IL265461B2 - Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor - Google Patents

Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor

Info

Publication number
IL265461B2
IL265461B2 IL265461A IL26546119A IL265461B2 IL 265461 B2 IL265461 B2 IL 265461B2 IL 265461 A IL265461 A IL 265461A IL 26546119 A IL26546119 A IL 26546119A IL 265461 B2 IL265461 B2 IL 265461B2
Authority
IL
Israel
Prior art keywords
mir
expression
seq
antagomir
oligoribonucleotide
Prior art date
Application number
IL265461A
Other languages
English (en)
Hebrew (he)
Other versions
IL265461B1 (en
IL265461A (en
Original Assignee
Univ Valencia
Aum Lifetech 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
Application filed by Univ Valencia, Aum Lifetech Inc filed Critical Univ Valencia
Publication of IL265461A publication Critical patent/IL265461A/en
Publication of IL265461B1 publication Critical patent/IL265461B1/en
Publication of IL265461B2 publication Critical patent/IL265461B2/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7115Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/113Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Psychology (AREA)
  • Neurosurgery (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (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)
IL265461A 2016-09-19 2017-09-19 Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor IL265461B2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
ES201631216A ES2659845B1 (es) 2016-09-19 2016-09-19 Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
PCT/EP2017/073685 WO2018050930A1 (en) 2016-09-19 2017-09-19 Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor

Publications (3)

Publication Number Publication Date
IL265461A IL265461A (en) 2019-05-30
IL265461B1 IL265461B1 (en) 2024-02-01
IL265461B2 true IL265461B2 (en) 2024-06-01

Family

ID=59974404

Family Applications (1)

Application Number Title Priority Date Filing Date
IL265461A IL265461B2 (en) 2016-09-19 2017-09-19 Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor

Country Status (8)

Country Link
US (2) US11202794B2 (https=)
EP (2) EP4269587A3 (https=)
JP (1) JP6919098B2 (https=)
AU (2) AU2017328728B2 (https=)
CA (1) CA3037288C (https=)
ES (1) ES2659845B1 (https=)
IL (1) IL265461B2 (https=)
WO (1) WO2018050930A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
JP7432929B2 (ja) * 2018-05-31 2024-02-19 コリア ユニバーシティ リサーチ アンド ビジネス ファウンデーション マイクロrnaの非正規標的を抑制するrna干渉誘導核酸およびその用途
US20210340537A1 (en) * 2020-04-15 2021-11-04 Duquesne University Of The Holy Spirit AN RNA G-QUADRUPLEX STRUCTURE IN PRE-miRNA-1229 AS A THERAPEUTIC TARGET FOR ALZHEIMER'S DISEASE AND VARIOUS CANCERS
WO2023194641A1 (es) 2022-04-06 2023-10-12 Universitat De València Antagonistas de los microrna humanos mir-100, mir-20, mir-222, mir-181, y mir-92 y usos de los mismos
ES2954008A1 (es) * 2022-04-06 2023-11-17 Univ Valencia Antagonistas de los microRNA humanos miR-100, miR-20, miR-222, miR-181, y miR-92 y usos de los mismos
EP4282964A1 (en) * 2022-05-23 2023-11-29 Universitat de Valéncia Oligonucleotides conjugated to oleic acid and uses thereof
ES3014830A1 (es) * 2023-10-20 2025-04-25 Arthex Biotech S L Oligonucleotidos o analogos de los mismos para el tratamiento de la distrofia del endotelio corneal de fuchs
CN117467664B (zh) * 2023-11-07 2024-06-21 江苏省家禽科学研究所 一种靶向鸡mbnl1基因的反义寡核苷酸及其应用
WO2025114442A1 (en) * 2023-11-28 2025-06-05 Arthex Biotech S.L. Oligonucleotides conjugated to oleic acid and uses thereof

Citations (3)

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WO2005013901A2 (en) * 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
WO2008116267A1 (en) * 2007-03-26 2008-10-02 Crc For Asthma And Airways Ltd Therapeutic targets and molecules
WO2013159091A2 (en) * 2012-04-20 2013-10-24 Aptamir Therapeutics, Inc. Mirna modulators of thermogenesis

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US5981505A (en) 1993-01-26 1999-11-09 The Trustees Of The University Of Pennsylvania Compositions and methods for delivery of genetic material
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5840710A (en) 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
US6217900B1 (en) 1997-04-30 2001-04-17 American Home Products Corporation Vesicular complexes and methods of making and using the same
DE69834038D1 (de) 1997-07-01 2006-05-18 Isis Pharmaceutical Inc Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
WO2003093449A2 (en) 2002-05-06 2003-11-13 Nucleonics, Inc. Methods for delivery of nucleic acids
EP1723162A4 (en) * 2004-02-13 2010-05-05 Univ Rockefeller ANTI-microRNA oligonucleotide molecules
WO2006069584A2 (en) * 2004-12-29 2006-07-06 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF microRNAs AND THEIR TARGET mRNAs
EP1984499B1 (en) * 2006-01-27 2015-05-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of micrornas
CA2648132C (en) * 2006-04-03 2019-05-28 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
EP3034083B1 (en) * 2006-09-21 2020-12-09 University of Rochester Antisense oligonucleotides for use in treating myotonic dystrophy
WO2009012263A2 (en) * 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
JPWO2009148137A1 (ja) 2008-06-04 2011-11-04 協和発酵キリン株式会社 肥満細胞の脱顆粒を制御する核酸
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
US8609615B2 (en) * 2008-10-15 2013-12-17 Valerion Therapeutics, Llc Methods and compositions for treatment of myotonic dystrophy
US8852940B2 (en) * 2009-04-01 2014-10-07 The Regents Of The University Of California Embryonic stem cell specific microRNAs promote induced pluripotency
WO2010139026A1 (en) * 2009-06-05 2010-12-09 Centenary Institute Of Cancer Medicine And Cell Biology Therapeutic and diagnostic molecules
EP2275545A1 (en) * 2009-07-16 2011-01-19 Julius-Maximilians-Universität Würzburg Use of microRNA-24 and/or its targets for the treatment and prevention of ischemia and induction of angiogenesis
CN102869386A (zh) 2010-01-20 2013-01-09 得克萨斯系统大学董事会 用于治疗红细胞增多症的antimiR-451
WO2012012467A2 (en) * 2010-07-19 2012-01-26 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US8729046B2 (en) * 2011-12-07 2014-05-20 Albert Einstein College Of Medicine Of Yeshiva University MIR27B is a novel target for treatment of liver fibrosis
KR20130083965A (ko) * 2012-01-16 2013-07-24 김승찬 시험관내 배양된 세포주 에 hsa-miR-218 miRNA에 대한 DNA antisense oligomer 처리 및 신호전달체계 변경 방법
GB201202228D0 (en) * 2012-02-08 2012-03-28 Queen Mary & Westfield College Reversal of replicative senescence
EP3230454A1 (en) 2014-12-09 2017-10-18 Pierfrancesco Tassone Inhibitors of mir-17-92 cluster for anti-tumor activity in multiple myeloma and other malignancies

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005013901A2 (en) * 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
WO2008116267A1 (en) * 2007-03-26 2008-10-02 Crc For Asthma And Airways Ltd Therapeutic targets and molecules
WO2013159091A2 (en) * 2012-04-20 2013-10-24 Aptamir Therapeutics, Inc. Mirna modulators of thermogenesis

Also Published As

Publication number Publication date
JP2019531092A (ja) 2019-10-31
JP6919098B2 (ja) 2021-08-18
AU2017328728B2 (en) 2021-07-29
ES2659845A1 (es) 2018-03-19
EP3516059A1 (en) 2019-07-31
IL265461B1 (en) 2024-02-01
IL265461A (en) 2019-05-30
CA3037288A1 (en) 2018-03-22
ES2659845B1 (es) 2019-01-04
WO2018050930A1 (en) 2018-03-22
AU2017328728A1 (en) 2019-05-16
US20220133774A1 (en) 2022-05-05
EP4269587A2 (en) 2023-11-01
CA3037288C (en) 2024-01-16
AU2021257890A1 (en) 2021-11-18
US20190231809A1 (en) 2019-08-01
EP4269587A3 (en) 2024-07-10
US11202794B2 (en) 2021-12-21

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