjugates and compositions for cellular delivery
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/US04/013456 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/US04/016390 2004-05-24

Publications (2)

Publication Number Publication Date
WO2005045032A2 WO2005045032A2 (fr) 2005-05-19
WO2005045032A3 true WO2005045032A3 (fr) 2006-03-02

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PCT/US2004/026941 WO2005045032A2 (fr) 2003-10-20 2004-08-19 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)

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Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2422819B1 (fr) 2006-01-26 2017-03-01 Ionis Pharmaceuticals, Inc. Compositions et leurs utilisations dirigées vers l'huntingtine
WO2007106488A2 (fr) * 2006-03-13 2007-09-20 Wyeth Modulateurs de la gluconeogenese
WO2008109526A1 (fr) * 2007-03-02 2008-09-12 Mdrna, Inc. Composés d'acides nucléiques inhibant l'expression du gène egr et leurs utilisations
CA2732343C (fr) 2008-07-29 2017-05-09 The Board Of Regents Of The University Of Texas System Inhibition selective d'expression de proteine de polyglutamine
RU2751847C9 (ru) 2009-09-11 2021-08-20 Ионис Фармасьютикалз, Инк. Модуляция экспрессии гентингтина
WO2013022966A1 (fr) 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Composés oligomères à brèche modifiés par liaison et leurs utilisations
ITMI20120275A1 (it) * 2012-02-24 2013-08-25 Biogenera Societa A Responsabilita Limitata Oligonucleotidi per la modulazione dell'espressione genica e loro usi
MX367076B (es) 2012-12-05 2019-08-05 Alnylam Pharmaceuticals Inc Composiciones de arni de proteina convertasa subtilisina kexina 9 (pcsk9) y metodos de uso de las mismas.
EP3340994A1 (fr) 2015-08-25 2018-07-04 Alnylam Pharmaceuticals, Inc. Procédés et compositions pour le traitement d'un trouble associé à un gène de proprotéine convertase subtilisine kexine (pcsk9)
EP4035659A1 (fr) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes destinés à l'administration d'agents thérapeutiques
WO2021081218A1 (fr) * 2019-10-22 2021-04-29 Aim Immunotech Inc. Procédés et compositions pour le traitement de l'endométriose

Citations (3)

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WO2002044321A2 (fr) * 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Petites molecules d'arn mediant l'interference arn
US20030157030A1 (en) * 2001-11-02 2003-08-21 Insert Therapeutics, Inc. Methods and compositions for therapeutic use of rna interference
WO2003070918A2 (fr) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Inhibition mediee par interference arn d'une expression genique faisant appel a des acides nucleiques interferants courts chimiquement modifies (sina)

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Publication number Priority date Publication date Assignee Title
WO2002044321A2 (fr) * 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Petites molecules d'arn mediant l'interference arn
US20030157030A1 (en) * 2001-11-02 2003-08-21 Insert Therapeutics, Inc. Methods and compositions for therapeutic use of rna interference
WO2003070918A2 (fr) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Inhibition mediee par interference arn d'une expression genique faisant appel a des acides nucleiques interferants courts chimiquement modifies (sina)

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Title
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GIRI RANJIT K ET AL: "Amyloid peptide-induced cytokine and chemokine expression in THP-1 monocytes is blocked by small inhibitory RNA duplexes for early growth response-1 messenger RNA.", JOURNAL OF IMMUNOLOGY, vol. 170, no. 10, 15 May 2003 (2003-05-15), pages 5281 - 5294, XP002340050, ISSN: 0022-1767 *
NANDAN MANDAYAM O ET AL: "Kruppel-like factor 5 mediates the transforming activity of oncogenic H-Ras", ONCOGENE, vol. 23, no. 19, 22 April 2004 (2004-04-22), pages 3404 - 3413, XP002340051, ISSN: 0950-9232 *
SUBBARAMAIAH KOTHA ET AL: "Microsomal prostaglandin E synthase-1 is overexpressed in inflammatory bowel disease. Evidence for involvement of the transcription factor Egr-1.", THE JOURNAL OF BIOLOGICAL CHEMISTRY. 26 MAR 2004, vol. 279, no. 13, 26 March 2004 (2004-03-26), pages 12647 - 12658, XP002340052, ISSN: 0021-9258 *
YANG SHAN-ZHONG ET AL: "Early growth response gene 1 modulates androgen receptor signaling in prostate carcinoma cells.", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 278, no. 41, 10 October 2003 (2003-10-10), pages 39906 - 39911, XP002340049, ISSN: 0021-9258 *

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