WO2008109368A3 - Nucleic acid compounds for inhibiting vegfr gene expression and uses thereof - Google Patents
Nucleic acid compounds for inhibiting vegfr gene expression and uses thereof Download PDFInfo
- Publication number
- WO2008109368A3 WO2008109368A3 PCT/US2008/055370 US2008055370W WO2008109368A3 WO 2008109368 A3 WO2008109368 A3 WO 2008109368A3 US 2008055370 W US2008055370 W US 2008055370W WO 2008109368 A3 WO2008109368 A3 WO 2008109368A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- nucleic acid
- gene expression
- vegfr
- acid compounds
- vegfr gene
- Prior art date
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Animal Behavior & Ethology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Pain & Pain Management (AREA)
- Rheumatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing VEGFR (e.g., FLTl, KDR, FLT4) gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a FLTl, KDR, or FLT4 mRNA. In addition, the meroduplex may have at least one uridine that is a 5-methyluridine, a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a VEGFR gene in a cell or in a subject to treat a VEGFR-related disease.
Priority Applications (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US12/552,082 US20100105134A1 (en) | 2007-03-02 | 2009-09-01 | Nucleic acid compounds for inhibiting gene expression and uses thereof |
US13/327,545 US20130011922A1 (en) | 2007-03-02 | 2011-12-15 | Nucleic acid compounds for inhibiting gene expression and uses thereof |
Applications Claiming Priority (10)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US93494007P | 2007-03-02 | 2007-03-02 | |
US60/934,940 | 2007-03-02 | ||
US93493007P | 2007-03-16 | 2007-03-16 | |
US60/934,930 | 2007-03-16 | ||
US93494107P | 2007-04-13 | 2007-04-13 | |
US60/934,941 | 2007-04-13 | ||
US93493207P | 2007-04-20 | 2007-04-20 | |
US93493307P | 2007-04-20 | 2007-04-20 | |
US60/934,932 | 2007-04-20 | ||
US60/934,933 | 2007-04-20 |
Related Parent Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2008/055362 Continuation-In-Part WO2008109362A1 (en) | 2007-03-02 | 2008-02-28 | Nucleic acid compounds for inhibiting vegf gene expression and uses thereof |
Related Child Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2008/055371 Continuation-In-Part WO2008109369A2 (en) | 2007-03-02 | 2008-02-28 | Nucleic acid compounds for inhibiting tnf gene expression and uses thereof |
US12/552,082 Continuation-In-Part US20100105134A1 (en) | 2007-03-02 | 2009-09-01 | Nucleic acid compounds for inhibiting gene expression and uses thereof |
AU2009212920A Division AU2009212920A1 (en) | 2007-03-02 | 2009-09-01 | Nucleic acid compounds for inhibiting gene expression and uses thereof |
Publications (3)
Publication Number | Publication Date |
---|---|
WO2008109368A2 WO2008109368A2 (en) | 2008-09-12 |
WO2008109368A3 true WO2008109368A3 (en) | 2008-12-11 |
WO2008109368A4 WO2008109368A4 (en) | 2009-01-29 |
Family
ID=39735091
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2008/055370 WO2008109368A2 (en) | 2007-03-02 | 2008-02-28 | Nucleic acid compounds for inhibiting vegfr gene expression and uses thereof |
Country Status (1)
Country | Link |
---|---|
WO (1) | WO2008109368A2 (en) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2008049078A1 (en) | 2006-10-18 | 2008-04-24 | Nastech Pharmaceutical Company Inc. | Nicked or gapped nucleic acid molecules and uses thereof |
US11541072B2 (en) | 2017-06-27 | 2023-01-03 | Massachusetts Eye And Ear Infirmary | AAV-CRISPR/Cas9 genome editing of VEGFR2 for treating ocular diseases |
Citations (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2003070910A2 (en) * | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) AND VEGF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
WO2005028649A1 (en) * | 2002-02-20 | 2005-03-31 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR AND VASCULAR ENDOTHELIAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US20060142230A1 (en) * | 2003-08-25 | 2006-06-29 | Nastech Pharmaceutical Company Inc. | Double-stranded ribonucleic acid molecules having ribothymidine |
WO2007056153A2 (en) * | 2005-11-04 | 2007-05-18 | Nastech Pharmaceutical Company Inc. | Peptide-dicer substrate rna conjugates as delivery vehicles for sirna |
WO2007107162A2 (en) * | 2006-03-23 | 2007-09-27 | Santaris Pharma A/S | Small internally segmented interfering rna |
WO2008049078A1 (en) * | 2006-10-18 | 2008-04-24 | Nastech Pharmaceutical Company Inc. | Nicked or gapped nucleic acid molecules and uses thereof |
-
2008
- 2008-02-28 WO PCT/US2008/055370 patent/WO2008109368A2/en active Application Filing
Patent Citations (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2003070910A2 (en) * | 2002-02-20 | 2003-08-28 | Ribozyme Pharmaceuticals, Incorporated | INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) AND VEGF RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
WO2005028649A1 (en) * | 2002-02-20 | 2005-03-31 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF VASCULAR ENDOTHELIAL GROWTH FACTOR AND VASCULAR ENDOTHELIAL GROWTH FACTOR RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US20060142230A1 (en) * | 2003-08-25 | 2006-06-29 | Nastech Pharmaceutical Company Inc. | Double-stranded ribonucleic acid molecules having ribothymidine |
WO2007056153A2 (en) * | 2005-11-04 | 2007-05-18 | Nastech Pharmaceutical Company Inc. | Peptide-dicer substrate rna conjugates as delivery vehicles for sirna |
WO2007107162A2 (en) * | 2006-03-23 | 2007-09-27 | Santaris Pharma A/S | Small internally segmented interfering rna |
WO2008049078A1 (en) * | 2006-10-18 | 2008-04-24 | Nastech Pharmaceutical Company Inc. | Nicked or gapped nucleic acid molecules and uses thereof |
Non-Patent Citations (4)
Title |
---|
BRAMSEN J B ET AL: "Improved silencing properties using small internally segmented interfering RNAs", NUCLEIC ACIDS RESEARCH, vol. 35, no. 17, 28 July 2007 (2007-07-28), pages 5886 - 5897, XP002467844, ISSN: 0305-1048 * |
LEUSCHNER P J F ET AL: "Cleavage of the siRNA passenger strand during RISC assembly in human cells", EMBO REPORTS, vol. 7, no. 3, 1 March 2006 (2006-03-01), pages 314 - 320, XP002467845 * |
MATRANGA CHRISTIAN ET AL: "Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes", CELL, vol. 123, no. 4, November 2005 (2005-11-01), pages 607 - 620, XP002484663, ISSN: 0092-8674 * |
MILLS JANINE B ET AL: "Origin of the intrinsic rigidity of DNA", NUCLEIC ACIDS RESEARCH, vol. 32, no. 13, 2004, pages 4055 - 4059, XP002484664, ISSN: 0305-1048 * |
Also Published As
Publication number | Publication date |
---|---|
WO2008109368A4 (en) | 2009-01-29 |
WO2008109368A2 (en) | 2008-09-12 |
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