CN101981189A - 能够以协同方式引起基因表达转录后沉默的方法和组合物 - Google Patents

能够以协同方式引起基因表达转录后沉默的方法和组合物 Download PDF

Info

Publication number
CN101981189A
CN101981189A CN2009801118031A CN200980111803A CN101981189A CN 101981189 A CN101981189 A CN 101981189A CN 2009801118031 A CN2009801118031 A CN 2009801118031A CN 200980111803 A CN200980111803 A CN 200980111803A CN 101981189 A CN101981189 A CN 101981189A
Authority
CN
China
Prior art keywords
polynucleotide
composition
mrna
target
vector
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
CN2009801118031A
Other languages
English (en)
Chinese (zh)
Inventor
哈比尔·阿瓦德略雷特
玛丽亚普里菲卡西·福特斯阿隆索
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.)
Proyecto de Biomedicina CIMA SL
Original Assignee
Proyecto de Biomedicina CIMA SL
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 Proyecto de Biomedicina CIMA SL filed Critical Proyecto de Biomedicina CIMA SL
Publication of CN101981189A publication Critical patent/CN101981189A/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
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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]
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • AIDS & HIV (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Tropical Medicine & Parasitology (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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN2009801118031A 2008-01-29 2009-01-29 能够以协同方式引起基因表达转录后沉默的方法和组合物 Pending CN101981189A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
ESP200800222 2008-01-29
ES200800222 2008-01-29
PCT/ES2009/000045 WO2009095517A2 (es) 2008-01-29 2009-01-29 Métodos y composiciones capaces de provocar un silenciamiento post-transcripcional de la expresión génica de forma sinérgica

Publications (1)

Publication Number Publication Date
CN101981189A true CN101981189A (zh) 2011-02-23

Family

ID=40807539

Family Applications (1)

Application Number Title Priority Date Filing Date
CN2009801118031A Pending CN101981189A (zh) 2008-01-29 2009-01-29 能够以协同方式引起基因表达转录后沉默的方法和组合物

Country Status (10)

Country Link
US (1) US20110030075A1 (enExample)
EP (1) EP2246432A2 (enExample)
JP (1) JP2011510647A (enExample)
CN (1) CN101981189A (enExample)
AU (1) AU2009209571A1 (enExample)
BR (1) BRPI0906657A2 (enExample)
CA (1) CA2713458A1 (enExample)
MX (1) MX2010008326A (enExample)
RU (1) RU2010136040A (enExample)
WO (1) WO2009095517A2 (enExample)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105324392A (zh) * 2013-04-20 2016-02-10 全国儿童医院研究所 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
CN107746857A (zh) * 2017-08-07 2018-03-02 中国农业大学 一种抑制基因表达的rna干扰方法
CN108893487A (zh) * 2018-07-19 2018-11-27 中国农业科学院北京畜牧兽医研究所 一种含有C-Myc蛋白融合标签的植物表达质粒载体及其载体的构建方法
CN113924364A (zh) * 2019-03-08 2022-01-11 罗格斯新泽西州立大学 治疗亨廷顿病的组合物和方法
CN114159459A (zh) * 2021-12-20 2022-03-11 塔里木大学 miR-26a在制备治疗子宫内膜炎的药物中的应用

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9441221B2 (en) 2007-03-30 2016-09-13 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
US8343941B2 (en) 2007-03-30 2013-01-01 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
US8907075B2 (en) 2007-03-30 2014-12-09 Samuel Ian Gunderson Compositions and methods for gene silencing
WO2012045894A1 (es) * 2010-10-05 2012-04-12 Proyecto De Biomedicina Cima, S.L. Compuestos y composiciones para el tratamiento de enfermedades debidas a la infección por el virus de la hepatitis b
EP2853595A1 (en) * 2013-09-30 2015-04-01 Soluventis GmbH NOTCH 1 specific siRNA molecules
EP4004204A1 (en) 2019-07-23 2022-06-01 Oxford BioMedica (UK) Limited Enhancing production of lentiviral vectors

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3626187B2 (ja) 1993-06-07 2005-03-02 バイカル インコーポレイテッド 遺伝子治療に適するプラスミド
CA2225460A1 (en) 1995-06-23 1997-01-09 Winston Campbell Patterson Transcriptional regulation of genes encoding vascular endothelial growth factor receptors
US6692910B2 (en) 1997-07-28 2004-02-17 The University Of Connecticut Inhibition of a target messenger RNA with a modified U1 small nuclear RNA
EP1384784A1 (en) * 2001-03-30 2004-01-28 Instituto Cientifico Y Tecnologico De Navarra, S.A. Method for reversible inhibition of gene expression by modified ribonucleoproteins
EP2428570B1 (en) 2001-09-28 2018-05-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Microrna molecules
CA2524569C (en) 2002-05-03 2013-10-22 Duke University A method of regulating gene expression
WO2005026322A2 (en) 2003-09-11 2005-03-24 Clontech Laboratories, Inc. siRNA ENCODING CONSTRUCTS AND METHODS FOR USING THE SAME
US20080131940A1 (en) 2004-06-25 2008-06-05 Robert Chiu Target Cell-Specific Short Interfering Rna and Methods of Use Thereof
WO2006135436A2 (en) 2004-10-22 2006-12-21 University Of Florida Research Foundation, Inc. Inhibition of gene expression and therapeutic uses thereof
PT3272872T (pt) 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
EP1969000A2 (en) 2005-12-06 2008-09-17 Centre National de la Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
GB0701722D0 (en) * 2007-01-30 2007-03-07 Scottish Crop Res Inst Inhibition of gene expression

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105324392A (zh) * 2013-04-20 2016-02-10 全国儿童医院研究所 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
CN107746857A (zh) * 2017-08-07 2018-03-02 中国农业大学 一种抑制基因表达的rna干扰方法
CN108893487A (zh) * 2018-07-19 2018-11-27 中国农业科学院北京畜牧兽医研究所 一种含有C-Myc蛋白融合标签的植物表达质粒载体及其载体的构建方法
CN113924364A (zh) * 2019-03-08 2022-01-11 罗格斯新泽西州立大学 治疗亨廷顿病的组合物和方法
CN114159459A (zh) * 2021-12-20 2022-03-11 塔里木大学 miR-26a在制备治疗子宫内膜炎的药物中的应用

Also Published As

Publication number Publication date
MX2010008326A (es) 2010-08-18
JP2011510647A (ja) 2011-04-07
EP2246432A2 (en) 2010-11-03
US20110030075A1 (en) 2011-02-03
WO2009095517A3 (es) 2009-09-24
RU2010136040A (ru) 2012-03-10
CA2713458A1 (en) 2009-08-06
AU2009209571A1 (en) 2009-08-06
BRPI0906657A2 (pt) 2015-07-14
WO2009095517A2 (es) 2009-08-06

Similar Documents

Publication Publication Date Title
US20110030075A1 (en) Methods and Compositions Capable of Causing Post-Transcriptional Silencing of Gene Expression in a Synergic Manner
US9523093B2 (en) Huntington's disease therapeutic compounds
US10006028B2 (en) Alternative export pathways for vector expressed RNA interference
US20090036395A1 (en) Rna interference suppression of neurodegenerative diseases and methods of use thereof
KR20140071949A (ko) Hbv 치료
JP2013509168A (ja) 心臓特異的核酸調節因子ならびにこの方法および使用
JP2024543782A (ja) Ｃａｇリピート疾患を治療するための組成物および方法
JP7862005B2 (ja) 環状ｒｎａ発現のための組成物および方法
WO2004016780A1 (en) Adeno-associated virus vector inhibiting gene's function by rnai
WO2025202080A1 (en) Acta1 expression control elements
CN118320132A (zh) 重组腺相关病毒rAAV2/Rec2作为胰腺靶向性载体的应用
WO2007100091A1 (ja) フコシルトランスフェラーゼの発現を抑制するための組成物
Li Modulation of Myocardial Genes via Use of Adenoviral Vectors and RNA Interference Approaches
HK1236224A1 (en) Huntington's disease therapeutic compounds
HK1236224B (en) Huntington's disease therapeutic compounds

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
AD01 Patent right deemed abandoned
AD01 Patent right deemed abandoned

Effective date of abandoning: 20110223