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

Info

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
Authority
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
Application number
CA2824604A
Other languages
English (en)
French (fr)
Inventor
Guy Abitbol
Original Assignee
NANODOC Ltd
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 NANODOC Ltd filed Critical NANODOC Ltd
Publication of CA2824604A1 publication Critical patent/CA2824604A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • A61K48/005—Medicinal 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
    • 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
    • A61K48/005—Medicinal 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
    • A61K48/0066—Manipulation 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
    • 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
    • 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
    • C07H21/04—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 deoxyribosyl 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
    • 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
    • C12N15/635—Externally inducible repressor mediated regulation of gene expression, e.g. tetR inducible by tetracyline
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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 nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • 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
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (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
PCT/IL2010/000894 WO2012056440A1 (en) 2010-10-28 2010-10-28 COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA
ILPCT/IL2010/000894 2010-10-28
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 (https=)
EP (1) EP2632932A4 (https=)
JP (1) JP2013544511A (https=)
CN (1) CN103314003A (https=)
AU (1) AU2011322114A1 (https=)
CA (1) CA2824604A1 (https=)
WO (2) WO2012056440A1 (https=)

Families Citing this family (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2911034T3 (es) 2006-08-08 2022-05-17 Univ Bonn Rheinische Friedrich Wilhelms Estructura y uso de oligonucleótidos 5' fosfato
WO2009079481A2 (en) 2007-12-14 2009-06-25 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods to diagnose and immunize against the virus causing human merkel cell carcinoma
WO2009141146A1 (en) 2008-05-21 2009-11-26 Gunther Hartmann 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
EP3604555B1 (en) 2011-10-14 2024-12-25 President and Fellows of Harvard College Sequencing by structure assembly
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
EP4249605B1 (en) 2011-12-22 2024-08-28 President And Fellows Of Harvard College Methods for analyte detection
US9914967B2 (en) 2012-06-05 2018-03-13 President And Fellows Of Harvard College Spatial sequencing of nucleic acids using DNA origami probes
EP2896291B1 (en) * 2012-09-13 2019-03-20 Chugai Seiyaku Kabushiki Kaisha Gene knock-in non-human animal
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
EP2971184B1 (en) 2013-03-12 2019-04-17 President and Fellows of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
SG10201913068PA (en) 2013-06-04 2020-02-27 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
WO2015105172A1 (ja) * 2014-01-10 2015-07-16 国立大学法人京都大学 ｍｉＲＮＡの発現を指標として所望の細胞種を判別する方法
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
EP3233132A4 (en) * 2014-12-19 2018-06-27 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
EP3359670B2 (en) 2015-10-05 2024-02-14 ModernaTX, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
AU2016349288A1 (en) 2015-11-03 2018-05-31 President And Fellows Of 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
EP3458077A4 (en) 2016-05-17 2020-04-01 Chimera Bioengineering Inc. METHODS OF MANUFACTURING NEW AREAS OF ANTIGEN BINDING
WO2018022747A1 (en) * 2016-07-26 2018-02-01 Senti Biosciences, Inc. Genetic erasers
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
CN118389650A (zh) 2016-08-31 2024-07-26 哈佛学院董事及会员团体 生成用于通过荧光原位测序检测的核酸序列文库的方法
CN112481217A (zh) * 2016-09-01 2021-03-12 嵌合体生物工程公司 Gold优化的car t-细胞
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
US11185568B2 (en) 2017-04-14 2021-11-30 President And Fellows Of Harvard College Methods for generation of cell-derived microfilament network
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
CN111989108B (zh) 2018-02-13 2024-07-16 嵌合体生物工程公司 利用rna去稳定元件协调基因表达
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
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
CN112770776B (zh) 2018-07-30 2025-08-19 瑞德库尔有限责任公司 用于样品处理或分析的方法和系统
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
EP4097251A1 (en) 2020-01-29 2022-12-07 10X Genomics, Inc. Compositions and methods for analyte detection
CA3189657A1 (en) * 2020-07-17 2022-01-20 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
EP4355882A2 (en) * 2021-06-15 2024-04-24 Modernatx, Inc. Engineered polynucleotides for cell-type or microenvironment-specific expression
WO2023056194A2 (en) 2021-09-29 2023-04-06 Chimera Bioengineering, Inc. Compositions and methods for anti-tnmuc1 gold car t-cells
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
EP1268763A2 (en) * 2000-03-28 2003-01-02 University Of Rochester Methods of producing a library and methods of selecting polynucleotides
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
EP2765189A1 (en) * 2004-12-21 2014-08-13 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
EP2632932A2 (en) 2013-09-04
CN103314003A (zh) 2013-09-18
AU2011322114A1 (en) 2013-05-30
EP2632932A4 (en) 2014-12-17
JP2013544511A (ja) 2013-12-19
WO2012056457A2 (en) 2012-05-03
WO2012056457A3 (en) 2012-08-02
WO2012056440A1 (en) 2012-05-03

Similar Documents

Publication Publication Date Title
US20130245096A1 (en) COMPOSITIONS AND METHODS FOR ACTIVATING EXPRESSION BY A SPECIFIC ENDOGENOUS miRNA
ES2969371T3 (es) Interferencia por ARN para el tratamiento de trastornos de ganancia de función
JP5986928B2 (ja) 遺伝子発現の特異的阻害のためのダイサー基質剤及び方法
US8486910B2 (en) SnoRNAi-small nucleolar RNA degradation by RNA interference in trypanosomatids
CN109415728A (zh) 逆转录病毒核酸序列的切除
US20130225660A1 (en) Compositions and methods for specific cleavage of exogenous rna in a cell
CA3184043A1 (en) Mirna-485 inhibitor for huntington's disease
CA3166604A1 (en) Use of mirna-485 inhibitors for treating tauopathy
CN121464217A (zh) 用于治疗KIF1A相关神经紊乱的靶向KIF1A错义突变的RNAi
WO2022264038A1 (en) Use of mirna-485 inhibitors for treating inflammasome-related diseases or disorders
US20240117350A1 (en) Use of mirna-485 inhibitor to regulate psd95, synaptophysin, and caspase-3 expression
WO2021156832A1 (en) Use of mirna-485 inhibitors for treating amyotrophic lateral sclerosis (als)
WO2024201420A1 (en) Mirna-484 inhibitors and uses thereof
US20230119699A1 (en) Diagnostic methods using sirt1 expression
WO2024228118A1 (en) Mirna-485 inhibitors
EP4118435A1 (en) Diagnostic methods using pgc-1? expression
WO2022168007A1 (en) Use of mirna-485 inhibitors for treating diseases or disorders associated with abnormal nlrp3 expression

Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20161027