WO2005045039A3 - RNA INTERFERENCE MEDIATED INHIBITION OF INTERCELLULAR ADHESION MOLECULE (ICAM) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - Google Patents

RNA INTERFERENCE MEDIATED INHIBITION OF INTERCELLULAR ADHESION MOLECULE (ICAM) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) Download PDF

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Publication number
WO2005045039A3
WO2005045039A3 PCT/US2004/027366 US2004027366W WO2005045039A3 WO 2005045039 A3 WO2005045039 A3 WO 2005045039A3 US 2004027366 W US2004027366 W US 2004027366W WO 2005045039 A3 WO2005045039 A3 WO 2005045039A3
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WO
WIPO (PCT)
Prior art keywords
nucleic acid
icam
short interfering
sina
gene expression
Prior art date
Application number
PCT/US2004/027366
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French (fr)
Other versions
WO2005045039A9 (en
WO2005045039B1 (en
WO2005045039A2 (en
Inventor
Ivan Richards
James Mcswiggen
Original Assignee
Ivan Richards
James Mcswiggen
Sirna Therapeutics Inc
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 US10/800,487 external-priority patent/US20050048529A1/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 Ivan Richards, James Mcswiggen, Sirna Therapeutics Inc filed Critical Ivan Richards
Publication of WO2005045039A2 publication Critical patent/WO2005045039A2/en
Publication of WO2005045039A9 publication Critical patent/WO2005045039A9/en
Publication of WO2005045039A3 publication Critical patent/WO2005045039A3/en
Publication of WO2005045039B1 publication Critical patent/WO2005045039B1/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
    • 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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    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

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  • Genetics & Genomics (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Public Health (AREA)
  • Rheumatology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

This invention relates to compounds, compositions, and methods useful for modulating intercellular adhesion molecule (ICAM) 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 ICAM 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 ICAM genes.
PCT/US2004/027366 2003-10-23 2004-08-20 RNA INTERFERENCE MEDIATED INHIBITION OF INTERCELLULAR ADHESION MOLECULE (ICAM) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) WO2005045039A2 (en)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
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)
US10720,448 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
US10/800,487 2004-03-15
US10/800,487 US20050048529A1 (en) 2002-02-20 2004-03-15 RNA interference mediated inhibition of intercellular adhesion molecule (ICAM) gene expression 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 (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

Publications (4)

Publication Number Publication Date
WO2005045039A2 WO2005045039A2 (en) 2005-05-19
WO2005045039A9 WO2005045039A9 (en) 2005-07-21
WO2005045039A3 true WO2005045039A3 (en) 2006-01-12
WO2005045039B1 WO2005045039B1 (en) 2006-02-16

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2004/027366 WO2005045039A2 (en) 2003-10-23 2004-08-20 RNA INTERFERENCE MEDIATED INHIBITION OF INTERCELLULAR ADHESION MOLECULE (ICAM) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

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Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4903146B2 (en) 2004-08-16 2012-03-28 イミューン ディズィーズ インスティテュート インコーポレイテッド Methods for delivering RNA interference and uses thereof
AU2011336467A1 (en) 2010-12-01 2013-07-04 Spinal Modulation, Inc. Agent delivery systems for selective neuromodulation
WO2013123996A1 (en) * 2012-02-24 2013-08-29 Astrazeneca Uk Limited Novel sirna inhibitors of human icam-1
EP3336187A1 (en) 2012-12-05 2018-06-20 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
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2004065546A2 (en) * 2003-01-16 2004-08-05 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ICAM-1

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2004065546A2 (en) * 2003-01-16 2004-08-05 The Trustees Of The University Of Pennsylvania COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ICAM-1

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
FAR ROSEL KRETSCHMER-KAZEMI ET AL: "The activity of siRNA in mammalian cells is related to structural target accessibility: A comparison with antisense oligonucleotides", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 31, no. 15, 1 August 2003 (2003-08-01), pages 4417 - 4424, XP002317031, ISSN: 0305-1048 *
WARREN DANIEL S ET AL: "Successful ICAM-1 gene inactivation in pluripotent stem cells using RNA interference and in situ expressed antisense/ribozyme transgenes", JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, vol. 13, no. Program and Abstracts Issue, September 2002 (2002-09-01), & MEETING OF THE AMERICAN SOCIETY OF NEPHROLOGY; PHILADELPHIA, PA, USA; OCTOBER 30-NOVEMBER 04, 2002, pages 101A, XP009047818, ISSN: 1046-6673 *

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WO2005045039A2 (en) 2005-05-19

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