MX2016004142A - Moleculas de ácido ribonucleico pequeño de interferencia especifico de notch 1. - Google Patents

Moleculas de ácido ribonucleico pequeño de interferencia especifico de notch 1.

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Publication number
MX2016004142A
MX2016004142A MX2016004142A MX2016004142A MX2016004142A MX 2016004142 A MX2016004142 A MX 2016004142A MX 2016004142 A MX2016004142 A MX 2016004142A MX 2016004142 A MX2016004142 A MX 2016004142A MX 2016004142 A MX2016004142 A MX 2016004142A
Authority
MX
Mexico
Prior art keywords
nucleotide
strand
indicates
notch
sirna molecule
Prior art date
Application number
MX2016004142A
Other languages
English (en)
Inventor
Schreiber Sören
Original Assignee
Soluventis Gmbh
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 Soluventis Gmbh filed Critical Soluventis Gmbh
Publication of MX2016004142A publication Critical patent/MX2016004142A/es

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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

Abstract

La presente invención se refiere a una molécula de ácido nucleico que comprende una estructura de doble hebra, en donde la estructura de doble hebra está formada por una primera hebra y una segunda hebra, en donde la primera hebra consiste de la siguiente secuencia de nucleótidos 5'acGaGcUgGaCcAcUgGuCdTSdT3', y la segunda hebra consiste de la siguiente secuencia de nucleótidos 5'GAcCaGuGgUcCaGcUcGudTSdT3', en donde un nucleótido menor indica que el nucleótido es 2'-F modificado y un nucleótido subrayado indica que el nucleótido es 2'-O-metilo modificado y en donde dTSdT indica que en el extremo 3' está unido un dinucleótido que consiste de dos nucleótidos dT, en donde dichos dos dTs están unidos covalentemente a través de un enlace de fosforotioato.
MX2016004142A 2013-09-30 2014-09-30 Moleculas de ácido ribonucleico pequeño de interferencia especifico de notch 1. MX2016004142A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP13004722.8A EP2853595A1 (en) 2013-09-30 2013-09-30 NOTCH 1 specific siRNA molecules
PCT/EP2014/002655 WO2015043768A1 (en) 2013-09-30 2014-09-30 Notch 1 specific sirna molecule

Publications (1)

Publication Number Publication Date
MX2016004142A true MX2016004142A (es) 2016-10-13

Family

ID=49304652

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016004142A MX2016004142A (es) 2013-09-30 2014-09-30 Moleculas de ácido ribonucleico pequeño de interferencia especifico de notch 1.

Country Status (16)

Country Link
US (2) US9896690B2 (es)
EP (2) EP2853595A1 (es)
JP (2) JP6561061B2 (es)
CN (2) CN105814202B (es)
AU (1) AU2014327809B2 (es)
CA (1) CA2924788A1 (es)
DK (1) DK3052625T3 (es)
HR (1) HRP20211525T1 (es)
HU (1) HUE056609T2 (es)
IL (1) IL244661B (es)
LT (1) LT3052625T (es)
MX (1) MX2016004142A (es)
RU (1) RU2651493C2 (es)
SG (2) SG10201802593WA (es)
SI (1) SI3052625T1 (es)
WO (1) WO2015043768A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2853595A1 (en) * 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules
EP4083213A4 (en) * 2019-12-27 2024-01-10 Univ Kobe Nat Univ Corp MEDICINAL PRODUCTS FOR CANCER THERAPY
CN112626068B (zh) * 2020-12-21 2021-12-03 徐州医科大学 一种靶向Notch1基因的LncRNA及其应用
CN116790603B (zh) * 2023-08-18 2023-10-31 成都中科奥格生物科技有限公司 一种sgRNA、CRISPR/Cas9载体及其构建方法和用途

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
ATE204879T1 (de) 1991-12-24 2001-09-15 Isis Pharmaceuticals Inc Antisense oligonukleotide
WO1994002595A1 (en) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US6001311A (en) 1997-02-05 1999-12-14 Protogene Laboratories, Inc. Apparatus for diverse chemical synthesis using two-dimensional array
US6395713B1 (en) 1997-07-23 2002-05-28 Ribozyme Pharmaceuticals, Inc. Compositions for the delivery of negatively charged molecules
JP2003525017A (ja) 1998-04-20 2003-08-26 リボザイム・ファーマシューティカルズ・インコーポレーテッド 遺伝子発現を調節しうる新規な化学組成を有する核酸分子
WO2000053722A2 (en) 1999-03-10 2000-09-14 Phogen Limited Delivery of nucleic acids and proteins to cells
EP2258847B2 (en) 2002-08-05 2020-07-01 Silence Therapeutics GmbH Futher novel forms of interfering RNA molecules
MXPA06012722A (es) 2004-05-05 2007-02-14 Atugen Ag Lipidos, complejos de lipido y su uso.
US20110030075A1 (en) * 2008-01-29 2011-02-03 Proyecto De Biomedicina Cima S.L. Methods and Compositions Capable of Causing Post-Transcriptional Silencing of Gene Expression in a Synergic Manner
EP2250265A1 (en) * 2008-03-12 2010-11-17 Intradigm Corporation Compositions comprising notch1 sirna and methods of use thereof
EP2123256A1 (en) 2008-05-19 2009-11-25 Ruhr-Universität Bochum Perfluorcarbon nanoemulsions with endocytosis enhancing surface for gene-transfer
CN104651408A (zh) * 2009-06-15 2015-05-27 阿尔尼拉姆医药品有限公司 靶向pcsk9基因的脂质配制的dsrna
JP2014528704A (ja) * 2011-08-03 2014-10-30 クォーク ファーマシューティカルズ インコーポレーティッドＱｕａｒｋ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 聴覚およびバランス障害を治療するための二本鎖オリゴヌクレオチド化合物
CN104080912A (zh) * 2012-01-12 2014-10-01 夸克制药公司 用于治疗听力和平衡障碍的组合疗法
EP2853595A1 (en) * 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules

Also Published As

Publication number Publication date
SG10201802593WA (en) 2018-05-30
WO2015043768A1 (en) 2015-04-02
RU2016116931A (ru) 2017-11-10
US20160215292A1 (en) 2016-07-28
HRP20211525T1 (hr) 2021-12-24
HUE056609T2 (hu) 2022-02-28
LT3052625T (lt) 2021-12-10
US9896690B2 (en) 2018-02-20
DK3052625T3 (da) 2021-11-15
JP2019196384A (ja) 2019-11-14
CN111197046A (zh) 2020-05-26
SI3052625T1 (sl) 2021-12-31
US20180237785A1 (en) 2018-08-23
CA2924788A1 (en) 2015-04-02
RU2651493C2 (ru) 2018-04-19
AU2014327809A1 (en) 2016-04-07
EP2853595A1 (en) 2015-04-01
IL244661A0 (en) 2016-04-21
CN105814202B (zh) 2020-03-06
JP2016533190A (ja) 2016-10-27
EP3052625B1 (en) 2021-08-25
US10669548B2 (en) 2020-06-02
JP6561061B2 (ja) 2019-08-14
IL244661B (en) 2021-08-31
SG11201602437VA (en) 2016-04-28
CN105814202A (zh) 2016-07-27
WO2015043768A8 (en) 2016-05-12
EP3052625A1 (en) 2016-08-10
AU2014327809B2 (en) 2021-11-18
JP7086039B2 (ja) 2022-06-17

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