WO2005014811A3 - RNA INTERFERENCE MEDIATED INHIBITION OF XIAP GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - Google Patents

RNA INTERFERENCE MEDIATED INHIBITION OF XIAP GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) Download PDF

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Publication number
WO2005014811A3
WO2005014811A3 PCT/US2004/025589 US2004025589W WO2005014811A3 WO 2005014811 A3 WO2005014811 A3 WO 2005014811A3 US 2004025589 W US2004025589 W US 2004025589W WO 2005014811 A3 WO2005014811 A3 WO 2005014811A3
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WO
WIPO (PCT)
Prior art keywords
nucleic acid
short interfering
sina
gene expression
rna
Prior art date
Application number
PCT/US2004/025589
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French (fr)
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WO2005014811A2 (en
Inventor
James Mcswiggen
Bharat M Chowrira
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Sirna Therapeutics Inc
James Mcswiggen
Bharat M Chowrira
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/en
Priority claimed from PCT/US2004/016390 external-priority patent/WO2005019453A2/en
Priority to US10/567,888 priority Critical patent/US20070093437A1/en
Application filed by Sirna Therapeutics Inc, James Mcswiggen, Bharat M Chowrira filed Critical Sirna Therapeutics Inc
Publication of WO2005014811A2 publication Critical patent/WO2005014811A2/en
Publication of WO2005014811A3 publication Critical patent/WO2005014811A3/en
Priority to US12/205,113 priority patent/US7897753B2/en

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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
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Y103/00—Oxidoreductases acting on the CH-CH group of donors (1.3)
    • C12Y103/01—Oxidoreductases acting on the CH-CH group of donors (1.3) with NAD+ or NADP+ as acceptor (1.3.1)
    • C12Y103/01022—3-Oxo-5alpha-steroid 4-dehydrogenase (NADP+) (1.3.1.22), i.e. cortisone alpha-reductase
    • 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
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    • C12N2310/31—Chemical structure of the backbone
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    • 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
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Abstract

This invention relates to compounds, compositions, and methods useful for modulating XIAP gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of XIAP gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features 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 and methods used to modulate the expression of XIAP genes.
PCT/US2004/025589 2001-05-18 2004-08-06 RNA INTERFERENCE MEDIATED INHIBITION OF XIAP GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) WO2005014811A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
US10/567,888 US20070093437A1 (en) 2001-05-18 2004-08-06 Rna interference mediated inhibition of xiap gene expression using short interfering nucleic acid (sina)
US12/205,113 US7897753B2 (en) 2002-02-20 2008-09-05 RNA interference mediated inhibition of XIAP gene expression using short interfering nucleic acid (siNA)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US49356103P 2003-08-08 2003-08-08
US60/493,561 2003-08-08
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)
US10/693,059 2003-10-23
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)
US10/720,448 2003-11-24
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 US7491805B2 (en) 2001-05-18 2004-02-13 Conjugates and compositions for cellular delivery
US10/780,447 2004-02-13
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)
PCT/US2004/013456 WO2005041859A2 (en) 2003-04-30 2004-04-30 Conjugates and compositions for cellular delivery.
USPCT/US04/13456 2004-04-30
PCT/US2004/016390 WO2005019453A2 (en) 2001-05-18 2004-05-24 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
USPCT/US04/16390 2004-05-24

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 (2)

Application Number Title Priority Date Filing Date
US10/567,888 A-371-Of-International US20070093437A1 (en) 2001-05-18 2004-08-06 Rna interference mediated inhibition of xiap gene expression using short interfering nucleic acid (sina)
US12/205,113 Continuation US7897753B2 (en) 2002-02-20 2008-09-05 RNA interference mediated inhibition of XIAP gene expression using short interfering nucleic acid (siNA)

Publications (2)

Publication Number Publication Date
WO2005014811A2 WO2005014811A2 (en) 2005-02-17
WO2005014811A3 true WO2005014811A3 (en) 2005-12-22

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PCT/US2004/025589 WO2005014811A2 (en) 2001-05-18 2004-08-06 RNA INTERFERENCE MEDIATED INHIBITION OF XIAP GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

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Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1765847A4 (en) 2004-05-27 2010-10-20 Alnylam Pharmaceuticals Inc Nuclease resistant double-stranded ribonucleic acid
CN1884530B (en) * 2006-07-04 2010-05-12 苏先狮 Human XIAP target gene siRNA expression template
US8367320B2 (en) * 2007-12-28 2013-02-05 Qiagen Sciences, Inc. Apoptosis inducing positive control for expression modulation experiments
KR101749356B1 (en) * 2009-05-18 2017-07-06 큐알엔에이, 인크. Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
ES2664591T3 (en) * 2009-09-25 2018-04-20 Curna, Inc. Treatment of phylagrin-related diseases (flg) by modulating the expression and activity of the FLG gene
SI2929031T1 (en) 2012-12-05 2018-02-28 Alnylam Pharmaceuticals, Inc. Pcsk9 irna compositions and methods of use thereof
IL310959A (en) 2015-08-25 2024-04-01 Alnylam Pharmaceuticals Inc Methods and compositions for treating a proprotein convertase subtilisin kexin (pcsk9) gene-associated disorder
CN110177544A (en) 2016-11-29 2019-08-27 普尔泰克健康有限公司 For delivering the excretion body of therapeutic agent
CA3091146A1 (en) * 2018-03-21 2019-09-26 Regeneron Pharmaceuticals, Inc. 17.beta.-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof

Citations (3)

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WO2000005366A2 (en) * 1998-07-24 2000-02-03 University Of Ottawa Xiap ires and uses thereof
WO2000044895A1 (en) * 1999-01-30 2000-08-03 Roland Kreutzer Method and medicament for inhibiting the expression of a defined gene
WO2002044321A2 (en) * 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna interference mediating small rna molecules

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000005366A2 (en) * 1998-07-24 2000-02-03 University Of Ottawa Xiap ires and uses thereof
WO2000044895A1 (en) * 1999-01-30 2000-08-03 Roland Kreutzer Method and medicament for inhibiting the expression of a defined gene
WO2002044321A2 (en) * 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna interference mediating small rna molecules

Non-Patent Citations (6)

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Title
BURSTEIN EZRA ET AL: "A novel role for XIAP in copper homeostasis through regulation of MURR1.", THE EMBO JOURNAL. 14 JAN 2004, vol. 23, no. 1, 14 January 2004 (2004-01-14), pages 244 - 254, XP002332238, ISSN: 0261-4189 *
CHAWLA-SARKAR M ET AL: "Trail-resistant cells sensitized to apoptosis by selective down regulation by siRNAs to inhibitors of apoptosis bcl-2, FLIP, survivin or XIAP.", EUROPEAN CYTOKINE NETWORK, vol. 14, no. Supplement 3, September 2003 (2003-09-01), & ANNUAL MEETING OF THE INTERNATIONAL CYTOKINE SOCIETY; DUBLIN, IRELAND; SEPTEMBER 20-24, 2003, pages 112, XP009042059, ISSN: 1148-5493 *
ELBASHIR S M ET AL: "Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate", EMBO JOURNAL, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 20, no. 23, 3 December 2001 (2001-12-03), pages 6877 - 6888, XP002225998, ISSN: 0261-4189 *
LIMA R T ET AL: "RNAi mediated downregulation of bcl-2 and xlAP may have therapeutical potential in human breast adenocarcinoma.", EJC SUPPLEMENTS, vol. 1, no. 5, September 2003 (2003-09-01), & 12TH ECCO (EUROPEAN CANCER CONFERENCE); COPENHAGEN, DENMARK; SEPTEMBER 21-25, 2003, pages S292, XP009049224, ISSN: 1359-6349 *
LIMA RAQUEL T ET AL: "Specific downregulation of bcl-2 and xIAP by RNAi enhances the effects of chemotherapeutic agents in MCF-7 human breast cancer cells", CANCER GENE THERAPY, vol. 11, no. 5, May 2004 (2004-05-01), pages 309 - 316, XP002332239, ISSN: 0929-1903 *
PARRISH S ET AL: "Functional anatomy of a dsRNA trigger: Differential requirement for the two trigger strands in RNA interference", MOLECULAR CELL, CELL PRESS, CAMBRIDGE, MA, US, vol. 6, no. 5, November 2000 (2000-11-01), pages 1077 - 1087, XP002226298, ISSN: 1097-2765 *

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