CN102869386A - 用于治疗红细胞增多症的antimiR-451 - Google Patents

用于治疗红细胞增多症的antimiR-451 Download PDF

Info

Publication number
CN102869386A
CN102869386A CN2011800142685A CN201180014268A CN102869386A CN 102869386 A CN102869386 A CN 102869386A CN 2011800142685 A CN2011800142685 A CN 2011800142685A CN 201180014268 A CN201180014268 A CN 201180014268A CN 102869386 A CN102869386 A CN 102869386A
Authority
CN
China
Prior art keywords
mir
seq
sequence
antisense oligonucleotide
subject
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN2011800142685A
Other languages
English (en)
Chinese (zh)
Inventor
E.N.奥尔森
D.M.帕特里克
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Texas System
Original Assignee
University of Texas System
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
Application filed by University of Texas System filed Critical University of Texas System
Publication of CN102869386A publication Critical patent/CN102869386A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Hematology (AREA)
  • Cardiology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Oncology (AREA)
  • Hospice & Palliative Care (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN2011800142685A 2010-01-20 2011-01-20 用于治疗红细胞增多症的antimiR-451 Pending CN102869386A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US29678310P 2010-01-20 2010-01-20
US61/296,783 2010-01-20
US28254610P 2010-02-26 2010-02-26
US61/282,546 2010-02-26
PCT/US2011/021886 WO2012148373A1 (en) 2010-01-20 2011-01-20 Antimir-451 for the treatment of polycythemias

Publications (1)

Publication Number Publication Date
CN102869386A true CN102869386A (zh) 2013-01-09

Family

ID=47072618

Family Applications (1)

Application Number Title Priority Date Filing Date
CN2011800142685A Pending CN102869386A (zh) 2010-01-20 2011-01-20 用于治疗红细胞增多症的antimiR-451

Country Status (5)

Country Link
US (1) US9340782B2 (enExample)
EP (1) EP2536436A4 (enExample)
JP (1) JP2013530160A (enExample)
CN (1) CN102869386A (enExample)
WO (1) WO2012148373A1 (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113332434A (zh) * 2021-05-25 2021-09-03 大连医科大学 miR-451a调节剂在制备调控血压制剂中的应用
CN115920054A (zh) * 2022-12-23 2023-04-07 扬州大学 miR-144/451作为靶点在治疗精神分裂症中的应用

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2970968B1 (en) 2013-03-15 2018-01-10 Miragen Therapeutics, Inc. Bridged bicyclic nucleosides
WO2016094406A1 (en) * 2014-12-08 2016-06-16 Thomas Jefferson University Treatment and prevention of thrombosis using an anti-mir
US10306081B2 (en) * 2015-07-09 2019-05-28 Turner Broadcasting System, Inc. Technologies for generating a point-of-view video
ES2659845B1 (es) 2016-09-19 2019-01-04 Univ Valencia Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
EP4506010A1 (en) 2022-04-06 2025-02-12 Universitat de València Antagonists of human micro rna mir-100, mir-20, mir-222, mir-181 and mir-92 and uses of same

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007112754A2 (en) * 2006-04-03 2007-10-11 Santaris Pharma A/S Pharmaceutical compositions comprising anti-mirna antisense oligonucleotides

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1883709A4 (en) 2005-04-29 2010-10-13 Univ Rockefeller HUMAN MICRO-RNAS AND METHOD OF INHIBITING THEM
EP2285960B1 (en) 2008-05-08 2015-07-08 Asuragen, INC. Compositions and methods related to mir-184 modulation of neovascularization or angiogenesis
CA2726052A1 (en) * 2008-06-04 2009-12-10 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small rna targeting of gene promoters

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007112754A2 (en) * 2006-04-03 2007-10-11 Santaris Pharma A/S Pharmaceutical compositions comprising anti-mirna antisense oligonucleotides

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
HANA BRUCOVA ET AL: "Erythropoiesis in polycythemia vera is hyper-proliferative and has accelerated maturation", 《BLOOD CELLS, MOLECULES, AND DISEASES 》, vol. 43, 4 March 2009 (2009-03-04), pages 81 - 87, XP026153339, DOI: doi:10.1016/j.bcmd.2009.02.001 *
HANA BRUCOVA ET AL: "Regulated expression of microRNAs in normal and polycythemia vera erythropoiesis", 《EXPERIMENTAL HEMATOLOGY》, vol. 35, 31 December 2007 (2007-12-31) *
MEI ZHAN ET AL: "MicroRNA expression dynamics during murine and human erythroid differentiation", 《EXPERIMENTAL HEMATOLOGY》, vol. 35, 31 December 2007 (2007-12-31) *

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113332434A (zh) * 2021-05-25 2021-09-03 大连医科大学 miR-451a调节剂在制备调控血压制剂中的应用
CN113332434B (zh) * 2021-05-25 2023-09-05 大连医科大学 miR-451a调节剂在制备调控血压制剂中的应用
CN115920054A (zh) * 2022-12-23 2023-04-07 扬州大学 miR-144/451作为靶点在治疗精神分裂症中的应用

Also Published As

Publication number Publication date
US20140011859A1 (en) 2014-01-09
JP2013530160A (ja) 2013-07-25
EP2536436A4 (en) 2015-01-14
US9340782B2 (en) 2016-05-17
WO2012148373A1 (en) 2012-11-01
EP2536436A1 (en) 2012-12-26

Similar Documents

Publication Publication Date Title
US20240238326A1 (en) Compositions and methods for modulation of smn2 splicing in a subject
AU2018329190A1 (en) RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
US9340782B2 (en) Antimir-451 for the treatment of polycythemias
AU2021242321B2 (en) RNAi agents for inhibiting expression of PNPLA3, pharmaceutical compositions thereof, and methods of use
WO2018227247A1 (en) Compositions and methods for treating conditions associated with gain-of-function mutations in kcnt1
CA3192372A1 (en) Methods for the reduction of z-aat protein levels
CA2784918A1 (en) Antimir-451 for the treatment of polycythemias
EP4642913A2 (en) Splice-switching oligonucleotides for treating syngap1-associated disorders
WO2024182580A2 (en) Rnai agents for inhibiting expression of proprotein convertase subtilisin kexin 9 (pcsk9), pharmaceutical compositions thereof, and methods of use
WO2024192379A1 (en) Rnai agents for inhibiting expression of mitochondrial amidoxime reducing component 1 (marc1), pharmaceutical compositions thereof, and methods of use
HK40084771A (en) Rnai agents for inhibiting expression of pnpla3, pharmaceutical compositions thereof, and methods of use
OA19513A (en) RNAI agents and compositions for inhibiting expression of apolipoprotein C-lll (APOC3).

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
C05 Deemed withdrawal (patent law before 1993)
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20130109