WO2003070885A3 - SHORT INTERFERING NUCLEIC ACID INHIBITION OF STEAROYL-CoA DESATURASE (SCD) GENE - Google Patents

SHORT INTERFERING NUCLEIC ACID INHIBITION OF STEAROYL-CoA DESATURASE (SCD) GENE Download PDF

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Publication number
WO2003070885A3
WO2003070885A3 PCT/US2003/004317 US0304317W WO03070885A3 WO 2003070885 A3 WO2003070885 A3 WO 2003070885A3 US 0304317 W US0304317 W US 0304317W WO 03070885 A3 WO03070885 A3 WO 03070885A3
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WIPO (PCT)
Prior art keywords
scd
nucleic acid
rna
short interfering
coa desaturase
Prior art date
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PCT/US2003/004317
Other languages
French (fr)
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WO2003070885A2 (en
Inventor
James Mcswiggen
Leonid Beigelman
James Thompson
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Sirna Therapeutics Inc
James Mcswiggen
Leonid Beigelman
James Thompson
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Publication date
Application filed by Sirna Therapeutics Inc, James Mcswiggen, Leonid Beigelman, James Thompson filed Critical Sirna Therapeutics Inc
Priority to AU2003215203A priority Critical patent/AU2003215203A1/en
Priority to EP03711017A priority patent/EP1499628A4/en
Publication of WO2003070885A2 publication Critical patent/WO2003070885A2/en
Priority to US10/923,451 priority patent/US20050256068A1/en
Publication of WO2003070885A3 publication Critical patent/WO2003070885A3/en
Priority to US12/192,853 priority patent/US8017761B2/en

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
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    • 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
    • C12N15/1131—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 viruses
    • C12N15/1132—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 viruses against retroviridae, e.g. HIV
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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
    • C—CHEMISTRY; METALLURGY
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    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/19—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with oxidation of a pair of donors resulting in the reduction of molecular oxygen to two molecules of water (1.14.19)
    • C12Y114/19001—Stearoyl-CoA 9-desaturase (1.14.19.1), i.e. DELTA9-desaturase
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • C—CHEMISTRY; METALLURGY
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
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    • C12Y207/11013—Protein kinase C (2.7.11.13)
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    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03048—Protein-tyrosine-phosphatase (3.1.3.48)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
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    • C12N2310/121—Hammerhead
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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Abstract

The present invention concerns methods and reagents useful in modulating Stearoyl-CoA desaturase (SCD) gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against Steraroyl-CoA desaturase (SCD) gene expression and/or activity. The siNA molecules are useful in the treatment of diabetes (type I and/or type II), atherosclerosis, cancer, obesity, and viral infection, and any other condition that responds to modulation of SCD expression or activity.
PCT/US2003/004317 2001-05-18 2003-02-13 SHORT INTERFERING NUCLEIC ACID INHIBITION OF STEAROYL-CoA DESATURASE (SCD) GENE WO2003070885A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
AU2003215203A AU2003215203A1 (en) 2002-02-20 2003-02-13 Short interfering nucleic acid inhibition of stearoyl-coa desaturase (scd) gene
EP03711017A EP1499628A4 (en) 2002-02-20 2003-02-13 Rna interference mediated inhibition of stearoyl-coa desaturase (scd) gene expression using short interfering nucleic acid (sina)
US10/923,451 US20050256068A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of stearoyl-CoA desaturase (SCD) gene expression using short interfering nucleic acid (siNA)
US12/192,853 US8017761B2 (en) 2001-05-18 2008-08-15 RNA interference mediated inhibition of Stearoyl-CoA desaturase (SCD) gene expression using short interfering nucelic acid (siNA)

Applications Claiming Priority (16)

Application Number Priority Date Filing Date Title
US35858002P 2002-02-20 2002-02-20
US60/358,580 2002-02-20
US36312402P 2002-03-11 2002-03-11
US60/363,124 2002-03-11
US38678202P 2002-06-06 2002-06-06
US60/386,782 2002-06-06
US40678402P 2002-08-29 2002-08-29
US60/406,784 2002-08-29
US40837802P 2002-09-05 2002-09-05
US60/408,378 2002-09-05
US40929302P 2002-09-09 2002-09-09
US60/409,293 2002-09-09
US41230402P 2002-09-20 2002-09-20
US60/412,304 2002-09-20
US44012903P 2003-01-15 2003-01-15
US60/440,129 2003-01-15

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2004/016390 Continuation-In-Part WO2005019453A2 (en) 2000-02-11 2004-05-24 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US10/923,451 Continuation-In-Part US20050256068A1 (en) 2001-05-18 2004-08-20 RNA interference mediated inhibition of stearoyl-CoA desaturase (SCD) gene expression using short interfering nucleic acid (siNA)

Publications (2)

Publication Number Publication Date
WO2003070885A2 WO2003070885A2 (en) 2003-08-28
WO2003070885A3 true WO2003070885A3 (en) 2004-11-11

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PCT/US2003/004317 WO2003070885A2 (en) 2001-05-18 2003-02-13 SHORT INTERFERING NUCLEIC ACID INHIBITION OF STEAROYL-CoA DESATURASE (SCD) GENE

Country Status (3)

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EP (1) EP1499628A4 (en)
AU (1) AU2003215203A1 (en)
WO (1) WO2003070885A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11970486B2 (en) 2017-10-24 2024-04-30 Janssen Pharmaceutica Nv Compounds and uses thereof

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050043256A1 (en) 2001-07-30 2005-02-24 Isis Pharmaceuticals, Inc. Antisense modulation of stearoyl-CoA desaturase expression
WO2011030312A1 (en) * 2009-09-10 2011-03-17 Institut National De La Sante Et De La Recherche Medicale (Inserm) NOVEL INHIBITORS OF STEAROYL-CoA-DESATURASE-1 AND THEIR USES
KR20190108118A (en) 2017-01-06 2019-09-23 유마니티 테라퓨틱스, 인크. Methods for the Treatment of Nervous System Disorders
CA3083000A1 (en) 2017-10-24 2019-05-02 Yumanity Therapeutics, Inc. Compounds and uses thereof

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WO2001062954A2 (en) * 2000-02-24 2001-08-30 Xenon Genetics, Inc. Stearoyl-coa desaturase to identify triglyceride reducing therapeutic agents

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JP2003533965A (en) * 1998-08-14 2003-11-18 ジョンソン・アンド・ジョンソン・コンシューマー・カンパニーズ・インコーポレイテッド Human stearoyl-CoA desaturase-related compositions and methods for treating abnormal skin conditions

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WO2001062954A2 (en) * 2000-02-24 2001-08-30 Xenon Genetics, Inc. Stearoyl-coa desaturase to identify triglyceride reducing therapeutic agents

Non-Patent Citations (3)

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Title
ELBASHIR S. ET AL: "Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells", NATURE, vol. 411, May 2001 (2001-05-01), pages 494 - 498, XP002213433 *
FIRE A. ET AL: "Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans", NATURE, vol. 391, February 1998 (1998-02-01), pages 806 - 811, XP002938528 *
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11970486B2 (en) 2017-10-24 2024-04-30 Janssen Pharmaceutica Nv Compounds and uses thereof

Also Published As

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EP1499628A4 (en) 2005-06-15
WO2003070885A2 (en) 2003-08-28
AU2003215203A1 (en) 2003-09-09
AU2003215203A8 (en) 2003-09-09
EP1499628A2 (en) 2005-01-26

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