CA2824604A1 - Compositions and methods for activating expression by a specific endogenous mirna - Google Patents

Compositions and methods for activating expression by a specific endogenous mirna Download PDF

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Publication number
CA2824604A1
CA2824604A1 CA2824604A CA2824604A CA2824604A1 CA 2824604 A1 CA2824604 A1 CA 2824604A1 CA 2824604 A CA2824604 A CA 2824604A CA 2824604 A CA2824604 A CA 2824604A CA 2824604 A1 CA2824604 A1 CA 2824604A1
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CA
Canada
Prior art keywords
mir
composition
exogenous
sequence
rna molecule
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.)
Abandoned
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CA2824604A
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English (en)
French (fr)
Inventor
Guy Abitbol
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NANODOC Ltd
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NANODOC Ltd
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Publication date
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Publication of CA2824604A1 publication Critical patent/CA2824604A1/en
Abandoned legal-status Critical Current

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    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl 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
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/635Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2320/00Applications; Uses
    • YGENERAL 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
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA2824604A 2010-10-28 2011-10-27 Compositions and methods for activating expression by a specific endogenous mirna Abandoned CA2824604A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
ILPCT/IL2010/000894 2010-10-28
PCT/IL2010/000894 WO2012056440A1 (en) 2010-10-28 2010-10-28 COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA
PCT/IL2011/000837 WO2012056457A2 (en) 2010-10-28 2011-10-27 Compositions and methods for activating expression by a specific endogenous mirna

Publications (1)

Publication Number Publication Date
CA2824604A1 true CA2824604A1 (en) 2012-05-03

Family

ID=45993239

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2824604A Abandoned CA2824604A1 (en) 2010-10-28 2011-10-27 Compositions and methods for activating expression by a specific endogenous mirna

Country Status (7)

Country Link
US (1) US20130245096A1 (enExample)
EP (1) EP2632932A4 (enExample)
JP (1) JP2013544511A (enExample)
CN (1) CN103314003A (enExample)
AU (1) AU2011322114A1 (enExample)
CA (1) CA2824604A1 (enExample)
WO (2) WO2012056440A1 (enExample)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2056845T3 (da) 2006-08-08 2017-11-27 Rheinische Friedrich-Wilhelms-Universität Bonn Struktur og anvendelse af 5'-phosphat-oligonukleotider
US8524248B2 (en) * 2007-12-14 2013-09-03 University of Pittsburgh—of the Commonwealth System of Higher Education Methods to diagnose and immunize against the virus causing human Merkel cell carcinoma
US9738680B2 (en) 2008-05-21 2017-08-22 Rheinische Friedrich-Wilhelms-Universität Bonn 5′ triphosphate oligonucleotide with blunt end and uses thereof
EP2508530A1 (en) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification of triphosphorylated oligonucleotides using capture tags
US10501791B2 (en) 2011-10-14 2019-12-10 President And Fellows Of Harvard College Sequencing by structure assembly
ES2953308T3 (es) 2011-12-22 2023-11-10 Harvard College Composiciones y métodos para la detección de analitos
WO2014163886A1 (en) 2013-03-12 2014-10-09 President And Fellows Of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
US9914967B2 (en) 2012-06-05 2018-03-13 President And Fellows Of Harvard College Spatial sequencing of nucleic acids using DNA origami probes
WO2014042251A1 (ja) * 2012-09-13 2014-03-20 中外製薬株式会社 遺伝子ノックイン非ヒト動物
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
MY177814A (en) 2013-06-04 2020-09-23 Harvard College Rna-guided transcriptional regulation
CN104419749B (zh) * 2013-08-22 2017-02-15 江苏命码生物科技有限公司 一种用于预测干扰素治疗慢性hbv疗效的微小核糖核酸及其应用
CN106574292B (zh) * 2014-01-10 2021-07-09 国立大学法人京都大学 利用作为指示剂的miRNA的表达区分期望的细胞类型的方法
US10179932B2 (en) 2014-07-11 2019-01-15 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
US20170362605A1 (en) * 2014-12-19 2017-12-21 Modernatx, Inc. Terminal modifications of polynucleotides
US9777064B2 (en) 2015-03-17 2017-10-03 Chimera Bioengineering, Inc. Smart CAR devices, DE CAR polypeptides, side CARs and uses thereof
JP6990176B2 (ja) 2015-10-05 2022-02-03 モデルナティエックス インコーポレイテッド メッセンジャーリボ核酸薬物の治療投与のための方法
GB2559526B (en) 2015-11-03 2021-02-17 Harvard College Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix
US11052111B2 (en) 2015-12-08 2021-07-06 Chimera Bioengineering, Inc. Smart CAR devices and DE CAR polypeptides for treating disease and methods for enhancing immune responses
EP4613756A3 (en) 2016-04-25 2025-11-12 President And Fellows Of Harvard College Hybridization chain reaction methods for in situ molecular detection
WO2017201019A1 (en) 2016-05-17 2017-11-23 Chimera Bioengineering, Inc. Methods for making novel antigen binding domains
US20190233844A1 (en) * 2016-07-26 2019-08-01 Senti Biosciences, Inc. Spatiotemporal regulators
CN118853848A (zh) 2016-08-31 2024-10-29 哈佛学院董事及会员团体 将生物分子的检测组合到使用荧光原位测序的单个试验的方法
GB2570412A (en) 2016-08-31 2019-07-24 Harvard College Methods of generating libraries of nucleic acid sequences for detection via fluorescent in situ sequencing
JP7045378B2 (ja) 2016-09-01 2022-03-31 キメラ・バイオエンジニアリング,インコーポレーテッド Gold最適化CAR T細胞
DE102017103383A1 (de) * 2017-02-20 2018-08-23 aReNA-Bio GbR (vertretungsberechtigter Gesellschafter: Dr. Heribert Bohlen, 50733 Köln) System und Verfahren zur Zelltyp-spezifischen Translation von RNA-Molekülen in Eukaryoten
CN109789167A (zh) 2017-04-14 2019-05-21 哈佛学院董事及会员团体 用于产生细胞衍生的微丝网络的方法
WO2019027869A1 (en) * 2017-07-31 2019-02-07 Massachusetts Institute Of Technology RNA-CLUSTER INDUCED TRANSCRIPT STABILIZER AND USES THEREOF
CN110016501A (zh) * 2018-01-09 2019-07-16 江苏命码生物科技有限公司 不明原因发热的标记物、检测方法及其应用
JP2021518747A (ja) 2018-02-13 2021-08-05 キメラ・バイオエンジニアリング,インコーポレーテッド Rna不安定化エレメントを使用した遺伝子発現の調整
CN108753836B (zh) * 2018-06-04 2021-10-12 北京大学 一种利用rna干扰机制的基因调控或编辑系统
CN108841864B (zh) * 2018-06-04 2021-10-15 北京大学 一种利用rna干扰机制的分子传感器
SG11202101934SA (en) 2018-07-30 2021-03-30 Readcoor Llc Methods and systems for sample processing or analysis
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
CN109628489B (zh) * 2019-01-07 2022-09-23 新乡医学院 一种提高cho细胞重组蛋白表达水平的方法及其应用,表达载体、表达系统及其制备方法
JP2022546282A (ja) 2019-08-18 2022-11-04 キメラ・バイオエンジニアリング,インコーポレーテッド Gold制御導入遺伝子による併用療法
CN111057790B (zh) * 2019-12-11 2022-08-30 石河子大学 miRNA在制备用于检测KSHV潜伏感染的试剂盒中的用途
US20230295654A1 (en) * 2020-07-17 2023-09-21 Children's Hospital Medical Center Methods and compositions for treatment of fragile x syndrome
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
WO2022182697A1 (en) * 2021-02-23 2022-09-01 Board Of Regents, The University Of Texas System A novel rna-based approach to cancer treatment
US20240384277A1 (en) * 2021-06-15 2024-11-21 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
US20250109397A1 (en) * 2021-11-30 2025-04-03 Kyoto University Rna molecule
CN116716351B (zh) * 2023-03-30 2024-02-23 湖北天勤生物技术研究院有限公司 用于构建食蟹猴阿尔兹海默症模型的组合物及其应用和构建方法

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001072995A2 (en) * 2000-03-28 2001-10-04 University Of Rochester Methods of producing a library and methods of selecting polynucletides
US20060275262A1 (en) * 2001-07-26 2006-12-07 Mathis James M Conditionally replicating viruses and methods for cancer virotherapy
DE10158517A1 (de) * 2001-11-29 2003-06-12 Focusgenomics Gmbh Verfahren zur Analyse der translationskontrollierten Genexpression
US20070265220A1 (en) * 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
AU2005323166B2 (en) * 2004-12-21 2011-11-10 Monsanto Technology, Llc Recombinant DNA constructs and methods for controlling gene expression
US20060265771A1 (en) * 2005-05-17 2006-11-23 Lewis David L Monitoring microrna expression and function
US20070179113A1 (en) * 2005-05-19 2007-08-02 Schering Aktiengesellachaft GM-CSF gene therapy for Crohn's disease using an improved regulated expression system
US20070054872A1 (en) * 2005-08-24 2007-03-08 Mirus Bio Corporation Regulatable or conditional expression systems
US20090156535A1 (en) * 2007-09-27 2009-06-18 The Trustees Of Princeton University MicroRNAs for Modulating Herpes Virus Gene Expression
US20090286242A1 (en) * 2007-12-10 2009-11-19 Cold Spring Harbor Laboratory MicroRNA Expression Profiling and Uses Thereof
CN102099472A (zh) * 2008-05-19 2011-06-15 新加坡科技研究局 使基因表达靶向于神经胶质瘤的核酸分子和方法
EP2364362B1 (en) * 2008-11-12 2015-10-21 Ospedale San Raffaele S.r.l. Gene vector for inducing transgene-specific immune tolerance

Also Published As

Publication number Publication date
US20130245096A1 (en) 2013-09-19
WO2012056457A3 (en) 2012-08-02
EP2632932A2 (en) 2013-09-04
WO2012056457A2 (en) 2012-05-03
JP2013544511A (ja) 2013-12-19
CN103314003A (zh) 2013-09-18
EP2632932A4 (en) 2014-12-17
AU2011322114A1 (en) 2013-05-30
WO2012056440A1 (en) 2012-05-03

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FZDE Discontinued

Effective date: 20161027