WO2010151674A2 - Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene - Google Patents
Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene Download PDFInfo
- Publication number
- WO2010151674A2 WO2010151674A2 PCT/US2010/039827 US2010039827W WO2010151674A2 WO 2010151674 A2 WO2010151674 A2 WO 2010151674A2 US 2010039827 W US2010039827 W US 2010039827W WO 2010151674 A2 WO2010151674 A2 WO 2010151674A2
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- oligonucleotide
- down syndrome
- syndrome gene
- expression
- nucleic acid
- 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
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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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]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/12—Drugs for disorders of the metabolism for electrolyte homeostasis
- A61P3/14—Drugs for disorders of the metabolism for electrolyte homeostasis for calcium homeostasis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P39/00—General protective or antinoxious agents
- A61P39/06—Free radical scavengers or antioxidants
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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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
- C12N15/1137—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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- 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
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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
- C12N2310/14—Type of nucleic acid interfering N.A.
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- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
- Y10T—TECHNICAL SUBJECTS COVERED BY FORMER US CLASSIFICATION
- Y10T436/00—Chemistry: analytical and immunological testing
- Y10T436/14—Heterocyclic carbon compound [i.e., O, S, N, Se, Te, as only ring hetero atom]
- Y10T436/142222—Hetero-O [e.g., ascorbic acid, etc.]
- Y10T436/143333—Saccharide [e.g., DNA, etc.]
Abstract
Description
Claims
Priority Applications (10)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CN201080038661.3A CN102482672B (en) | 2009-06-26 | 2010-06-24 | By suppressing the natural antisense transcript treatment Down syndrome gene-associated diseases of Down syndrome gene |
KR1020127001652A KR101807324B1 (en) | 2009-06-26 | 2010-06-24 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
ES10792668.5T ES2583691T3 (en) | 2009-06-26 | 2010-06-24 | Treatment of diseases related to a Down syndrome gene by inhibiting a natural antisense transcript to a Down syndrome gene |
EP10792668.5A EP2446037B1 (en) | 2009-06-26 | 2010-06-24 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
US13/379,743 US8921330B2 (en) | 2009-06-26 | 2010-06-24 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
CA2765815A CA2765815A1 (en) | 2009-06-26 | 2010-06-24 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
JP2012517732A JP5907866B2 (en) | 2009-06-26 | 2010-06-24 | Treatment of Down syndrome gene-related diseases by repression of natural antisense transcripts for Down syndrome genes |
US14/546,828 US10036014B2 (en) | 2009-06-26 | 2014-11-18 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
US16/026,359 US10450567B2 (en) | 2009-06-26 | 2018-07-03 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
US16/579,160 US10876117B2 (en) | 2009-06-26 | 2019-09-23 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US22074709P | 2009-06-26 | 2009-06-26 | |
US61/220,747 | 2009-06-26 | ||
US23575209P | 2009-08-21 | 2009-08-21 | |
US61/235,752 | 2009-08-21 |
Related Child Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US13/379,743 A-371-Of-International US8921330B2 (en) | 2009-06-26 | 2010-06-24 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
US14/546,828 Division US10036014B2 (en) | 2009-06-26 | 2014-11-18 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2010151674A2 true WO2010151674A2 (en) | 2010-12-29 |
WO2010151674A3 WO2010151674A3 (en) | 2011-07-21 |
Family
ID=43387128
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2010/039827 WO2010151674A2 (en) | 2009-06-26 | 2010-06-24 | Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene |
Country Status (8)
Country | Link |
---|---|
US (4) | US8921330B2 (en) |
EP (1) | EP2446037B1 (en) |
JP (1) | JP5907866B2 (en) |
KR (1) | KR101807324B1 (en) |
CN (1) | CN102482672B (en) |
CA (1) | CA2765815A1 (en) |
ES (1) | ES2583691T3 (en) |
WO (1) | WO2010151674A2 (en) |
Cited By (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9816094B2 (en) | 2010-11-12 | 2017-11-14 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
US10058623B2 (en) | 2012-05-16 | 2018-08-28 | Translate Bio Ma, Inc. | Compositions and methods for modulating UTRN expression |
US10059941B2 (en) | 2012-05-16 | 2018-08-28 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
US10174315B2 (en) | 2012-05-16 | 2019-01-08 | The General Hospital Corporation | Compositions and methods for modulating hemoglobin gene family expression |
US10174323B2 (en) | 2012-05-16 | 2019-01-08 | The General Hospital Corporation | Compositions and methods for modulating ATP2A2 expression |
EP3511416A1 (en) | 2012-05-16 | 2019-07-17 | Translate Bio MA, Inc. | Compositions and methods for modulating gene expression |
US10655128B2 (en) | 2012-05-16 | 2020-05-19 | Translate Bio Ma, Inc. | Compositions and methods for modulating MECP2 expression |
US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
US10858650B2 (en) | 2014-10-30 | 2020-12-08 | The General Hospital Corporation | Methods for modulating ATRX-dependent gene repression |
US10900036B2 (en) | 2015-03-17 | 2021-01-26 | The General Hospital Corporation | RNA interactome of polycomb repressive complex 1 (PRC1) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US8212019B2 (en) | 2008-07-30 | 2012-07-03 | University Of Massachusetts | Nucleic acid silencing sequences |
WO2014152607A2 (en) * | 2013-03-14 | 2014-09-25 | University Of Massachusetts | Dosage compensating transgenes and cells |
US9681646B2 (en) | 2013-03-15 | 2017-06-20 | University Of Massachusetts | Dosage compensating transgenes and cells |
CN108384787B (en) * | 2018-03-09 | 2020-05-19 | 山东大学深圳研究院 | RCAN1 aptamer RCAN1-s14 |
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US10858650B2 (en) | 2014-10-30 | 2020-12-08 | The General Hospital Corporation | Methods for modulating ATRX-dependent gene repression |
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EP2446037A2 (en) | 2012-05-02 |
ES2583691T3 (en) | 2016-09-21 |
KR101807324B1 (en) | 2017-12-08 |
US10876117B2 (en) | 2020-12-29 |
US20200032263A1 (en) | 2020-01-30 |
US20150073040A1 (en) | 2015-03-12 |
US10450567B2 (en) | 2019-10-22 |
US20120142758A1 (en) | 2012-06-07 |
WO2010151674A3 (en) | 2011-07-21 |
JP2012531427A (en) | 2012-12-10 |
KR20120086282A (en) | 2012-08-02 |
US10036014B2 (en) | 2018-07-31 |
US20180305693A1 (en) | 2018-10-25 |
CN102482672B (en) | 2016-11-09 |
CN102482672A (en) | 2012-05-30 |
EP2446037A4 (en) | 2013-11-06 |
CA2765815A1 (en) | 2010-12-29 |
US8921330B2 (en) | 2014-12-30 |
JP5907866B2 (en) | 2016-04-26 |
EP2446037B1 (en) | 2016-04-20 |
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