WO2005035759A3 - RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - Google Patents

RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) Download PDF

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Publication number
WO2005035759A3
WO2005035759A3 PCT/US2004/027294 US2004027294W WO2005035759A3 WO 2005035759 A3 WO2005035759 A3 WO 2005035759A3 US 2004027294 W US2004027294 W US 2004027294W WO 2005035759 A3 WO2005035759 A3 WO 2005035759A3
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WIPO (PCT)
Prior art keywords
nucleic acid
hif1
short interfering
sina
gene expression
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PCT/US2004/027294
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French (fr)
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WO2005035759A2 (en
WO2005035759A8 (en
Inventor
Nassim Usman
James Mcswiggen
Original Assignee
Nassim Usman
James Mcswiggen
Sirna Therapeutics Inc
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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
Application filed by Nassim Usman, James Mcswiggen, Sirna Therapeutics Inc filed Critical Nassim Usman
Publication of WO2005035759A2 publication Critical patent/WO2005035759A2/en
Publication of WO2005035759A8 publication Critical patent/WO2005035759A8/en
Publication of WO2005035759A3 publication Critical patent/WO2005035759A3/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
    • C12N15/113—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
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    • 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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    • C12N15/11—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
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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
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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Abstract

This invention relates to compounds, compositions, and methods useful for modulating hyproxia inducable factor (e.g., HIF1) 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 HIF1 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 HIF1 genes.
PCT/US2004/027294 2003-08-20 2004-08-20 RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) WO2005035759A2 (en)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US49665503P 2003-08-20 2003-08-20
US60/496,655 2003-08-20
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 2003-12-03
US10/727,780 US20050233329A1 (en) 2002-02-20 2003-12-03 Inhibition of gene expression using duplex forming oligonucleotides
US10/757,803 2004-01-14
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)
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)
PCT/US2004/013456 WO2005041859A2 (en) 2003-04-30 2004-04-30 Conjugates and compositions for cellular delivery.
USPCT/US2004/013456 2004-04-30
USPCT/US2004/016390 2004-05-24
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)

Publications (3)

Publication Number Publication Date
WO2005035759A2 WO2005035759A2 (en) 2005-04-21
WO2005035759A8 WO2005035759A8 (en) 2006-02-02
WO2005035759A3 true WO2005035759A3 (en) 2006-03-16

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PCT/US2004/027294 WO2005035759A2 (en) 2003-08-20 2004-08-20 RNA INTERFERENCE MEDIATED INHIBITION OF HYPOXIA INDUCIBLE FACTOR 1 (HIF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

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Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7144999B2 (en) * 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
CA2551100A1 (en) 2003-12-23 2005-07-14 The Trustees Of The University Of Pennsylvania Compositions and methods for combined therapy of disease
TW200731980A (en) 2005-12-29 2007-09-01 Alcon Mfg Ltd RNAi-mediated inhibition of HIF1A for treatment of ocular angiogenesis
FR2944437B1 (en) * 2009-04-16 2013-05-10 Oreal USE OF HIF 1 ALPHA EXPRESSION INHIBITORS TO PROTECT THE SKIN FROM DERMAL DAMAGE INDUCED BY UVA RADIATION
KR20200035490A (en) 2012-12-05 2020-04-03 알닐람 파마슈티칼스 인코포레이티드 PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CN114668774A (en) 2015-08-25 2022-06-28 阿尔尼拉姆医药品有限公司 Methods and compositions for treating proprotein convertase subtilisin KEXIN (PCSK9) gene-related disorders
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
WO2023001234A1 (en) * 2021-07-22 2023-01-26 Microbio (Shanghai) Co., Ltd. Modified small interfering rna molecules with reduced off-target effects

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002044321A2 (en) * 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna interference mediating small rna molecules
WO2004042024A2 (en) * 2002-11-01 2004-05-21 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF HIF-1 ALPHA

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002044321A2 (en) * 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna interference mediating small rna molecules
WO2004042024A2 (en) * 2002-11-01 2004-05-21 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF HIF-1 ALPHA

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
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 *
FUKUDA RYO ET AL: "Vascular endothelial growth factor gene expression in colon cancer cells exposed to prostaglandin E2 is mediated by hypoxia-inducible factor 1.", CANCER RESEARCH, vol. 63, no. 9, 1 May 2003 (2003-05-01), pages 2330 - 2334, XP002341114, ISSN: 0008-5472 *
HANZE J ET AL: "RNA interference for HIF-1alpha inhibits its downstream signalling and affects cellular proliferation", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, ACADEMIC PRESS INC. ORLANDO, FL, US, vol. 312, no. 3, 19 December 2003 (2003-12-19), pages 571 - 577, XP004474884, ISSN: 0006-291X *
KRISHNAMACHARY BALAJI ET AL: "Regulation of colon carcinoma cell invasion by hypoxia-inducible factor 1.", CANCER RESEARCH, vol. 63, no. 5, 1 March 2003 (2003-03-01), pages 1138 - 1143, XP002341115, ISSN: 0008-5472 *
SOWTER HEIDI M ET AL: "Predominant role of hypoxia-inducible transcription factor (Hif)-1alpha versus Hif-2alpha in regulation of the transcriptional response to hypoxia.", CANCER RESEARCH. 1 OCT 2003, vol. 63, no. 19, 1 October 2003 (2003-10-01), pages 6130 - 6134, XP002341116, ISSN: 0008-5472 *

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